RAVULIZUMAB ( DrugBank: Ravulizumab )
7 diseases
| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 2 | 筋萎縮性側索硬化症 | 10 |
| 11 | 重症筋無力症 | 11 |
| 13 | 多発性硬化症／視神経脊髄炎 | 12 |
| 50 | 皮膚筋炎／多発性筋炎 | 6 |
| 62 | 発作性夜間ヘモグロビン尿症 | 52 |
| 66 | IgA腎症 | 5 |
| 109 | 非典型溶血性尿毒症症候群 | 7 |
2. 筋萎縮性側索硬化症
臨床試験数 : 645 / 薬物数 : 589 - (DrugBank : 163) / 標的遺伝子数 : 150 - 標的パスウェイ数 : 225
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2019-004619-30-IE (EUCTR) | 14/09/2020 | 03/02/2020 | An Efficacy and Safety Study of Ravulizumab in ALS Patients | A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Ravulizumab in Patients With Amyotrophic Lateral Sclerosis (ALS) - An Efficacy and Safety Study of Ravulizumab in ALS Patients | Amyotrophic Lateral Sclerosis (ALS), motor neuron disease MedDRA version: 21.1;Level: PT;Classification code 10002026;Term: Amyotrophic lateral sclerosis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Ultomiris Product Name: ravulizumab Product Code: ALXN1210 INN or Proposed INN: RAVULIZUMAB | Alexion Pharmaceuticals | NULL | Not Recruiting | Female: yes Male: yes | 354 | Phase 3 | United States;Spain;Ireland;Israel;United Kingdom;Switzerland;Italy;France;Canada;Belgium;Poland;Australia;Denmark;Germany;Netherlands;Japan;Sweden;Korea, Republic of | ||
| 2 | EUCTR2019-004619-30-DK (EUCTR) | 12/08/2020 | 03/03/2020 | An Efficacy and Safety Study of Ravulizumab in ALS Patients | A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Ravulizumab in Patients With Amyotrophic Lateral Sclerosis (ALS) - An Efficacy and Safety Study of Ravulizumab in ALS Patients | Amyotrophic Lateral Sclerosis (ALS), motor neuron disease MedDRA version: 21.1;Level: PT;Classification code 10002026;Term: Amyotrophic lateral sclerosis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Ultomiris Product Name: ravulizumab Product Code: ALXN1210 INN or Proposed INN: RAVULIZUMAB | Alexion Pharmaceuticals | NULL | Not Recruiting | Female: yes Male: yes | 354 | Phase 3 | France;Canada;Belgium;Poland;Denmark;Australia;Germany;Netherlands;Japan;Sweden;United States;Spain;Ireland;Israel;United Kingdom;Switzerland;Italy | ||
| 3 | EUCTR2019-004619-30-DE (EUCTR) | 07/08/2020 | 11/02/2020 | An Efficacy and Safety Study of Ravulizumab in ALS Patients | A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Ravulizumab in Patients With Amyotrophic Lateral Sclerosis (ALS) - An Efficacy and Safety Study of Ravulizumab in ALS Patients | Amyotrophic Lateral Sclerosis (ALS), motor neuron disease MedDRA version: 21.1;Level: PT;Classification code 10002026;Term: Amyotrophic lateral sclerosis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Ultomiris Product Name: ravulizumab Product Code: ALXN1210 INN or Proposed INN: RAVULIZUMAB | Alexion Pharmaceuticals | NULL | Not Recruiting | Female: yes Male: yes | 354 | Phase 3 | United States;Spain;Ireland;Israel;United Kingdom;Switzerland;Italy;France;Canada;Belgium;Poland;Australia;Denmark;Germany;Netherlands;Japan;Sweden | ||
| 4 | EUCTR2019-004619-30-IT (EUCTR) | 03/07/2020 | 17/01/2022 | An Efficacy and Safety Study of Ravulizumab in ALS Patients | A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Ravulizumab in Patients With Amyotrophic Lateral Sclerosis (ALS) - An Efficacy and Safety Study of Ravulizumab in ALS Patients | Amyotrophic Lateral Sclerosis (ALS), motor neuron disease MedDRA version: 21.1;Level: PT;Classification code 10002026;Term: Amyotrophic lateral sclerosis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Ultomiris Product Name: ravulizumab Product Code: [ALXN1210] INN or Proposed INN: RAVULIZUMAB Other descriptive name: RAVULIZUMAB | ALEXION PHARMACEUTICALS INCORPORATED | NULL | Not Recruiting | Female: yes Male: yes | 354 | Phase 3 | United States;Spain;Ireland;Israel;United Kingdom;Switzerland;Italy;France;Canada;Belgium;Poland;Australia;Denmark;Germany;Netherlands;Japan;Sweden;Korea, Republic of | ||
| 5 | EUCTR2019-004619-30-SE (EUCTR) | 22/06/2020 | 04/02/2020 | An Efficacy and Safety Study of Ravulizumab in ALS Patients | A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Ravulizumab in Patients With Amyotrophic Lateral Sclerosis (ALS) - An Efficacy and Safety Study of Ravulizumab in ALS Patients | Amyotrophic Lateral Sclerosis (ALS), motor neuron disease MedDRA version: 21.1;Level: PT;Classification code 10002026;Term: Amyotrophic lateral sclerosis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Ultomiris Product Name: ravulizumab Product Code: ALXN1210 INN or Proposed INN: RAVULIZUMAB | Alexion Pharmaceuticals | NULL | Not Recruiting | Female: yes Male: yes | 354 | Phase 3 | United States;Spain;Ireland;Israel;United Kingdom;Switzerland;Italy;France;Canada;Poland;Belgium;Australia;Denmark;Germany;Netherlands;Japan;Sweden | ||
| 6 | EUCTR2019-004619-30-GB (EUCTR) | 26/05/2020 | 07/02/2020 | An Efficacy and Safety Study of Ravulizumab in ALS Patients | A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Ravulizumab in Patients With Amyotrophic Lateral Sclerosis (ALS) - An Efficacy and Safety Study of Ravulizumab in ALS Patients | Amyotrophic Lateral Sclerosis (ALS), motor neuron disease MedDRA version: 21.1;Level: PT;Classification code 10002026;Term: Amyotrophic lateral sclerosis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Ultomiris Product Name: Ultomiris Product Code: ALXN1210 INN or Proposed INN: RAVULIZUMAB | Alexion Pharmaceuticals | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 354 | Phase 3 | United States;Spain;Ireland;Israel;Switzerland;United Kingdom;Italy;France;Canada;Poland;Belgium;Australia;Denmark;Germany;Netherlands;Japan;Sweden;Korea, Republic of | ||
| 7 | EUCTR2019-004619-30-FR (EUCTR) | 20/05/2020 | 20/08/2020 | An Efficacy and Safety Study of Ravulizumab in ALS Patients | A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Ravulizumab in Patients With Amyotrophic Lateral Sclerosis (ALS) - An Efficacy and Safety Study of Ravulizumab in ALS Patients | Amyotrophic Lateral Sclerosis (ALS), motor neuron disease MedDRA version: 21.1;Level: PT;Classification code 10002026;Term: Amyotrophic lateral sclerosis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Ultomiris Product Name: ravulizumab Product Code: ALXN1210 INN or Proposed INN: RAVULIZUMAB | Alexion Pharmaceuticals | NULL | Not Recruiting | Female: yes Male: yes | 354 | Phase 3 | United States;Spain;Ireland;Israel;United Kingdom;Switzerland;Italy;France;Canada;Belgium;Poland;Australia;Denmark;Germany;Netherlands;Japan;Sweden;Korea, Republic of | ||
| 8 | EUCTR2019-004619-30-ES (EUCTR) | 14/05/2020 | 06/03/2020 | An Efficacy and Safety Study of Ravulizumab in ALS Patients | A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Ravulizumab in Patients With Amyotrophic Lateral Sclerosis (ALS) - An Efficacy and Safety Study of Ravulizumab in ALS Patients | Amyotrophic Lateral Sclerosis (ALS), motor neuron disease MedDRA version: 21.1;Level: PT;Classification code 10002026;Term: Amyotrophic lateral sclerosis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Ultomiris Product Name: ravulizumab Product Code: ALXN1210 INN or Proposed INN: RAVULIZUMAB | Alexion Pharmaceuticals | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 354 | Phase 3 | France;Canada;Poland;Belgium;Australia;Denmark;Germany;Netherlands;United States;Spain;Ireland;Israel;United Kingdom;Switzerland;Italy;Japan;Sweden;Korea, Republic of | ||
| 9 | EUCTR2019-004619-30-NL (EUCTR) | 23/04/2020 | 09/03/2020 | An Efficacy and Safety Study of Ravulizumab in ALS Patients | A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Ravulizumab in Patients With Amyotrophic Lateral Sclerosis (ALS) - An Efficacy and Safety Study of Ravulizumab in ALS Patients | Amyotrophic Lateral Sclerosis (ALS), motor neuron disease MedDRA version: 21.1;Level: PT;Classification code 10002026;Term: Amyotrophic lateral sclerosis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Ultomiris Product Name: ravulizumab Product Code: ALXN1210 INN or Proposed INN: RAVULIZUMAB | Alexion Pharmaceuticals | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 354 | Phase 3 | United States;Spain;Ireland;Israel;United Kingdom;Switzerland;Italy;France;Canada;Poland;Belgium;Australia;Denmark;Netherlands;Germany;Japan;Sweden;Korea, Republic of | ||
| 10 | NCT04248465 (ClinicalTrials.gov) | March 30, 2020 | 27/1/2020 | An Efficacy and Safety Study of Ravulizumab in ALS Participants | A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Ravulizumab in Patients With Amyotrophic Lateral Sclerosis (ALS) | Amyotrophic Lateral Sclerosis;ALS | Drug: Placebo;Biological: Ravulizumab | Alexion Pharmaceuticals | NULL | Active, not recruiting | 18 Years | N/A | All | 382 | Phase 3 | United States;Australia;Belgium;Canada;Denmark;France;Germany;Ireland;Israel;Italy;Japan;Netherlands;Poland;Spain;Sweden;Switzerland;United Kingdom |
11. 重症筋無力症
臨床試験数 : 332 / 薬物数 : 234 - (DrugBank : 81) / 標的遺伝子数 : 45 - 標的パスウェイ数 : 127
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT05644561 (ClinicalTrials.gov) | January 2, 2023 | 1/12/2022 | Evaluation of Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants With Generalized Myasthenia Gravis (gMG) | A Phase 3, Open-label, Single-arm, Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants (6 to < 18 Years of Age) With Generalized Myasthenia Gravis (gMG) | Generalized Myasthenia Gravis;gMG | Drug: Ravulizumab | Alexion Pharmaceuticals | NULL | Not yet recruiting | 6 Years | 18 Years | All | 12 | Phase 3 | NULL |
| 2 | EUCTR2018-003243-39-PT (EUCTR) | 15/06/2020 | 06/02/2020 | Study to evaluate the safety and efficacy of ravulizumab in adult patients with generalized Myasthenia Gravis who have never been treated with a complement inhibitor. | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Ravulizumab in Complement-Inhibitor-Naïve Adult Patients With Generalized Myasthenia Gravis | Generalized Myasthenia Gravis MedDRA version: 20.0;Level: HLT;Classification code 10071942;Term: Myasthenia gravis and related conditions;System Organ Class: 100000004859;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: Ultomiris Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5 | Alexion Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 160 | Phase 3 | United States;Portugal;Spain;Austria;Russian Federation;Israel;United Kingdom;Switzerland;Italy;France;Czech Republic;Canada;Belgium;Denmark;Germany;Netherlands;Japan;Korea, Republic of | ||
