CEMDISIRAN ( DrugBank: Cemdisiran )
4 diseases
| ID | Disease name (Link within this page) | Number of trials |
|---|---|---|
| 11 | Myasthenia gravis | 6 |
| 62 | Paroxysmal nocturnal hemoglobinuria | 14 |
| 66 | IgA nephropathy | 4 |
| 109 | Atypical hemolytic uremic syndrome | 7 |
11. Myasthenia gravis
Clinical trials : 332 / Drugs : 234 - (DrugBank : 81) / Drug target genes : 45 - Drug target pathways : 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 | EUCTR2020-003272-41-DK (EUCTR) | 15/06/2022 | 02/11/2021 | A study to examine the efficacy and safety of pozelimab and cemdisiran combination therapy in patients with symptomatic Generalized Myasthenia Gravis | Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients with Symptomatic Generalized Myasthenia Gravis - NIMBLE | Generalized Myasthenia Gravis MedDRA version: 21.1;Level: PT;Classification code 10028417;Term: Myasthenia gravis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab Product Name: Cemdisiran Product Code: ALN-CC5 INN or Proposed INN: Cemdisiran | Regeneron Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 235 | Phase 3 | Serbia;United States;Czechia;Taiwan;Spain;Turkey;United Kingdom;Italy;India;France;Canada;Belgium;Brazil;Poland;Denmark;Australia;Germany;China;Japan;Korea, Republic of | ||
| 2 | EUCTR2020-003272-41-DE (EUCTR) | 04/03/2022 | 04/01/2022 | A study to examine the efficacy and safety of pozelimab and cemdisiran combination therapy in patients with symptomatic Generalized Myasthenia Gravis | Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients with Symptomatic Generalized Myasthenia Gravis - NIMBLE | Generalized Myasthenia Gravis MedDRA version: 21.1;Level: PT;Classification code 10028417;Term: Myasthenia gravis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab Product Name: Cemdisiran Product Code: ALN-CC5 INN or Proposed INN: Cemdisiran | Regeneron Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 235 | Phase 3 | United States;Czechia;Taiwan;Spain;Ukraine;Russian Federation;United Kingdom;Italy;France;Canada;Poland;Belgium;Australia;Denmark;Germany;Korea, Republic of | ||
| 3 | EUCTR2020-003272-41-ES (EUCTR) | 03/02/2022 | 28/09/2021 | A study to examine the efficacy and safety of pozelimab and cemdisiran combination therapy in patients with symptomatic Generalized Myasthenia Gravis | Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients with Symptomatic Generalized Myasthenia Gravis | Generalized Myasthenia Gravis MedDRA version: 21.1;Level: PT;Classification code 10028417;Term: Myasthenia gravis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab Product Name: Cemdisiran Product Code: ALN-CC5 INN or Proposed INN: Cemdisiran | Regeneron Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 210 | Phase 3 | United States;Czechia;Taiwan;Spain;Ukraine;Russian Federation;United Kingdom;Italy;France;Canada;Poland;Belgium;Australia;Denmark;Germany;Korea, Republic of | ||
| 4 | NCT05070858 (ClinicalTrials.gov) | December 14, 2021 | 27/9/2021 | A Study to Examine the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Symptomatic Generalized Myasthenia Gravis | Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Symptomatic Generalized Myasthenia Gravis | Generalized Myasthenia Gravis | Drug: Pozelimab + Cemdisiran;Drug: Cemdisiran;Other: Placebo;Drug: Pozelimab | Regeneron Pharmaceuticals | NULL | Recruiting | 18 Years | N/A | All | 235 | Phase 3 | United States;Australia;Belgium;Canada;Czechia;Denmark;France;Italy;Japan;Korea, Republic of;Poland;Spain;Taiwan;Turkey;United Kingdom |
| 5 | EUCTR2020-003272-41-IT (EUCTR) | 13/12/2021 | 19/10/2021 | A study to examine the efficacy and safety of pozelimab and cemdisiran combination therapy in patients with symptomatic Generalized Myasthenia Gravis | Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients with Symptomatic Generalized Myasthenia Gravis - Study of Pozelimab and Cemdisiran combination therapy in patients with Myasthenia Gravis | Generalized Myasthenia Gravis MedDRA version: 21.1;Level: PT;Classification code 10028417;Term: Myasthenia gravis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Cemdisiran Product Code: [ALN-CC5] INN or Proposed INN: Cemdisiran Product Name: Pozelimab Product Code: [REGN3918] INN or Proposed INN: Pozelimab Trade Name: Bexsero Product Name: Bexsero Product Code: [NA] INN or Proposed INN: Outer membrane vesicles (OMV) from Neisseria meningitidis group B strain NZ98/254measured as amount of total protein containing the PorA P1.4 Other descriptive name: RECOMBINANT NEISSERIA MENINGITIDIS GROUP B FHBP FUSION PROTEIN PRODUCED IN E.COLI CELLS BY RECOMBINANT DNA TECHNOLOGY ADSORBED ON ALUMINIUM HYDROXIDE INN or Proposed INN: RECOMBINANT NEISSERIA MENINGITIDIS GROUP B NHBA FUSION PROTEIN Other descriptive name: RECOMBINANT NEISSERIA MENINGITIDIS GROUP B NHBA FUSION PROTEIN INN or Proposed INN: RECOMBINANT NEISSERIA MENINGITIDIS GROUP B NADA PROTEIN Other descriptive name: RECOMBINANT NEISSERIA | REGENERON PHARMACEUTICALS, INC. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 210 | Phase 3 | United States;Czechia;Taiwan;Spain;Ukraine;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Poland;Australia;Denmark;Germany;Korea, Republic of | ||
