62. 発作性夜間ヘモグロビン尿症 Paroxysmal nocturnal hemoglobinuria Clinical trials / Disease details
臨床試験数 : 271 / 薬物数 : 163 - (DrugBank : 49) / 標的遺伝子数 : 22 - 標的パスウェイ数 : 106
Showing 1 to 10 of 12 diseases
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 |
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1 | NCT04729062 (ClinicalTrials.gov) | April 5, 2021 | 25/1/2021 | Pegcetacoplan Early Access Program for PNH | Early Access Program (EAP) For The Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) With Pegcetacoplan Early Access Program (EAP) For The Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) With Pegce ... | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: Pegcetacoplan | Apellis Pharmaceuticals, Inc. | NULL | No longer available | 18 Years | N/A | All | United States | ||
2 | NCT04901936 (ClinicalTrials.gov) | February 4, 2021 | 8/3/2021 | A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) | An Open Label, Single-Arm, Phase 2 Study to Evaluate the Safety, Pharmacokinestics, and Biologic Activity of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria An Open Label, Single-Arm, Phase 2 Study to Evaluate the Safety, Pharmacokinestics, and Biologic Act ... | Paroxysmal Nocturnal Hemoglobinuria (PNH);Paroxysmal Hemoglobinuria | Drug: Pegcetacoplan | Apellis Pharmaceuticals, Inc. | NULL | Recruiting | 12 Years | 17 Years | All | 12 | Phase 2 | United States |
3 | EUCTR2019-001106-23-FR (EUCTR) | 04/02/2020 | 15/10/2019 | An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long Term Safety and Efficacy of APL-2 in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH). An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long Term Safety and Eff ... | An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long Term Safety and Efficacy of APL-2 in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH). An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long Term Safety and Eff ... | 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] Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Ter ... | Product Name: APL-2 Product Code: APL-2 INN or Proposed INN: pegcetacoplan Other descriptive name: APL-2 Product Name: APL-2 Product Code: APL-2 INN or Proposed INN: pegcetacoplan Other descriptive name: APL- ... | Apellis Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 119 | Phase 3 | France;Canada;Belgium;Malaysia;Poland;Australia;Bulgaria;Germany;Japan;Korea, Republic of;United Kingdom;Russian Federation;Spain;Thailand;Hong Kong;Serbia;United States France;Canada;Belgium;Malaysia;Poland;Australia;Bulgaria;Germany;Japan;Korea, Republic of;United Kin ... | ||
4 | EUCTR2019-001106-23-ES (EUCTR) | 22/01/2020 | 14/01/2020 | An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long Term Safety and Efficacy of APL-2 in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH). An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long Term Safety and Eff ... | An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long Term Safety and Efficacy of APL-2 in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH). An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long Term Safety and Eff ... | 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] Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Ter ... | Product Name: APL-2 Product Code: APL-2 INN or Proposed INN: pegcetacoplan Other descriptive name: APL-2 Product Name: APL-2 Product Code: APL-2 INN or Proposed INN: pegcetacoplan Other descriptive name: APL- ... | Apellis Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 119 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (P ... | Serbia;United States;Hong Kong;Spain;Thailand;Russian Federation;United Kingdom;France;Canada;Poland;Malaysia;Belgium;Australia;Bulgaria;Germany;Japan;Korea, Republic of Serbia;United States;Hong Kong;Spain;Thailand;Russian Federation;United Kingdom;France;Canada;Poland ... | ||
5 | EUCTR2019-001106-23-GB (EUCTR) | 19/12/2019 | 30/09/2019 | An Open Label, Nonrandomized, Multicenter Extension Study to Evaluate the Long Term Safety and Efficacy of Pegcetacoplan in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH). An Open Label, Nonrandomized, Multicenter Extension Study to Evaluate the Long Term Safety and Effic ... | An Open Label, Nonrandomized, Multicenter Extension Study to Evaluate the Long Term Safety and Efficacy of Pegcetacoplan in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH). An Open Label, Nonrandomized, Multicenter Extension Study to Evaluate the Long Term Safety and Effic ... | 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] Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Ter ... | Product Name: Pegcetacoplan (APL-2) Product Code: Pegcetacoplan (APL-2) INN or Proposed INN: pegcetacoplan Other descriptive name: Pegcetacoplan (APL-2) Product Name: Pegcetacoplan(APL-2) Product Code: Pegcetacoplan(APL-2) INN or Proposed INN: pegcetacopl ... | Apellis Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 160 | Phase 3 | Serbia;United States;Hong Kong;Spain;Thailand;Russian Federation;United Kingdom;France;Canada;Malaysia;Australia;Bulgaria;Japan;Korea, Republic of Serbia;United States;Hong Kong;Spain;Thailand;Russian Federation;United Kingdom;France;Canada;Malays ... | ||
6 | NCT04085601 (ClinicalTrials.gov) | August 15, 2019 | 9/9/2019 | A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH | A Phase 3, Randomized, Multicenter, Open-Label, Controlled Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) A Phase 3, Randomized, Multicenter, Open-Label, Controlled Study to Evaluate the Efficacy and Safety ... | Paroxysmal Nocturnal Hemoglobinuria | Drug: APL-2 | Apellis Pharmaceuticals, Inc. | NULL | Completed | 18 Years | N/A | All | 53 | Phase 3 | Colombia;Hong Kong;Malaysia;Mexico;Peru;Philippines;Poland;Serbia;Singapore;Thailand |