| 3 | EUCTR2018-003243-39-GB (EUCTR) | 11/02/2020 | 08/10/2019 | Study to evaluate the safety and efficacy of ravulizumab in adult patients with generalized Myasthenia Gravis who have never been treated with a complement inhibitor. | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Ravulizumab in Complement-Inhibitor-Naïve Adult Patients With Generalized Myasthenia Gravis | Generalized Myasthenia Gravis MedDRA version: 20.0;Level: HLT;Classification code 10071942;Term: Myasthenia gravis and related conditions;System Organ Class: 100000004859;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: Ultomiris Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5 | Alexion Pharmaceuticals Incorporated | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 160 | Phase 3 | Portugal;United States;Spain;Turkey;Austria;Russian Federation;Israel;Italy;United Kingdom;Switzerland;France;Czech Republic;Canada;Belgium;Denmark;Netherlands;Germany;Japan;Korea, Republic of | ||
| 4 | EUCTR2018-003243-39-CZ (EUCTR) | 02/01/2020 | 13/09/2019 | Study to evaluate the safety and efficacy of ravulizumab in adult patients with generalized Myasthenia Gravis who have never been treated with a complement inhibitor | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Ravulizumab in Complement-Inhibitor-Naïve Adult Patients With Generalized Myasthenia Gravis | Generalized Myasthenia Gravis MedDRA version: 20.0;Level: HLT;Classification code 10071942;Term: Myasthenia gravis and related conditions;System Organ Class: 100000004859 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: Ultomiris Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5 | Alexion Pharmaceuticals Incorporated | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 160 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | Portugal;United States;Spain;Turkey;Austria;Russian Federation;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Belgium;Denmark;Netherlands;Germany;Japan;Korea, Republic of | ||
| 5 | EUCTR2018-003243-39-AT (EUCTR) | 09/10/2019 | 26/04/2019 | Study to evaluate the safety and efficacy of ravulizumab in adult patients with generalized Myasthenia Gravis who have never been treated with a complement inhibitor. | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Ravulizumab in Complement-Inhibitor-Naïve Adult Patients With Generalized Myasthenia Gravis | Generalized Myasthenia Gravis MedDRA version: 20.0;Level: HLT;Classification code 10071942;Term: Myasthenia gravis and related conditions;System Organ Class: 100000004859;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Ravulizumab Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5 | Alexion Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 160 | Phase 3 | United States;Portugal;Spain;Austria;Russian Federation;Israel;United Kingdom;Switzerland;Italy;France;Czech Republic;Canada;Belgium;Denmark;Germany;Netherlands;Japan;Korea, Republic of | ||
| 6 | EUCTR2018-003243-39-DK (EUCTR) | 20/09/2019 | 22/05/2019 | Study to evaluate the safety and efficacy of ravulizumab in adult patients with generalized Myasthenia Gravis who have never been treated with a complement inhibitor. | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Ravulizumab in Complement-Inhibitor-Naïve Adult Patients With Generalized Myasthenia Gravis | Generalized Myasthenia Gravis MedDRA version: 20.0;Level: HLT;Classification code 10071942;Term: Myasthenia gravis and related conditions;System Organ Class: 100000004859;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: Ultomiris Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5 | Alexion Pharmaceuticals Incorporated | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 160 | Phase 3 | Portugal;United States;Czechia;Spain;Austria;Israel;United Kingdom;Italy;Switzerland;France;Czech Republic;Canada;Denmark;Germany;Netherlands;Japan;Korea, Republic of | ||
| 7 | EUCTR2018-003243-39-DE (EUCTR) | 30/07/2019 | 25/03/2019 | Study to evaluate the safety and efficacy of ravulizumab in adult patients with generalized Myasthenia Gravis who have never been treated with a complement inhibitor. | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Ravulizumab in Complement-Inhibitor-Naïve Adult Patients With Generalized Myasthenia Gravis - Safety and Efficacy Study of Ravulizumab in Adults With Generalized Myasthenia Gravis | Generalized Myasthenia Gravis MedDRA version: 20.0;Level: HLT;Classification code 10071942;Term: Myasthenia gravis and related conditions;System Organ Class: 100000004859;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: Ultomiris Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5 Trade Name: Ultomiris 1,100 mg/11 mL Product Name: ravulizumab Product Code: ALXN1210 INN or Proposed INN: RAVULIZUMAB | Alexion Pharmaceuticals Incorporated | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 160 | Phase 3 | United States;Czechia;Spain;Austria;Israel;United Kingdom;Switzerland;Italy;France;Czech Republic;Canada;Denmark;Germany;Netherlands;Japan;Korea, Republic of;Portugal | ||
| 8 | EUCTR2018-003243-39-NL (EUCTR) | 22/07/2019 | 01/05/2019 | Study to evaluate the safety and efficacy of ravulizumab in adult patients with generalized Myasthenia Gravis who have never been treated with a complement inhibitor. | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Ravulizumab in Complement-Inhibitor-Naïve Adult Patients With Generalized Myasthenia Gravis - Safety and Efficacy Study of Ravulizumab in Adults With Generalized Myasthenia Gravis | Generalized Myasthenia Gravis MedDRA version: 20.0;Level: HLT;Classification code 10071942;Term: Myasthenia gravis and related conditions;System Organ Class: 100000004859;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: Ultomiris Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5 | Alexion Pharmaceuticals Incorporated | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 160 | Phase 3 | United States;Portugal;Czechia;Spain;Austria;Israel;United Kingdom;Switzerland;Italy;France;Czech Republic;Canada;Denmark;Netherlands;Germany;Japan;Korea, Republic of | ||
| 9 | EUCTR2018-003243-39-IT (EUCTR) | 17/06/2019 | 22/01/2021 | Study to evaluate the safety and efficacy of ravulizumab in adult patients with generalized Myasthenia Gravis who have never been treated with a complement inhibitor. | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Ravulizumab in Complement-Inhibitor-Naïve Adult Patients With Generalized Myasthenia Gravis - NA | Generalized Myasthenia Gravis MedDRA version: 20.0;Level: HLT;Classification code 10071942;Term: Myasthenia gravis and related conditions;System Organ Class: 100000004859;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: Ultomiris Product Name: Ravulizumab Product Code: [ALXN1210] | ALEXION PHARMACEUTICALS INCORPORATED | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 158 | Phase 3 | Portugal;United States;Czechia;Spain;Austria;Israel;Russian Federation;United Kingdom;Switzerland;Italy;France;Czech Republic;Canada;Belgium;Denmark;Netherlands;Germany;Japan;Korea, Republic of | ||
| 10 | EUCTR2018-003243-39-ES (EUCTR) | 21/05/2019 | 27/05/2019 | Study to evaluate the safety and efficacy of ravulizumab in adult patients with generalized Myasthenia Gravis who have never been treated with a complement inhibitor. | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Ravulizumab in Complement-Inhibitor-Naïve Adult Patients With Generalized Myasthenia Gravis | Generalized Myasthenia Gravis MedDRA version: 20.0;Level: HLT;Classification code 10071942;Term: Myasthenia gravis and related conditions;System Organ Class: 100000004859 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5 | Alexion Pharmaceuticals Incorporated | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 160 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Canada;Spain;Denmark;Austria;Israel;Netherlands;Germany;Japan;Italy;Switzerland;Korea, Republic of | ||
| 11 | NCT03920293 (ClinicalTrials.gov) | March 26, 2019 | 16/4/2019 | Safety and Efficacy Study of Ravulizumab in Adults With Generalized Myasthenia Gravis | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Ravulizumab in Complement-Inhibitor-Naïve Adult Patients With Generalized Myasthenia Gravis | Generalized Myasthenia Gravis | Biological: Ravulizumab;Drug: Placebo | Alexion Pharmaceuticals | NULL | Active, not recruiting | 18 Years | N/A | All | 175 | Phase 3 | United States;Austria;Canada;Czechia;Denmark;France;Germany;Israel;Italy;Japan;Korea, Republic of;Netherlands;Portugal;Spain;Switzerland |
13. 多発性硬化症／視神経脊髄炎
臨床試験数 : 3,340 / 薬物数 : 2,163 - (DrugBank : 383) / 標的遺伝子数 : 241 - 標的パスウェイ数 : 238
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT05346354 (ClinicalTrials.gov) | June 23, 2022 | 20/4/2022 | Efficacy and Safety Study of Ravulizumab IV in Pediatric Participants With NMOSD | A Phase 2/3, Open-label, Historical-controlled, Single-arm, Multicenter Study to Evaluate the Efficacy, Pharmacokinetics, Pharmacodynamics, and Safety of Ravulizumab in Children and Adolescents With Aquaporin-4 Antibody Positive (AQP4-Ab [+]) Neuromyelitis Optica Spectrum Disorder (NMOSD) | Neuromyelitis Optica Spectrum Disorder | Drug: Ravulizumab | Alexion Pharmaceuticals | NULL | Recruiting | 2 Years | 17 Years | All | 12 | Phase 2/Phase 3 | United States;Japan;Korea, Republic of;Spain |
| 2 | EUCTR2021-006075-42-ES (EUCTR) | 17/05/2022 | 24/02/2022 | Clinical study testing the efficacy and safety of Ravulizumab in pediatric patients with NMOSD | A Phase 2/3, Open-label, Historical-controlled, Single-arm, Multicenter Study to Evaluate the Efficacy, Pharmacokinetics, Pharmacodynamics, and Safety of Ravulizumab in Children and Adolescents With Aquaporin-4 antibody Positive (AQP4-Ab [+]) Neuromyelitis Optica Spectrum Disorder (NMOSD) - A Phase 2/3 Efficacy and Safety Study of Ravulizumab in Pediatric Patients with NMOSD | Neuromyelitis Optica Spectrum Disorder (NMOSD) MedDRA version: 21.1;Level: PT;Classification code 10077875;Term: Neuromyelitis optica spectrum disorder;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: ULTOMIRIS® Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Ravulizumab | Alexion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 12 | Phase 2;Phase 3 | United States;France;Canada;Spain;Germany;Japan;Italy;Korea, Republic of | ||
| 3 | JPRN-jRCT2031220259 | 06/07/2021 | 09/08/2022 | A Phase 2/3, Open-label, Historical-controlled, Single-arm, Multicenter Studyto Evaluate the Efficacy, Pharmacokinetics, Pharmacodynamics, and Safety ofRavulizumab in Children and Adolescents With Aquaporin-4 Antibody Positive(AQP4-Ab [+]) Neuromyelitis Optica Spectrum Disorder (NMOSD) | A Phase 2/3, Open-label, Historical-controlled, Single-arm, Multicenter Studyto Evaluate the Efficacy, Pharmacokinetics, Pharmacodynamics, and Safety ofRavulizumab in Children and Adolescents With Aquaporin-4 Antibody Positive(AQP4-Ab [+]) Neuromyelitis Optica Spectrum Disorder (NMOSD) - Phase 2/3 Efficacy and Safety Study of Ravulizumab IV in Pediatric ParticipantsWith NMOSD | Neuromyelitis Optica Spectrum Disorder NMOSD | Drug: Ravulizumab Participants will receive a weight-based loading dose of ravulizumab on Day 1, followed by weight-based maintenance treatment with ravulizumab on Day 15 and every 8 weeks (q8w) after or once every 4 weeks (q4w) depending on weight. During the Extension Period, participants will continue to receive weight- based maintenance doses of ravulizumab IV on Day 351 and q8w or q4w, depending on weight. | Wuebbenhorst Hiroaki | NULL | Recruiting | >= 2age old | < 18age old | Both | 12 | Phase 2-3 | US;Spain;Japan |
