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
| 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 | EUCTR2019-004931-21-PT (EUCTR) | 19/05/2021 | 28/10/2020 | A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING THE EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN ADULT AND ADOLESCENT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) NOT PREVIOUSLY TREATED WITHCOMPLEMENT INHIBITORS | A PHASE III, RANDOMIZED, OPEN-LABEL, ACTIVE-CONTROLLED, MULTICENTER STUDY EVALUATING THE EFFICACY AND SAFETY OF CROVALIMAB VERSUS ECULIZUMAB IN ADULT AND ADOLESCENT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) NOT PREVIOUSLY TREATED WITHCOMPLEMENT INHIBITORS | 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] - Immune System Diseases [C20] | Trade Name: Soliris 300 mg concentrate for solution for infusion INN or Proposed INN: ECULIZUMAB Product Name: Crovalimab Product Code: RO7112689/F03-10 INN or Proposed INN: CROVALIMAB | F. Hoffman-La Roche Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 200 | Phase 3 | United States;Portugal;Philippines;Estonia;Taiwan;Hong Kong;Greece;Spain;Thailand;Ireland;Israel;Colombia;Italy;France;Malaysia;Peru;Australia;South Africa;Netherlands;China;Korea, Republic of;Czechia;Lithuania;Turkey;Austria;United Kingdom;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Brazil;Romania;Germany;Japan;Sweden | ||
| 2 | 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 | ||
| 3 | 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;Hungary;Canada;Brazil;Netherlands;Germany;Japan;Korea, Republic of | ||
| 4 | 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 |