7 | EUCTR2019-001106-23-BG (EUCTR) | 09/08/2019 | 09/05/2019 | An Open-label, Nonrandomized, Multicenter Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan in the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) An Open-label, Nonrandomized, Multicenter Extension Study to Evaluate the Long-term Safety and Effic ... | An Open-label, Nonrandomized, Multicenter Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan in the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) An Open-label, Nonrandomized, Multicenter Extension Study to Evaluate the Long-term Safety and Effic ... | 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] Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1;Level: LLT;Classification code 10055629;Ter ... | Product Name: pegcetacoplan Product Code: pegcetacoplan INN or Proposed INN: pegcetacoplan Other descriptive name: pegcetacoplan Product Name: pegcetacoplan Product Code: pegcetacoplan INN or Proposed INN: pegcetacoplan Other descri ... | Apellis Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 160 | Phase 3 | Serbia;United States;Hong Kong;Spain;Thailand;Ireland;Russian Federation;United Kingdom;Italy;France;Canada;Poland;Malaysia;Australia;Bulgaria;Netherlands;Germany;Japan;Korea, Republic of Serbia;United States;Hong Kong;Spain;Thailand;Ireland;Russian Federation;United Kingdom;Italy;France ... | ||
8 | EUCTR2018-004220-11-PL (EUCTR) | 23/07/2019 | 13/05/2019 | A Research Study to Gather Scientific Information About the Efficacy and Safety of the Investigational Drug APL-2 In Treating Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH), a Disease Associated with Anemia, In a Randomly Assigned Comparison with the Current Standard of Care Treatment Approved for PNH A Research Study to Gather Scientific Information About the Efficacy and Safety of the Investigation ... | A Phase 3, Randomized, Multicenter, Open-Label, Controlled Study to Evaluate the Efficacy and Safety of APL-2 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) - PRINCE A Phase 3, Randomized, Multicenter, Open-Label, Controlled Study to Evaluate the Efficacy and Safety ... | Paroxysmal Nocturnal Hemoglobinuria (PNH) 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] Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 20.1;Level: LLT;Classification code 100556 ... | Product Name: APL-2 Product Code: APL-2 INN or Proposed INN: pegcetacoplan Other descriptive name: APL-2 Product Name: APL-2 Product Code: APL-2 INN or Proposed INN: pegcetacoplan Other descriptive name: APL- ... | Apellis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 54 | Phase 3 | Serbia;Philippines;Hungary;Hong Kong;Mexico;Argentina;Thailand;Malaysia;Brazil;Poland;Peru;Colombia | ||
9 | EUCTR2017-004268-36-GB (EUCTR) | 08/01/2019 | 21/08/2018 | A Research Study to Gather Scientific Information About the Efficacy, Safety, and Tolerability of the Investigational Drug Pegcetacoplan In Treating Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH), a Disease Associated With Anemia, In A Randomly Assigned Comparison With the Current Standard of Care Treatment Approved for PNH At Multiple Research Centres. A Research Study to Gather Scientific Information About the Efficacy, Safety, and Tolerability of th ... | A Phase III, Randomized, Multi-Center, Open-Label, Active-Comparator Controlled Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) - PEGASUS A Phase III, Randomized, Multi-Center, Open-Label, Active-Comparator Controlled Study to Evaluate th ... | PNH is an acquired, rare, clonal, non-malignant hematologic disease characterized by complement-mediated RBC hemolysis with or without hemoglobinuria, an increased susceptibility to thrombotic episodes, and/or some degree of bone marrow dysfunction.PNH is caused by complement-mediated lysis of erythrocyte clones lacking functional CD55 and CD59 on their surface. These RBCs are particularly susceptible to the MAC and have been shown to lyse readily in the presence of complement activation. 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] PNH is an acquired, rare, clonal, non-malignant hematologic disease characterized by complement-medi ... | Product Name: Pegcetacoplan (APL-2) Product Code: Pegcetacoplan (APL-2) INN or Proposed INN: n/a Other descriptive name: n/a Trade Name: Soliris Product Name: Soliris INN or Proposed INN: ECULIZUMAB Trade Name: Soliris Product Name: Soliris INN or Proposed INN: ECULIZUMAB Product Name: Pegcetacoplan(APL-2) Product Code: Pegcetacoplan(APL-2) INN or Proposed INN: n/a Other de ... | Apellis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 70 | Phase 3 | United States;Spain;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Netherlands;Japan;Korea, Republic of United States;Spain;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany; ... | ||
10 | NCT03531255 (ClinicalTrials.gov) | August 27, 2018 | 9/5/2018 | Pegcetacoplan Long Term Safety and Efficacy Extension Study | An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegcetacoplan in the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long-Term Safety and Eff ... | PNH | Drug: Pegcetacoplan | Apellis Pharmaceuticals, Inc. | NULL | Active, not recruiting | 18 Years | N/A | All | 160 | Phase 3 | United States;Australia;Belgium;Bulgaria;Canada;France;Germany;Hong Kong;Japan;Korea, Republic of;Malaysia;Russian Federation;Serbia;Singapore;Spain;Thailand;United Kingdom United States;Australia;Belgium;Bulgaria;Canada;France;Germany;Hong Kong;Japan;Korea, Republic of;Ma ... |