| 4 | EUCTR2019-003352-37-PL (EUCTR) | 05/10/2020 | 06/07/2020 | Clinical study testing the efficacy and safety of Ravulizumab in adults with NMOSD. | A Phase 3, External Placebo-Controlled, Open-Label, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Patients with Neuromyelitis Optica Spectrum Disorder (NMOSD) - A Phase 3 Efficacy and Safety Study of Ravulizumab in Adult Patients with NMOSD | Neuromyelitis Optica Spectrum Disorder MedDRA version: 21.1;Level: PT;Classification code 10077875;Term: Neuromyelitis optica spectrum disorder;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Ultomiris Product Name: ravulizumab Product Code: ALXN1210 INN or Proposed INN: RAVULIZUMAB Trade Name: Ultomiris Product Name: ravulizumab Product Code: ALXN1210 INN or Proposed INN: Ravulizumab | Alexion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 55 | Phase 3 | United States;Spain;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Canada;Poland;Australia;Denmark;Netherlands;Germany;Japan;Korea, Republic of | ||
| 5 | EUCTR2019-003352-37-NL (EUCTR) | 11/06/2020 | 24/02/2020 | Clinical study testing the efficacy and safety of Ravulizumab in adults with NMOSD. | A Phase 3, External Placebo-Controlled, Open-Label, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Patients with Neuromyelitis Optica Spectrum Disorder (NMOSD) - A Phase 3 Efficacy and Safety Study of Ravulizumab in Adult Patients with NMOSD | Neuromyelitis Optica Spectrum Disorder MedDRA version: 21.1;Level: PT;Classification code 10077875;Term: Neuromyelitis optica spectrum disorder;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Ultomiris Product Name: ravulizumab Product Code: ALXN1210 INN or Proposed INN: RAVULIZUMAB | Alexion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 55 | Phase 3 | United States;Spain;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Canada;Australia;Denmark;Netherlands;Germany;Japan;Korea, Republic of | ||
| 6 | EUCTR2019-003352-37-DE (EUCTR) | 11/05/2020 | 06/01/2020 | Clinical study testing the efficacy and safety of Ravulizumab in adults with NMOSD. | A Phase 3, External Placebo-Controlled, Open-Label, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Patients with Neuromyelitis Optica Spectrum Disorder (NMOSD) - A Phase 3 Efficacy and Safety Study of Ravulizumab in Adult Patients with NMOSD | Neuromyelitis Optica Spectrum Disorder MedDRA version: 21.1;Level: PT;Classification code 10077875;Term: Neuromyelitis optica spectrum disorder;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Ultomiris Product Name: ravulizumab Product Code: ALXN1210 INN or Proposed INN: RAVULIZUMAB | Alexion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 55 | Phase 3 | Germany;Netherlands;Japan;Korea, Republic of;United States;Spain;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Canada;Poland;Australia;Denmark | ||
| 7 | EUCTR2019-003352-37-AT (EUCTR) | 27/03/2020 | 13/02/2020 | Clinical study testing the efficacy and safety of Ravulizumab in adults with NMOSD. | A Phase 3, External Placebo-Controlled, Open-Label, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Patients with Neuromyelitis Optica Spectrum Disorder (NMOSD) - A Phase 3 Efficacy and Safety Study of Ravulizumab in Adult Patients with NMOSD | Neuromyelitis Optica Spectrum Disorder MedDRA version: 21.1;Level: PT;Classification code 10077875;Term: Neuromyelitis optica spectrum disorder;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Ultomiris Product Name: ravulizumab Product Code: ALXN1210 INN or Proposed INN: RAVULIZUMAB | Alexion Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 55 | Phase 3 | United States;Spain;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Canada;Poland;Australia;Denmark;Germany;Netherlands;Japan;Korea, Republic of | ||
| 8 | EUCTR2019-003352-37-IT (EUCTR) | 04/03/2020 | 17/06/2021 | A Phase 3 Efficacy and Safety Study of Ravulizumab in Adult Patients with NMOSD | A Phase 3, External Placebo-Controlled, Open-Label, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Patients with Neuromyelitis Optica Spectrum Disorder (NMOSD) - NA | Neuromyelitis Optica Spectrum Disorder MedDRA version: 21.1;Level: PT;Classification code 10077875;Term: Neuromyelitis optica spectrum disorder;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Ultromiris Product Name: Ravulizumab Product Code: [ALXN1210] | ALEXION PHARMACEUTICALS INCORPORATED | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 55 | Phase 3 | United States;Spain;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Canada;Poland;Australia;Denmark;Germany;Netherlands;Japan;Korea, Republic of | ||
| 9 | EUCTR2019-003352-37-ES (EUCTR) | 25/02/2020 | 14/01/2020 | Clinical study testing the efficacy and safety of Ravulizumab in adults with NMOSD. | A Phase 3, External Placebo-Controlled, Open-Label, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Patients with Neuromyelitis Optica Spectrum Disorder (NMOSD) - A Phase 3 Efficacy and Safety Study of Ravulizumab in Adult Patients with NMOSD | Neuromyelitis Optica Spectrum Disorder MedDRA version: 21.1;Level: PT;Classification code 10077875;Term: Neuromyelitis optica spectrum disorder;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Ultomiris Product Name: ravulizumab Product Code: ALXN1210 INN or Proposed INN: RAVULIZUMAB | Alexion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 55 | Phase 3 | Russian Federation;United States;Japan;United Kingdom;Spain;Canada;Austria;Netherlands;Turkey;Denmark;Korea, Republic of;Poland;Italy;Australia;France;Germany | ||
| 10 | EUCTR2019-003352-37-DK (EUCTR) | 15/01/2020 | 19/12/2019 | Clinical study testing the efficacy and safety of Ravulizumab in adults with NMOSD. | A Phase 3, External Placebo-Controlled, Open-Label, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Patients with Neuromyelitis Optica Spectrum Disorder (NMOSD) - A Phase 3 Efficacy and Safety Study of Ravulizumab in Adult Patients with NMOSD | Neuromyelitis Optica Spectrum Disorder MedDRA version: 21.1;Level: PT;Classification code 10077875;Term: Neuromyelitis optica spectrum disorder;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Ultomiris Product Name: ravulizumab Product Code: ALXN1210 INN or Proposed INN: RAVULIZUMAB Trade Name: Ultomiris Product Name: ravulizumab Product Code: ALXN1210 INN or Proposed INN: Ravulizumab | Alexion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 55 | Phase 3 | United States;Spain;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Canada;Poland;Denmark;Australia;Germany;Netherlands;Japan;Korea, Republic of | ||
| 11 | NCT04201262 (ClinicalTrials.gov) | December 13, 2019 | 11/12/2019 | An Efficacy and Safety Study of Ravulizumab in Adult Participants With NMOSD | A Phase 3, External Placebo-Controlled, Open-Label, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Patients With Neuromyelitis Optica Spectrum Disorder (NMOSD) | Neuromyelitis Optica;Neuromyelitis Optica Spectrum Disorder | Biological: Ravulizumab | Alexion | NULL | Active, not recruiting | 18 Years | N/A | All | 58 | Phase 3 | United States;Australia;Austria;Canada;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Poland;Spain;United Kingdom |
| 12 | EUCTR2021-006075-42-DE (EUCTR) | 17/06/2022 | Clinical study testing the efficacy and safety of Ravulizumab in pediatric patients with NMOSD | A Phase 2/3, Open-label, Historical-controlled, Single-arm, Multicenter Study to Evaluate the Efficacy, Pharmacokinetics, Pharmacodynamics, and Safety of Ravulizumab in Children and Adolescents With Aquaporin-4 antibody Positive (AQP4-Ab [+]) Neuromyelitis Optica Spectrum Disorder (NMOSD) - A Phase 2/3 Efficacy and Safety Study of Ravulizumab in Pediatric Patients with NMOSD | Neuromyelitis Optica Spectrum Disorder (NMOSD) MedDRA version: 21.1;Level: PT;Classification code 10077875;Term: Neuromyelitis optica spectrum disorder;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: ULTOMIRIS® Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Ravulizumab | Alexion Pharmaceuticals, Inc. | NULL | NA | Female: yes Male: yes | 12 | Phase 2;Phase 3 | France;United States;Canada;Spain;Germany;Italy;Japan;Korea, Republic of |
50. 皮膚筋炎／多発性筋炎
臨床試験数 : 194 / 薬物数 : 244 - (DrugBank : 89) / 標的遺伝子数 : 50 - 標的パスウェイ数 : 151
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | JPRN-jRCT2031210681 | 16/03/2022 | 18/03/2022 | Ravulizumab versus Placebo in Adult Participants with Dermatomyositis | A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Participants with Dermatomyositis | Dermatomyositis | Eligible participants will be enrolled into the study and will be randomized in a 2:1 ratio (Part A) or a 1:1 ratio (Part B) to receive intravenous (IV) ravulizumab or placebo. The study intervention dose for each participant will be based on body weight. The dosing regimen for both Parts A and B consists of a loading dose followed by maintenance doses administered every 8 weeks (q8w). The maintenance dosing will be initiated 2 weeks after the loading dose is administered. The Randomized Controlled Period for Part A will be 26 weeks in duration. The Randomized Controlled Period for Part B will be 50 weeks in duration. Participants will receive ravulizumab during the OLE Period until ravulizumab is registered or approved (in accordance with country-specific regulations) or for up to 74 weeks (approximately 1.5 years), whichever occurs first. The total study treatment duration for Part A will be approximately 2 years and for Part B approximately 2.5 years. | Ishida Hirokazu | NULL | Recruiting | >= 18age old | Not applicable | Both | 180 | Phase 2-3 | USA;Italy;France;Germany;UK;Japan |
| 2 | NCT04999020 (ClinicalTrials.gov) | December 2, 2021 | 4/8/2021 | Ravulizumab Versus Placebo in Adult Participants With Dermatomyositis | A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Participants With Dermatomyositis | Dermatomyositis | Drug: Ravulizumab;Drug: Placebo | Alexion Pharmaceuticals | NULL | Recruiting | 18 Years | N/A | All | 150 | Phase 2/Phase 3 | United States;France;Germany;Italy;Japan;Korea, Republic of;Spain;United Kingdom |
| 3 | EUCTR2021-001200-15-DE (EUCTR) | 05/10/2021 | 15/06/2021 | Ravulizumab versus Placebo in Adult Participants with Dermatomyositis. | A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Participants with Dermatomyositis. - Ravulizumab versus Placebo in Adult Participants with Dermatomyositis. | Dermatomyositis MedDRA version: 20.0;Level: PT;Classification code 10012503;Term: Dermatomyositis;System Organ Class: 10040785 - Skin and subcutaneous tissue disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: Ultomiris Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: RAVULIZUMAB Other descriptive name: ALXN1210 Trade Name: Ultomiris Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: RAVULIZUMAB Other descriptive name: ALXN1210 | Alexion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 180 | Phase 2;Phase 3 | France;United States;Spain;Germany;United Kingdom;Italy;Japan;Korea, Republic of | ||
| 4 | EUCTR2021-001200-15-ES (EUCTR) | 27/09/2021 | 16/09/2021 | Ravulizumab versus Placebo in Adult Participants with Dermatomyositis. | A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Participants with Dermatomyositis. - Ravulizumab versus Placebo in Adult Participants with Dermatomyositis. | Dermatomyositis MedDRA version: 20.0;Level: PT;Classification code 10012503;Term: Dermatomyositis;System Organ Class: 10040785 - Skin and subcutaneous tissue disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: Ultomiris Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: RAVULIZUMAB Other descriptive name: ALXN1210 Trade Name: Ultomiris Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: RAVULIZUMAB Other descriptive name: ALXN1210 | Alexion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 180 | Phase 2;Phase 3 | France;United States;Spain;Germany;United Kingdom;Italy;Japan;Korea, Republic of | ||
| 5 | EUCTR2021-001200-15-IT (EUCTR) | 16/08/2021 | 18/10/2021 | Ravulizumab versus Placebo in Adult Participants with Dermatomyositis. | A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Participants with Dermatomyositis. - Ravulizumab versus Placebo in Adult Participants with Dermatomyositis. | Dermatomyositis;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: Ultomiris Product Name: Ravulizumab Product Code: [ALXN1210] INN or Proposed INN: RAVULIZUMAB Other descriptive name: ALXN1210 Trade Name: Ultomiris Product Name: Ravulizumab Product Code: [ALXN1210] INN or Proposed INN: RAVULIZUMAB Other descriptive name: ALXN1210 Trade Name: Nimenrix Product Name: Nimenrix Product Code: [NA] INN or Proposed INN: tetanus protein Other descriptive name: tetanus protein INN or Proposed INN: NEISSERIA MENINGITIDIS GROUP A POLYSACCHARIDE CONJUGATED WITH TETANUS TOXOID CARRIER PROTEIN Other descriptive name: NEISSERIA MENINGITIDIS GROUP A POLYSACCHARIDE CONJUGATED WITH TETANUS TOXOID CARRIER PROTEIN INN or Proposed INN: NEISSERIA MENINGITIDIS GROUP W-135 POLYSACCHARIDE CONJUGATED TO TETANUS TOXOID CARRIER PROTEIN Other descriptive name: NEISSERIA MENINGITIDIS GROUP W-135 POLYSACCHARIDE CONJUGATED TO TETA | ALEXION PHARMACEUTICALS INCORPORATED | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 180 | Phase 2;Phase 3 | France;United States;Spain;Germany;United Kingdom;Japan;Italy;Korea, Republic of | ||
| 6 | EUCTR2021-001200-15-FR (EUCTR) | 10/08/2021 | 28/06/2021 | Ravulizumab versus Placebo in Adult Participants with Dermatomyositis. | A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Participants with Dermatomyositis. - Ravulizumab versus Placebo in Adult Participants with Dermatomyositis. | Dermatomyositis MedDRA version: 20.0;Level: PT;Classification code 10012503;Term: Dermatomyositis;System Organ Class: 10040785 - Skin and subcutaneous tissue disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: Ultomiris Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: RAVULIZUMAB Other descriptive name: ALXN1210 Trade Name: Ultomiris Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: RAVULIZUMAB Other descriptive name: ALXN1210 | Alexion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 180 | Phase 2;Phase 3 | United States;France;Spain;Germany;United Kingdom;Italy;Japan;Korea, Republic of |
62. 発作性夜間ヘモグロビン尿症
臨床試験数 : 292 / 薬物数 : 151 - (DrugBank : 49) / 標的遺伝子数 : 22 - 標的パスウェイ数 : 108
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2020-002761-33-FR (EUCTR) | 06/12/2022 | 21/03/2022 | Efficacy and Safety of the Combination of Pozelimab and Cemdisiran versus continued Eculizumab or Ravulizumab Treatment in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria | A RANDOMIZED, OPEN-LABEL ECULIZUMAB AND RAVULIZUMAB CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF POZELIMAB AND CEMDISIRAN COMBINATION THERAPY IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA WHO ARE CURRENTLY TREATED WITH ECULIZUMAB OR RAVULIZUMAB | Paroxysmal nocturnal hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab Product Name: Cemdisiran Product Code: ALN-CC5 INN or Proposed INN: CEMDISIRAN Other descriptive name: CEMDISIRAN Trade Name: ULTOMIRIS ™ (ravulizumab-cwvz) INN or Proposed INN: Ravulizumab Other descriptive name: Ravulizumab Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab Trade Name: Soliris ™ (eculizumab) Product Name: Eculizumab INN or Proposed INN: Eculizumab | Regeneron Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 140 | Phase 3 | Portugal;United States;Philippines;Taiwan;Greece;Spain;Turkey;Colombia;United Kingdom;Italy;France;Mexico;Poland;Brazil;Singapore;Romania;Netherlands;Germany;Japan;Korea, Republic of | ||
| 2 | NCT05131204 (ClinicalTrials.gov) | October 6, 2022 | 12/11/2021 | Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria | A Randomized, Open-Label, Eculizumab and Ravulizumab Controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Currently Treated With Eculizumab or Ravulizumab | Paroxysmal Nocturnal Hemoglobinuria | Drug: Cemdisiran;Drug: Eculizumab;Drug: Pozelimab;Drug: Ravulizumab | Regeneron Pharmaceuticals | NULL | Active, not recruiting | 18 Years | N/A | All | 2 | Phase 3 | United States |
| 3 | EUCTR2020-004438-39-NL (EUCTR) | 04/10/2022 | 05/01/2022 | BCX9930 treatment in patients with an inadequate response to C5 inhibitors | A Randomized, Open-Label, Multicenter, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria in Subjects with Inadequate Response to C5 Inhibitor Therapy - REDEEM 1 | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride Trade Name: Solaris Product Name: eculizumab Product Code: eculizumab INN or Proposed INN: Eculizumab Other descriptive name: Soliris Trade Name: Ultomiris Product Name: Ravulizumab Product Code: ravulizumab INN or Proposed INN: RAVULIZUMAB Product Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride | BioCryst Pharmaceuticals Inc | NULL | Not Recruiting | Female: yes Male: yes | 81 | Phase 2 | United States;Taiwan;Slovakia;Spain;Turkey;Colombia;Italy;United Kingdom;France;Hungary;European Union;Canada;Argentina;Brazil;Netherlands;China;Japan;Korea, Republic of | ||
| 4 | NCT05133531 (ClinicalTrials.gov) | August 1, 2022 | 12/11/2021 | Ravulizumab-Controlled Non-Inferiority Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Complement Inhibitor Treatment-Naive or Have Not Recently Received Complement Inhibitor Therapy | A Randomized, Open-Label, Ravulizumab-Controlled,Non-Inferiority Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Complement Inhibitor Treatment-Naive or Have Not Recently Received Complement Inhibitor Therapy | Paroxysmal Nocturnal Hemoglobinuria | Drug: Ravulizumab;Drug: Pozelimab;Drug: Cemdisiran | Regeneron Pharmaceuticals | NULL | Recruiting | 18 Years | N/A | All | 148 | Phase 3 | United States;Canada;Greece;Italy;Japan;Korea, Republic of;Malaysia;Mexico;Poland;Romania;Singapore;Spain;Taiwan;Turkey |
| 5 | EUCTR2020-002761-33-DE (EUCTR) | 20/06/2022 | 03/03/2022 | Efficacy and Safety of the Combination of Pozelimab and Cemdisiran versus continued Eculizumab or Ravulizumab Treatment in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria | A RANDOMIZED, OPEN-LABEL ECULIZUMAB AND RAVULIZUMAB CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF POZELIMAB AND CEMDISIRAN COMBINATION THERAPY IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA WHO ARE CURRENTLY TREATED WITH ECULIZUMAB OR RAVULIZUMAB | Paroxysmal nocturnal hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab Product Name: Cemdisiran Product Code: ALN-CC5 INN or Proposed INN: CEMDISIRAN Other descriptive name: CEMDISIRAN Trade Name: ULTOMIRIS ™ (ravulizumab-cwvz) INN or Proposed INN: Ravulizumab Other descriptive name: Ravulizumab Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab Trade Name: Soliris ™ (eculizumab) Product Name: Eculizumab INN or Proposed INN: Eculizumab | Regeneron Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 140 | Phase 3 | Portugal;United States;Philippines;Taiwan;Greece;Spain;Turkey;Colombia;United Kingdom;Italy;France;Mexico;Brazil;Poland;Singapore;Romania;Germany;Netherlands;Japan;Korea, Republic of | ||
| 6 | EUCTR2020-002761-33-GR (EUCTR) | 17/06/2022 | 12/04/2022 | Efficacy and Safety of the Combination of Pozelimab and Cemdisiran versus continued Eculizumab or Ravulizumab Treatment in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria | A RANDOMIZED, OPEN-LABEL ECULIZUMAB AND RAVULIZUMAB CONTROLLED, NON-INFERIORITY STUDY TO EVALUATE THE EFFICACY AND SAFETY OF POZELIMAB AND CEMDISIRAN COMBINATION THERAPY IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA WHO ARE CURRENTLY TREATED WITH ECULIZUMAB OR RAVULIZUMAB - ACCESS-2 | Paroxysmal nocturnal hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab Product Name: Cemdisiran Product Code: ALN-CC5 INN or Proposed INN: CEMDISIRAN Other descriptive name: CEMDISIRAN Trade Name: ULTOMIRIS ™ (ravulizumab-cwvz) INN or Proposed INN: Ravulizumab Other descriptive name: Ravulizumab Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab Trade Name: Soliris ™ (eculizumab) Product Name: Eculizumab INN or Proposed INN: Eculizumab | Regeneron Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 134 | Phase 3 | Portugal;United States;Philippines;Taiwan;Greece;Spain;Thailand;Turkey;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Brazil;Poland;Singapore;Romania;Germany;Netherlands;Japan;Korea, Republic of | ||
| 7 | EUCTR2020-004486-40-GR (EUCTR) | 17/06/2022 | 08/04/2022 | Ravulizumab-Controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria who are Complement Inhibitor Treatment-Naive or Have Not Recently Received Complement Inhibitor Therapy | A RANDOMIZED, OPEN-LABEL, RAVULIZUMAB-CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF POZELIMAB AND CEMDISIRAN COMBINATION THERAPY IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA WHO ARE COMPLEMENT INHIBITOR TREATMENT-NAIVE OR HAVE NOT RECENTLY RECEIVED COMPLEMENT INHIBITOR THERAPY | Paroxysmal nocturnal hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab Product Name: Cemdisiran Product Code: ALN-CC5 INN or Proposed INN: CEMDISIRAN Other descriptive name: CEMDISIRAN Trade Name: ULTOMIRIS ™ (ravulizumab-cwvz) INN or Proposed INN: Ravulizumab Other descriptive name: Ravulizumab Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab | Regeneron Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 124 | Phase 3 | Portugal;United States;Taiwan;Greece;Spain;Turkey;Colombia;United Kingdom;Italy;France;Mexico;Canada;Jordan;Poland;Brazil;Malaysia;Singapore;Romania;Peru;South Africa;Netherlands;China;Japan;Korea, Republic of | ||
| 8 | EUCTR2020-004486-40-ES (EUCTR) | 09/05/2022 | 11/03/2022 | Ravulizumab-Controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria who are Complement Inhibitor Treatment-Naive or Have Not Recently Received Complement Inhibitor Therapy | A RANDOMIZED, OPEN-LABEL, RAVULIZUMAB-CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF POZELIMAB AND CEMDISIRAN COMBINATION THERAPY IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA WHO ARE COMPLEMENT INHIBITOR TREATMENT-NAIVE OR HAVE NOT RECENTLY RECEIVED COMPLEMENT INHIBITOR THERAPY | Paroxysmal nocturnal hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab Product Name: Cemdisiran Product Code: ALN-CC5 INN or Proposed INN: CEMDISIRAN Other descriptive name: CEMDISIRAN Trade Name: ULTOMIRIS ™ (ravulizumab-cwvz) INN or Proposed INN: Ravulizumab Other descriptive name: Ravulizumab Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab | Regeneron Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 124 | Phase 3 | Portugal;United States;Taiwan;Greece;Spain;Turkey;Colombia;United Kingdom;Italy;France;Mexico;Canada;Jordan;Poland;Brazil;Malaysia;Singapore;Romania;Peru;South Africa;Netherlands;China;Japan;Korea, Republic of | ||
| 9 | EUCTR2020-002761-33-ES (EUCTR) | 09/05/2022 | 11/03/2022 | Efficacy and Safety of the Combination of Pozelimab and Cemdisiran versus continued Eculizumab or Ravulizumab Treatment in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria | A RANDOMIZED, OPEN-LABEL ECULIZUMAB AND RAVULIZUMAB CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF POZELIMAB AND CEMDISIRAN COMBINATION THERAPY IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA WHO ARE CURRENTLY TREATED WITH ECULIZUMAB OR RAVULIZUMAB | Paroxysmal nocturnal hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab Product Name: Cemdisiran Product Code: ALN-CC5 INN or Proposed INN: CEMDISIRAN Other descriptive name: CEMDISIRAN Trade Name: ULTOMIRIS ™ (ravulizumab-cwvz) INN or Proposed INN: Ravulizumab Other descriptive name: Ravulizumab Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab Trade Name: Soliris ™ (eculizumab) Product Name: Eculizumab INN or Proposed INN: Eculizumab | Regeneron Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 140 | Phase 3 | Portugal;United States;Philippines;Taiwan;Greece;Spain;Turkey;Colombia;United Kingdom;Italy;France;Mexico;Poland;Brazil;Singapore;Romania;Netherlands;Germany;Japan;Korea, Republic of | ||
| 10 | EUCTR2020-002761-33-PL (EUCTR) | 14/04/2022 | 22/03/2022 | Efficacy and Safety of the Combination of Pozelimab and Cemdisiran versus continued Eculizumab or Ravulizumab Treatment in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria | A RANDOMIZED, OPEN-LABEL ECULIZUMAB AND RAVULIZUMAB CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF POZELIMAB AND CEMDISIRAN COMBINATION THERAPY IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA WHO ARE CURRENTLY TREATED WITH ECULIZUMAB OR RAVULIZUMAB | Paroxysmal nocturnal hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab Product Name: Cemdisiran Product Code: ALN-CC5 INN or Proposed INN: CEMDISIRAN Other descriptive name: CEMDISIRAN Trade Name: ULTOMIRIS ™ (ravulizumab-cwvz) INN or Proposed INN: Ravulizumab Other descriptive name: Ravulizumab Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab Trade Name: Soliris ™ (eculizumab) Product Name: Eculizumab INN or Proposed INN: Eculizumab | Regeneron Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 140 | Phase 3 | Portugal;United States;Philippines;Taiwan;Greece;Spain;Turkey;Colombia;United Kingdom;Italy;France;Mexico;Poland;Brazil;Singapore;Romania;Germany;Netherlands;Japan;Korea, Republic of | ||
| 11 | EUCTR2019-003829-18-GR (EUCTR) | 08/03/2022 | 14/01/2022 | Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH) (ALPHA) | A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) | Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Danicopan Product Code: ALXN2040 INN or Proposed INN: DANICOPAN Other descriptive name: DANICOPAN Product Name: Danicopan Product Code: ALXN2040 INN or Proposed INN: DANICOPAN Other descriptive name: DANICOPAN | Alexion Pharmaceuticals Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 84 | Phase 3 | United States;Czechia;Taiwan;Greece;Thailand;Spain;Turkey;Israel;Colombia;United Kingdom;Italy;France;Canada;Argentina;Poland;Brazil;Malaysia;Netherlands;Germany;Japan;Korea, Republic of | ||
| 12 | NCT05116774 (ClinicalTrials.gov) | December 6, 2021 | 2/11/2021 | BCX9930 for Treatment of PNH in Subjects With Inadequate Response to C5 Inhibitor Therapy | A Randomized, Open-Label, Multicenter, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of PNH in Subjects With Inadequate Response to C5 Inhibitor Therapy | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: BCX9930;Drug: Eculizumab;Drug: Ravulizumab | BioCryst Pharmaceuticals | NULL | Active, not recruiting | 18 Years | N/A | All | 81 | Phase 2 | France;Hungary;Italy;Spain;United Kingdom;Taiwan;United States |
| 13 | EUCTR2020-004438-39-SK (EUCTR) | 29/11/2021 | 29/07/2021 | BCX9930 treatment in patients with an inadequate response to C5 inhibitors | A Randomized, Open-Label, Multicenter, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria in Subjects with Inadequate Response to C5 Inhibitor Therapy - REDEEM 1 | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride Trade Name: Solaris Product Name: eculizumab Product Code: eculizumab INN or Proposed INN: Eculizumab Other descriptive name: Soliris Trade Name: Ultomiris Product Name: Ravulizumab Product Code: ravulizumab INN or Proposed INN: RAVULIZUMAB Product Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride | BioCryst Pharmaceuticals Inc | NULL | Not Recruiting | Female: yes Male: yes | 81 | Phase 2 | United States;Taiwan;Slovakia;Spain;Turkey;Colombia;Italy;United Kingdom;France;Hungary;European Union;Canada;Argentina;Brazil;China;Japan;Korea, Republic of | ||
| 14 | EUCTR2020-004438-39-IT (EUCTR) | 10/11/2021 | 27/01/2022 | BCX9930 treatment in PNH patients with an inadequate response to C5 inhibitors | A Randomized, Open-Label, Multicenter, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria in Subjects with Inadequate Response to C5 Inhibitor Therapy - REDEEM 1 | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: ULTOMIRIS - 300 MG - CONCENTRATO PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO (VETRO) 3 ML (100 MG / ML) - 1 FLACONCINO Product Name: ravulizumab Product Code: [ravulizumab] INN or Proposed INN: Ravulizumab Other descriptive name: Ultomiris Trade Name: SOLIRIS - 300 MG CONCENTRATO PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO 1 FLACONCINO (VETRO) 30 ML (10 MG/ML) Product Name: Eculizumab Product Code: [Eculizumab] INN or Proposed INN: ECULIZUMAB Other descriptive name: Soliris Product Name: BCX9930 Product Code: [BCX9930] INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride | BIOCRYST PHARMACEUTICALS INC. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 81 | Phase 2 | United States;Taiwan;Spain;Turkey;Colombia;Italy;United Kingdom;France;Hungary;European Union;Canada;Argentina;Brazil;China;Japan;Korea, Republic of | ||
| 15 | EUCTR2020-004438-39-ES (EUCTR) | 02/11/2021 | 23/07/2021 | BCX9930 treatment in patients with an inadequate response to C5 inhibitors | A Randomized, Open-Label, Multicenter, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria in Subjects with Inadequate Response to C5 Inhibitor Therapy - REDEEM 1 | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride Trade Name: Solaris Product Name: eculizumab Product Code: eculizumab INN or Proposed INN: Eculizumab Other descriptive name: Soliris Trade Name: Ultomiris Product Name: Ravulizumab Product Code: ravulizumab INN or Proposed INN: RAVULIZUMAB | BioCryst Pharmaceuticals Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 81 | Phase 2 | United States;Taiwan;Spain;Turkey;Colombia;United Kingdom;France;Hungary;European Union;Canada;Argentina;Brazil;China;Japan;Korea, Republic of | ||
| 16 | EUCTR2020-004438-39-HU (EUCTR) | 01/09/2021 | 02/07/2021 | BCX9930 treatment in patients with an inadequate response to C5 inhibitors | A Randomized, Open-Label, Multicenter, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria in Subjects with Inadequate Response to C5 Inhibitor Therapy - REDEEM 1 | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride Trade Name: Solaris Product Name: eculizumab Product Code: eculizumab INN or Proposed INN: Eculizumab Other descriptive name: Soliris Trade Name: Ultomiris Product Name: Ravulizumab Product Code: ravulizumab INN or Proposed INN: RAVULIZUMAB | BioCryst Pharmaceuticals Inc | NULL | Not Recruiting | Female: yes Male: yes | 81 | Phase 2 | United States;Taiwan;Slovakia;Spain;Turkey;Colombia;Italy;United Kingdom;France;Hungary;European Union;Canada;Argentina;Brazil;Netherlands;China;Japan;Korea, Republic of | ||
| 17 | EUCTR2020-004438-39-FR (EUCTR) | 26/08/2021 | 16/06/2021 | BCX9930 treatment in patients with an inadequate response to C5 inhibitors | A Randomized, Open-Label, Multicenter, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria in Subjects with Inadequate Response to C5 Inhibitor Therapy - REDEEM 1 | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride Trade Name: Solaris Product Name: eculizumab Product Code: eculizumab INN or Proposed INN: Eculizumab Other descriptive name: Soliris Trade Name: Ultomiris Product Name: Ravulizumab Product Code: ravulizumab INN or Proposed INN: RAVULIZUMAB | BioCryst Pharmaceuticals Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 81 | Phase 2 | United States;Taiwan;Turkey;Colombia;United Kingdom;France;Hungary;European Union;Canada;Argentina;Brazil;China;Japan;Korea, Republic of | ||
| 18 | JPRN-jRCT2021210014 | 28/06/2021 | 28/06/2021 | Pivotal study of danicopan as add-on therapy to a C5 inhibitor (eculizumab or ravulizumab) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have Clinically Evident EVH | A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) | Paroxysmal Nocturnal Hemoglobinuria Paroxysmal Nocturnal Hemoglobinuria;D006457 | Additional Danicopan (ALXN2040) given to patients diagnosed with paroxysmal nocturnal hemoglobinuria and receiving complement C5 inhibitors | Kamata Takeo | NULL | Recruiting | >= 18age old | Not applicable | Both | 84 | Phase 3 | America;Japan |
| 19 | EUCTR2019-003829-18-NL (EUCTR) | 21/05/2021 | 07/10/2020 | Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH) | A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) | Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Danicopan (ALXN2040) Product Code: ACH-0144471 INN or Proposed INN: DANICOPAN Other descriptive name: DANICOPAN Product Name: Danicopan (ALXN2040) Product Code: ACH-0144471 INN or Proposed INN: DANICOPAN Other descriptive name: DANICOPAN | Alexion Pharmaceuticals Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 84 | Phase 3 | United States;Taiwan;Finland;Thailand;Spain;Austria;United Kingdom;Switzerland;Italy;France;Canada;Poland;Brazil;Malaysia;Australia;Denmark;Netherlands;Germany;Japan;Sweden;Korea, Republic of | ||
| 20 | EUCTR2019-003829-18-PL (EUCTR) | 08/04/2021 | 04/11/2020 | Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH) | A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) | Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Danicopan (ALXN2040) Product Code: ACH-0144471 INN or Proposed INN: DANICOPAN Other descriptive name: DANICOPAN Product Name: Danicopan (ALXN2040) Product Code: ACH-0144471 INN or Proposed INN: DANICOPAN Other descriptive name: DANICOPAN | Alexion Pharmaceuticals Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 84 | Phase 3 | United States;Taiwan;Spain;Thailand;United Kingdom;Italy;France;Canada;Poland;Brazil;Malaysia;Germany;Netherlands;Japan;Korea, Republic of | ||
| 21 | EUCTR2019-004665-40-CZ (EUCTR) | 31/03/2021 | 03/11/2020 | Study of efficacy and safety of twice daily oral LNP023 in adult PNH patients with residual anemia despite anti-C5 antibody treatment | A randomized, multicenter, active-comparator controlled, open-label trial to evaluate efficacy and safety of oral, twice daily LNP023 in adult patients with PNH and residual anemia, despite treatment with an intravenous anti-C5 antibody | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: LNP023 Product Code: LNP023 INN or Proposed INN: not yet established Other descriptive name: LNP023 HYDROCHLORIDE SALT Product Name: LNP023 Product Code: LNP023 INN or Proposed INN: not yet established Other descriptive name: LNP023 HYDROCHLORIDE SALT Trade Name: Soliris Product Name: Eculizumab INN or Proposed INN: ECULIZUMAB Trade Name: Ultomiris Product Name: Ravulizumab INN or Proposed INN: RAVULIZUMAB Other descriptive name: ravulizumab | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 91 | Phase 3 | United States;Czechia;Taiwan;Spain;United Kingdom;Italy;France;Czech Republic;Hungary;Canada;Brazil;Germany;Netherlands;Japan;Korea, Republic of | ||
| 22 | NCT04320602 (ClinicalTrials.gov) | March 26, 2021 | 23/3/2020 | Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab | Phase 4, Single-Arm Study of Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab | Paroxysmal Nocturnal Hemoglobinuria | Biological: Eculizumab;Biological: Ravulizumab | Alexion Pharmaceuticals | NULL | Completed | 18 Years | N/A | All | 18 | Phase 4 | United Kingdom |
| 23 | JPRN-jRCT2051200132 | 26/03/2021 | 09/02/2021 | Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment | Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment - APPLY-PNH | Paroxysmal nocturnal hemoglobinuria PNH | Arm A : LNP023 monotherapy Arm B : Anti-C5 antibody treatment(Eculizumab or Ravulizumab) | Suzuki Kazuyuki | NULL | Not Recruiting | >= 18age old | Not applicable | Both | 91 | Phase 3 | Netherlands;Japan |
| 24 | EUCTR2019-003829-18-DE (EUCTR) | 03/02/2021 | 23/01/2020 | Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH) (ALPHA) | A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) | Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Danicopan Product Code: ALXN2040 INN or Proposed INN: DANICOPAN Other descriptive name: DANICOPAN Product Name: Danicopan Product Code: ALXN2040 INN or Proposed INN: DANICOPAN Other descriptive name: DANICOPAN | Alexion Pharmaceuticals Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 84 | Phase 3 | United States;Czechia;Taiwan;Greece;Spain;Thailand;Turkey;Israel;United Kingdom;Italy;France;Canada;Argentina;Brazil;Malaysia;Poland;Netherlands;Germany;Japan;Korea, Republic of | ||
| 25 | EUCTR2019-004665-40-DE (EUCTR) | 29/01/2021 | 22/09/2020 | Study of efficacy and safety of twice daily oral LNP023 in adult PNH patients with residual anemia despite anti-C5 antibody treatment | A randomized, multicenter, active-comparator controlled, open-label trial to evaluate efficacy and safety of oral, twice daily LNP023 in adult patients with PNH and residual anemia, despite treatment with an intravenous anti-C5 antibody | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: iptacopan Product Code: LNP023 INN or Proposed INN: iptacopan Other descriptive name: LNP023 HYDROCHLORIDE SALT Product Name: iptacopan Product Code: LNP023 INN or Proposed INN: iptacopan Other descriptive name: LNP023 HYDROCHLORIDE SALT Trade Name: Soliris 300mg/30ml Product Name: Eculizumab INN or Proposed INN: ECULIZUMAB Trade Name: Ultomiris 300mg/30ml Product Name: Ravulizumab INN or Proposed INN: RAVULIZUMAB Other descriptive name: ravulizumab | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 91 | Phase 3 | United States;Czechia;Taiwan;Spain;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Brazil;Germany;Netherlands;Japan;Korea, Republic of | ||
| 26 | NCT04558918 (ClinicalTrials.gov) | January 25, 2021 | 27/8/2020 | Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment | A Randomized, Multicenter, Active-comparator Controlled, Open-label Trial to Evaluate Efficacy and Safety of Oral, Twice Daily LNP023 in Adult Patients With PNH and Residual Anemia, Despite Treatment With an Intravenous Anti-C5 Antibody. | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: LNP023;Drug: Eculizumab;Drug: Ravulizumab | Novartis Pharmaceuticals | NULL | Active, not recruiting | 18 Years | N/A | All | 97 | Phase 3 | United States;Brazil;Czechia;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Spain;Taiwan;United Kingdom;Hungary |
| 27 | NCT04469465 (ClinicalTrials.gov) | December 16, 2020 | 9/7/2020 | Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA) | A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) | Paroxysmal Nocturnal Hemoglobinuria | Drug: Danicopan;Drug: Placebo;Drug: C5 Inhibitor | Alexion Pharmaceuticals | NULL | Active, not recruiting | 18 Years | N/A | All | 86 | Phase 3 | United States;Brazil;Canada;Czechia;France;Germany;Greece;Israel;Italy;Japan;Korea, Republic of;Malaysia;Netherlands;Poland;Spain;Taiwan;Thailand;United Kingdom |
| 28 | EUCTR2019-004665-40-NL (EUCTR) | 20/11/2020 | 09/11/2020 | Study of efficacy and safety of twice daily oral LNP023 in adult PNH patients with residual anemia despite anti-C5 antibody treatment | A randomized, multicenter, active-comparator controlled, open-label trial to evaluate efficacy and safety of oral, twice daily LNP023 in adult patients with PNH and residual anemia, despite treatment with an intravenous anti-C5 antibody | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: iptacopan Product Code: LNP023 INN or Proposed INN: iptacopan Other descriptive name: LNP023 HYDROCHLORIDE SALT Product Name: iptacopan Product Code: LNP023 INN or Proposed INN: iptacopan Other descriptive name: LNP023 HYDROCHLORIDE SALT Trade Name: Soliris 300mg/30ml Product Name: Eculizumab INN or Proposed INN: ECULIZUMAB Trade Name: Ultomiris 300mg/30ml Product Name: Ravulizumab INN or Proposed INN: RAVULIZUMAB Other descriptive name: ravulizumab | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 91 | Phase 3 | United States;Czechia;Taiwan;Spain;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Brazil;Netherlands;Germany;Japan;Korea, Republic of | ||
| 29 | EUCTR2019-004665-40-HU (EUCTR) | 17/11/2020 | 23/09/2020 | Study of efficacy and safety of twice daily oral LNP023 in adult PNH patients with residual anemia despite anti-C5 antibody treatment | A randomized, multicenter, active-comparator controlled, open-label trial to evaluate efficacy and safety of oral, twice daily LNP023 in adult patients with PNH and residual anemia, despite treatment with an intravenous anti-C5 antibody | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: LNP023 Product Code: LNP023 INN or Proposed INN: not yet established Other descriptive name: LNP023 HYDROCHLORIDE SALT Product Name: LNP023 Product Code: LNP023 INN or Proposed INN: not yet established Other descriptive name: LNP023 HYDROCHLORIDE SALT Trade Name: Soliris Product Name: Eculizumab INN or Proposed INN: ECULIZUMAB Trade Name: Ultomiris Product Name: Ravulizumab INN or Proposed INN: RAVULIZUMAB Other descriptive name: ravulizumab | Novartis Pharma AG | NULL | Not Recruiting | Female: yes Male: yes | 91 | Phase 3 | United States;Taiwan;Spain;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Brazil;Germany;Netherlands;Japan;Korea, Republic of | ||
| 30 | EUCTR2019-003829-18-FR (EUCTR) | 04/11/2020 | 22/01/2020 | Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH) | A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) | Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Danicopan (ALXN2040) Product Code: ACH-0144471 INN or Proposed INN: DANICOPAN Other descriptive name: DANICOPAN Product Name: Danicopan (ALXN2040) Product Code: ACH-0144471 INN or Proposed INN: DANICOPAN Other descriptive name: DANICOPAN | Alexion Pharmaceuticals Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 84 | Phase 3 | United States;Taiwan;Finland;Spain;Thailand;Austria;United Kingdom;Switzerland;Italy;France;Canada;Poland;Brazil;Malaysia;Australia;Denmark;Netherlands;Germany;Japan;Sweden;Korea, Republic of | ||
| 31 | EUCTR2019-003829-18-IT (EUCTR) | 03/11/2020 | 24/05/2021 | Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH) | A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) - . | Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Danicopan (ALXN2040) Product Code: [ACH-0144471] Product Name: Danicopan (ALXN2040) Product Code: [ACH-0144471] | ALEXION PHARMACEUTICALS INCORPORATED | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 84 | Phase 3 | United States;Taiwan;Finland;Spain;Thailand;Austria;United Kingdom;Switzerland;Italy;France;Canada;Poland;Brazil;Malaysia;Australia;Denmark;Netherlands;Germany;Japan;Sweden;Korea, Republic of | ||
| 32 | EUCTR2019-003829-18-GB (EUCTR) | 18/09/2020 | 05/02/2020 | Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH) | A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) | Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Danicopan (ALXN2040) Product Code: ACH-0144471 INN or Proposed INN: DANICOPAN Other descriptive name: DANICOPAN Product Name: Danicopan (ALXN2040) Product Code: ACH-0144471 INN or Proposed INN: DANICOPAN Other descriptive name: DANICOPAN | Alexion Pharmaceuticals Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 84 | Phase 3 | United States;Taiwan;Finland;Thailand;Spain;Austria;Switzerland;United Kingdom;Italy;France;Canada;Poland;Brazil;Malaysia;Australia;Denmark;Netherlands;Germany;Japan;Sweden;Korea, Republic of | ||
| 33 | EUCTR2019-003440-74-GB (EUCTR) | 14/05/2020 | 17/03/2020 | Phase 4, Single-Arm Study of Ravulizumab in Adult Participants with Paroxysmal Nocturnal Hemoglobinuria Currently Treated with High-Dose Eculizumab | Phase 4, Single-Arm Study of Ravulizumab in Adult Participants with Paroxysmal Nocturnal Hemoglobinuria Currently Treated with High-Dose Eculizumab - ALXN1210-PNH-401 | Paroxysmal nocturnal hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Trade Name: ULTOMIRIS Product Name: ULTOMIRIS Product Code: ALXN1210 INN or Proposed INN: RAVULIZUMAB | Alexion Pharmaceuticals Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 4 | United Kingdom | ||
| 34 | EUCTR2019-001453-10-ES (EUCTR) | 16/09/2019 | 09/08/2019 | A Low-Interventional Study Documenting the Efficacy, Health-Related Quality of Life, and Safety of Standard-Of-Care Treatment with Eculizumab or Treatment with Ravulizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria | A MULTICENTER, LOW-INTERVENTIONAL STUDY DOCUMENTING THE EFFICACY, HEALTH-RELATED QUALITY OF LIFE, AND SAFETY OF STANDARD-OF-CARE TREATMENT WITH ECULIZUMAB OR TREATMENT WITH RAVULIZUMAB IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA | Paroxysmal nocturnal hemoglobinuria (PNH) that is treated with either eculizumab or ravulizumab as per local label MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Trade Name: Soliris 300 Product Name: ECULIZUMAB INN or Proposed INN: ECULIZUMAB Trade Name: ULTOMIRIS Product Name: Ultomiris INN or Proposed INN: RAVULIZUMAB Other descriptive name: Ultomiris | Roche Farma S. A. U. que realiza el ensayo en España y que actua como representante F.Hoffmann-La Roche Ltd | NULL | Not Recruiting | Female: yes Male: yes | 200 | Phase 4 | United States;Taiwan;Greece;Spain;Turkey;Israel;Colombia;United Kingdom;Italy;France;Czech Republic;Hungary;Canada;Argentina;Poland;Brazil;Romania;Netherlands;Germany;Japan;Sweden;Korea, Republic of | ||
| 35 | EUCTR2017-002370-39-DE (EUCTR) | 02/07/2019 | 15/11/2018 | This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With Eculizumab | A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label,Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab | Alexion Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 105 | Phase 3 | Russian Federation;United States;United Kingdom;Spain;Canada;Austria;Czech Republic;Netherlands;Sweden;Turkey;Belgium;Finland;Brazil;Korea, Republic of;Italy;Australia;France;Germany | ||
| 36 | EUCTR2017-002370-39-NL (EUCTR) | 01/07/2019 | 27/11/2018 | This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With Eculizumab | A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label,Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab | Alexion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 105 | Phase 3 | United States;Czechia;Finland;Spain;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Canada;Brazil;Belgium;Australia;Netherlands;Germany;Sweden;Korea, Republic of | ||
| 37 | EUCTR2017-002370-39-CZ (EUCTR) | 09/04/2019 | 02/01/2019 | This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With Eculizumab | A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label,Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab | Alexion Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 105 | Phase 3 | Russian Federation;United States;United Kingdom;Spain;Canada;Czech Republic;Austria;Netherlands;Sweden;Turkey;Belgium;Finland;Brazil;Korea, Republic of;Italy;Australia;France;Germany | ||
| 38 | EUCTR2017-002370-39-AT (EUCTR) | 01/04/2019 | 24/01/2019 | This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With Eculizumab | A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label,Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab | Alexion Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 105 | Phase 3 | United States;Finland;Spain;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Brazil;Australia;Germany;Netherlands;Sweden;Korea, Republic of | ||
| 39 | EUCTR2017-002370-39-SE (EUCTR) | 27/03/2019 | 30/11/2018 | This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With Eculizumab | A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label,Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab Trade Name: ULTOMIRIS Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab | Alexion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 105 | Phase 3 | United States;Czechia;Finland;Spain;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Brazil;Australia;Netherlands;Germany;Sweden;Korea, Republic of | ||
| 40 | EUCTR2017-002370-39-BE (EUCTR) | 13/03/2019 | 23/11/2018 | This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With Eculizumab | A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label,Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab | Alexion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 105 | Phase 3 | United States;Czechia;Finland;Spain;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Brazil;Australia;Germany;Netherlands;Korea, Republic of;Sweden | ||
| 41 | NCT03748823 (ClinicalTrials.gov) | March 5, 2019 | 19/11/2018 | Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab | A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label, Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab | Paroxysmal Nocturnal Hemoglobinuria | Combination Product: Ravulizumab OBDS;Biological: Ravulizumab | Alexion Pharmaceuticals | NULL | Active, not recruiting | 18 Years | N/A | All | 136 | Phase 3 | United States;Australia;Austria;Belgium;Brazil;Canada;Finland;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey |
| 42 | EUCTR2017-002370-39-IT (EUCTR) | 21/02/2019 | 18/01/2021 | This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With Eculizumab. | A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label, Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab. - NA | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Trade Name: NA Product Name: Ravulizumab Product Code: [ALXN1210] Other descriptive name: Ravulizumab Trade Name: NA Product Name: Ravulizumab Product Code: [ALXN1210] Other descriptive name: Ravulizumab | ALEXION PHARMACEUTICALS INCORPORATED | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 105 | Phase 3 | United States;Czechia;Finland;Spain;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Canada;Brazil;Belgium;Australia;Netherlands;Germany;Sweden;Korea, Republic of | ||
| 43 | EUCTR2017-002370-39-GB (EUCTR) | 19/02/2019 | 26/11/2018 | This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With Eculizumab | A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label, Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab | Alexion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 105 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Finland;Spain;Turkey;Austria;Russian Federation;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Brazil;Australia;Netherlands;Germany;Korea, Republic of;Sweden | ||
| 44 | EUCTR2017-002370-39-ES (EUCTR) | 08/02/2019 | 18/01/2019 | This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With Eculizumab | A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label, Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab | Alexion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 105 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Finland;Spain;Turkey;Austria;Russian Federation;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Brazil;Australia;Netherlands;Germany;Korea, Republic of;Sweden | ||
| 45 | EUCTR2017-002370-39-FI (EUCTR) | 29/01/2019 | 21/12/2018 | This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With Eculizumab | A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label,Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab | Alexion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 105 | Phase 3 | United States;Czechia;Finland;Spain;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Canada;Brazil;Belgium;Australia;Netherlands;Germany;Sweden;Korea, Republic of | ||
| 46 | EUCTR2017-002370-39-FR (EUCTR) | 28/12/2018 | 20/11/2018 | This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With Eculizumab | A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label, Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab | Alexion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 105 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Finland;Spain;Turkey;Austria;Russian Federation;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Brazil;Australia;Netherlands;Germany;Korea, Republic of;Sweden | ||
| 47 | EUCTR2017-002820-26-NL (EUCTR) | 21/03/2018 | 10/01/2018 | A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH) | A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Trade Name: Ultomiris Product Name: ravulizumab Product Code: ALXN1210 INN or Proposed INN: Ravulizumab Other descriptive name: ALXN1210 | Alexion Pharmaceuticals Incorporated | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 13 | Phase 3 | France;United States;Russian Federation;Norway;Netherlands;United Kingdom | ||
| 48 | NCT03406507 (ClinicalTrials.gov) | February 22, 2018 | 5/1/2018 | A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria | A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria | Biological: Ravulizumab | Alexion Pharmaceuticals | NULL | Completed | N/A | 17 Years | All | 13 | Phase 3 | United States;France;Netherlands;Norway;Russian Federation;United Kingdom |
| 49 | EUCTR2017-002820-26-GB (EUCTR) | 24/11/2017 | 26/10/2017 | A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH) | A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: PT;Classification code 10034042;Term: Paroxysmal nocturnal haemoglobinuria;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Trade Name: Ultomiris Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Ravulizumab Other descriptive name: ALXN1210 | Alexion Pharmaceuticals Incorporated | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 13 | Phase 3 | United States;France;Russian Federation;Norway;Netherlands;United Kingdom | ||
| 50 | EUCTR2016-002026-36-NL (EUCTR) | 27/07/2017 | 27/03/2017 | ALXN1210 Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) currently Treated With Eculizumab | A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Trade Name: Ultomiris Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Ravulizumab Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5 Trade Name: Soliris INN or Proposed INN: ECULIZUMAB | Alexion Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 197 | Phase 3 | United States;Spain;Germany;Netherlands;Italy;United Kingdom | ||
| 51 | NCT02946463 (ClinicalTrials.gov) | December 20, 2016 | 25/10/2016 | ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) | A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Biological: Ravulizumab;Biological: Eculizumab | Alexion Pharmaceuticals | NULL | Active, not recruiting | 18 Years | N/A | All | 246 | Phase 3 | United States;Argentina;Australia;Austria;Belgium;Brazil;Canada;Czechia;Estonia;France;Germany;Italy;Japan;Korea, Republic of;Malaysia;Mexico;Poland;Russian Federation;Singapore;Spain;Sweden;Taiwan;Thailand;Turkey;United Kingdom;Colombia |
| 52 | NCT02605993 (ClinicalTrials.gov) | January 4, 2016 | 11/11/2015 | Open-label, Multiple Ascending Dose Study of Ravulizumab (ALXN1210) in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) | A Phase 2, Open-label, Multiple Ascending Dose Study to Evaluate the Efficacy, Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients With Paroxysmal Nocturnal Hemoglobinuria | Paroxysmal Nocturnal Hemoglobinuria;PNH | Biological: Ravulizumab | Alexion Pharmaceuticals | NULL | Completed | 18 Years | N/A | All | 26 | Phase 2 | Canada;France;Germany;Korea, Republic of;Spain;Taiwan;United Kingdom;Sweden |
66. IgA腎症
臨床試験数 : 275 / 薬物数 : 258 - (DrugBank : 82) / 標的遺伝子数 : 36 - 標的パスウェイ数 : 140
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2021-005609-28-ES (EUCTR) | 18/03/2022 | 03/01/2022 | Study to assess whether the drug Ravulizumab works in patients with kidney problems due to IgA immunoglobulin and who do not respond to corticosteroids | A PILOT PROOF-OF–CONCEPT STUDY TO ASSESS THE EFFICACY AND SAFETY OF A 6 MONTHS RAVULIZUMAB TREATMENT IN PATIENTS WITH FLARES OF CORTICOSTEROID-RESISTANT IDIOPATHIC IgA NEPHROPATHY | Treatment for IgA nephropathy flares;Therapeutic area: Body processes [G] - Immune system processes [G12] | Trade Name: Ultomiris 300 mg/30 ml INN or Proposed INN: Ravulizumab Other descriptive name: ALXN1210 | Dr. Alfons Segarra Medrano | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 1 | Phase 4 | Spain | ||
| 2 | EUCTR2020-001537-13-DE (EUCTR) | 30/06/2021 | 29/01/2021 | Study of Ravulizumab in Proliferative Lupus Nephritis (LN) or Immunoglobulin A Nephropathy (IgAN) | A Phase 2, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Participants With Proliferative Lupus Nephritis (LN) or Immunoglobulin A Nephropathy (IgAN) - Ravulizumab in LN or IgAN | Lupus Nephritis (LN)Immunoglobulin A Nephropathy (IgAN) MedDRA version: 21.1;Level: PT;Classification code 10025140;Term: Lupus nephritis;System Organ Class: 10038359 - Renal and urinary disorders MedDRA version: 20.0;Classification code 10021263;Term: IgA nephropathy;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: RAVULIZUMAB Other descriptive name: BNJ441 | Alexion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 120 | Phase 2 | United States;Taiwan;Spain;United Kingdom;Italy;France;Canada;Belgium;Poland;Singapore;Australia;Germany;Netherlands;Sweden;Korea, Republic of | ||
| 3 | EUCTR2020-001537-13-IT (EUCTR) | 19/05/2021 | 08/06/2021 | Study of Ravulizumab in Proliferative Lupus Nephritis (LN) or Immunoglobulin A Nephropathy (IgAN) | A Phase 2, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Participants With Proliferative Lupus Nephritis (LN) or Immunoglobulin A Nephropathy (IgAN) - Ravulizumab in LN or IgAN | Lupus Nephritis (LN) - Immunoglobulin A Nephropathy (IgAN) MedDRA version: 21.1;Level: PT;Classification code 10025140;Term: Lupus nephritis;System Organ Class: 10038359 - Renal and urinary disorders MedDRA version: 20.0;Classification code 10021263;Term: IgA nephropathy;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Trade Name: Ultomiris Product Name: Ravulizumab Product Code: [ALXN1210] INN or Proposed INN: Ravulizumab | ALEXION PHARMACEUTICALS INCORPORATED | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 120 | Phase 2 | United States;Taiwan;Spain;Korea, Democratic People's Republic of;United Kingdom;Italy;France;Canada;Belgium;Poland;Singapore;Australia;Germany;Netherlands;Sweden | ||
| 4 | EUCTR2020-001537-13-ES (EUCTR) | 29/04/2021 | 09/02/2021 | Study of Ravulizumab in Proliferative Lupus Nephritis (LN) or Immunoglobulin A Nephropathy (IgAN) | A Phase 2, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Participants With Proliferative Lupus Nephritis (LN) or Immunoglobulin A Nephropathy (IgAN) - Ravulizumab in LN or IgAN | Lupus Nephritis (LN)Immunoglobulin A Nephropathy (IgAN) MedDRA version: 21.1;Level: PT;Classification code 10025140;Term: Lupus nephritis;System Organ Class: 10038359 - Renal and urinary disorders MedDRA version: 20.0;Classification code 10021263;Term: IgA nephropathy;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: RAVULIZUMAB Other descriptive name: BNJ441 | Alexion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 120 | Phase 2 | United States;Taiwan;Spain;United Kingdom;Italy;France;Canada;Belgium;Poland;Singapore;Australia;Germany;Netherlands;Sweden;Korea, Republic of | ||
| 5 | EUCTR2020-001537-13-SE (EUCTR) | 23/04/2021 | 29/01/2021 | Study of Ravulizumab in Proliferative Lupus Nephritis (LN) or Immunoglobulin A Nephropathy (IgAN) | A Phase 2, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Participants With Proliferative Lupus Nephritis (LN) or Immunoglobulin A Nephropathy (IgAN) - Ravulizumab in LN or IgAN | Lupus Nephritis (LN)Immunoglobulin A Nephropathy (IgAN) MedDRA version: 21.1;Level: PT;Classification code 10025140;Term: Lupus nephritis;System Organ Class: 10038359 - Renal and urinary disorders MedDRA version: 20.0;Classification code 10021263;Term: IgA nephropathy;System Organ Class: 10038359 - Renal and urinary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: RAVULIZUMAB Other descriptive name: BNJ441 | Alexion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 120 | Phase 2 | United States;Taiwan;Spain;United Kingdom;Italy;France;Canada;Belgium;Poland;Singapore;Australia;Germany;Netherlands;Sweden;Korea, Republic of |
109. 非典型溶血性尿毒症症候群
臨床試験数 : 114 / 薬物数 : 36 - (DrugBank : 7) / 標的遺伝子数 : 3 - 標的パスウェイ数 : 11
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03131219 (ClinicalTrials.gov) | August 31, 2017 | 24/4/2017 | Study of Ravulizumab in Children and Adolescents With Atypical Hemolytic Uremic Syndrome (aHUS) | A Phase 3, Open-Label, Multicenter Study of ALXN1210 in Children and Adolescents With Atypical Hemolytic Uremic Syndrome (aHUS) | Atypical Hemolytic Uremic Syndrome (aHUS) | Biological: Ravulizumab | Alexion Pharmaceuticals | NULL | Active, not recruiting | N/A | 17 Years | All | 31 | Phase 3 | United States;Belgium;Germany;Italy;Japan;Korea, Republic of;Spain;United Kingdom |
| 2 | EUCTR2016-002499-29-BE (EUCTR) | 18/08/2017 | 11/05/2017 | Study of ALXN1210 in Children and Adolescents with Atypical Hemolytic Uremic Syndrome (aHUS) | A Phase 3, Open-Label, Multicenter Study of ALXN1210 in Children and Adolescents with Atypical Hemolytic Uremic Syndrome (aHUS) | Atypical Hemolytic Uremic Syndrome (aHUS) MedDRA version: 20.0;Level: LLT;Classification code 10019515;Term: Hemolytic uremic syndrome;System Organ Class: 100000004851;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Ravulizumab Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5 | Alexion Pharmaceuticals Incorporated | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 23 | Phase 3 | United States;Taiwan;Spain;Austria;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden | ||
| 3 | EUCTR2016-002499-29-SE (EUCTR) | 21/06/2017 | 21/03/2017 | Study of ALXN1210 in Children and Adolescents with Atypical Hemolytic Uremic Syndrome (aHUS) | A Phase 3, Open-Label, Multicenter Study of ALXN1210 in Children and Adolescents with Atypical Hemolytic Uremic Syndrome (aHUS) | Atypical Hemolytic Uremic Syndrome (aHUS) MedDRA version: 20.0;Level: LLT;Classification code 10019515;Term: Hemolytic uremic syndrome;System Organ Class: 100000004851;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Ravulizumab Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5 | Alexion Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 23 | Phase 3 | United States;Taiwan;Spain;Austria;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden | ||
| 4 | EUCTR2016-002499-29-DE (EUCTR) | 24/05/2017 | 15/02/2017 | Study of ALXN1210 in Children and Adolescents with Atypical Hemolytic Uremic Syndrome (aHUS) | A Phase 3, Open-Label, Multicenter Study of ALXN1210 in Children and Adolescents with Atypical Hemolytic Uremic Syndrome (aHUS) | Atypical Hemolytic Uremic Syndrome (aHUS) MedDRA version: 20.0;Level: LLT;Classification code 10019515;Term: Hemolytic uremic syndrome;System Organ Class: 100000004851;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Ravulizumab Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5 | Alexion Pharmaceuticals Inc. | NULL | Not Recruiting | Female: yes Male: yes | 23 | Phase 3 | United States;Taiwan;Spain;Austria;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden | ||
| 5 | EUCTR2016-002499-29-GB (EUCTR) | 18/05/2017 | 09/02/2017 | Study of ALXN1210 in Children and Adolescents with Atypical Hemolytic Uremic Syndrome (aHUS) | A Phase 3, Open-Label, Multicenter Study of ALXN1210 in Children and Adolescents with Atypical Hemolytic Uremic Syndrome (aHUS) | Atypical Hemolytic Uremic Syndrome (aHUS) MedDRA version: 20.0;Level: LLT;Classification code 10019515;Term: Hemolytic uremic syndrome;System Organ Class: 100000004851;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab | Alexion Pharmaceuticals Incorporated | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 23 | Phase 3 | United States;Taiwan;Spain;Austria;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden | ||
| 6 | EUCTR2016-002499-29-AT (EUCTR) | 28/04/2017 | 08/03/2017 | Study of ALXN1210 in Children and Adolescents with Atypical Hemolytic Uremic Syndrome (aHUS) | A Phase 3, Open-Label, Multicenter Study of ALXN1210 in Children and Adolescents with Atypical Hemolytic Uremic Syndrome (aHUS) | Atypical Hemolytic Uremic Syndrome (aHUS) MedDRA version: 20.0;Level: LLT;Classification code 10019515;Term: Hemolytic uremic syndrome;System Organ Class: 100000004851;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Ravulizumab Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5 | Alexion Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 23 | Phase 3 | United States;Taiwan;Spain;Austria;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden | ||
| 7 | NCT02949128 (ClinicalTrials.gov) | March 18, 2017 | 25/10/2016 | Study of ALXN1210 in Complement Inhibitor Treatment-Naïve Adult and Adolescent Participants With Atypical Hemolytic Uremic Syndrome (aHUS) | Single Arm Study of ALXN1210 in Complement Inhibitor Treatment-naïve Adult and Adolescent Patients With Atypical Hemolytic Uremic Syndrome (aHUS) | Atypical Hemolytic Uremic Syndrome (aHUS) | Biological: Ravulizumab | Alexion Pharmaceuticals | NULL | Active, not recruiting | 12 Years | N/A | All | 58 | Phase 3 | United States;Australia;Austria;Belgium;Canada;France;Germany;Italy;Japan;Korea, Republic of;Russian Federation;Spain;Taiwan;United Kingdom |