| 6 | EUCTR2020-003272-41-PL (EUCTR) | 01/12/2021 | 21/10/2021 | A study to examine the efficacy and safety of pozelimab and cemdisiran combination therapy in patients with symptomatic Generalized Myasthenia Gravis | Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients with Symptomatic Generalized Myasthenia Gravis | Generalized Myasthenia Gravis MedDRA version: 21.1;Level: PT;Classification code 10028417;Term: Myasthenia gravis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Pozelimab Product Code: REGN3918 INN or Proposed INN: Pozelimab Product Name: Cemdisiran Product Code: ALN-CC5 INN or Proposed INN: Cemdisiran | Regeneron Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 210 | Phase 3 | United States;Czechia;Taiwan;Spain;Ukraine;Russian Federation;United Kingdom;Italy;France;Canada;Poland;Belgium;Australia;Denmark;Germany;Korea, Republic of |
62. Paroxysmal nocturnal hemoglobinuria
Clinical trials : 292 / Drugs : 151 - (DrugBank : 49) / Drug target genes : 22 - Drug target pathways : 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 | NCT05744921 (ClinicalTrials.gov) | March 9, 2023 | 6/2/2023 | A Study to Examine the Long-term Safety, Tolerability, and Effectiveness of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria | An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria | Paroxysmal Nocturnal Hemoglobinuria | Drug: Pozelimab;Drug: Cemdisiran | Regeneron Pharmaceuticals | NULL | Not yet recruiting | 18 Years | N/A | All | 300 | Phase 3 | NULL |
| 2 | EUCTR2021-004931-10-GR (EUCTR) | 04/01/2023 | 22/11/2022 | A study to examine the long-term safety, tolerability, and effectiveness of Pozelimab and Cemdisiran combination therapy in adult patients with Paroxysmal Nocturnal Hemoglobinuria | An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of Pozelimab and Cemdisiran Combination Therapy in Patients with Paroxysmal Nocturnal Hemoglobinuria - ACCESS-EXTENSION | 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 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 | 288 | Phase 3 | United States;Philippines;Taiwan;Greece;Thailand;Spain;Colombia;Italy;India;France;Jordan;Malaysia;Peru;South Africa;China;Korea, Democratic People's Republic of;Turkey;United Kingdom;Hungary;Mexico;Canada;Brazil;Poland;Singapore;Romania;Germany;Japan | ||
| 3 | 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 | ||
| 4 | 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 |
| 5 | 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 |
| 6 | 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 | ||
| 7 | 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 | ||
| 8 | 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 | ||
| 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 | EUCTR2020-004486-40-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 | ||
| 11 | 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 | ||
| 12 | NCT04811716 (ClinicalTrials.gov) | July 28, 2021 | 19/3/2021 | Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy | A Randomized, Open-label, Two-arm Study to Evaluate the Safety, Efficacy, and Pharmacodynamic Effects of Pozelimab and Cemdisiran Combination Treatment in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy | Paroxysmal Nocturnal Hemoglobinuria | Drug: Pozelimab;Drug: Cemdisiran | Regeneron Pharmaceuticals | NULL | Active, not recruiting | 18 Years | N/A | All | 24 | Phase 2 | Hong Kong;Hungary;Korea, Republic of;Malaysia;Taiwan;United Kingdom |
| 13 | NCT04888507 (ClinicalTrials.gov) | July 8, 2021 | 23/4/2021 | Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy | A Single Arm, Open-Label Study to Assess the Safety, Efficacy, and Pharmacodynamic Effects of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy | Paroxysmal Nocturnal Hemoglobinuria | Drug: Pozelimab;Drug: Cemdisiran | Regeneron Pharmaceuticals | NULL | Active, not recruiting | 18 Years | N/A | All | 6 | Phase 2 | United Kingdom |
| 14 | EUCTR2020-005005-17-HU (EUCTR) | 07/05/2021 | 05/03/2021 | Pozelimab and Cemdisiran Combination Treatment in Adult Participants with Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy | A Randomized, Open-label, Two-arm Study to Evaluate the Safety, Efficacy, and Pharmacodynamic Effects of Pozelimab and Cemdisiran Combination Treatment in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy | 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: 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 | Regeneron Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 24 | Phase 2 | Taiwan;Hong Kong;Hungary;Malaysia;United Kingdom;Korea, Republic of |
66. IgA nephropathy
Clinical trials : 275 / Drugs : 258 - (DrugBank : 82) / Drug target genes : 36 - Drug target pathways : 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 | EUCTR2018-002716-27-GB (EUCTR) | 11/06/2019 | 07/01/2019 | A Phase 2 Study of Cemdisiran in Adult Patients with IgA Nephropathy | A Phase 2, Randomized, Double-blind, Placebo-controlled Study of Cemdisiran in Adult Patients with IgA Nephropathy - A Phase 2 Study of Cemdisiran in Adult Patients with IgA Nephropathy | Immunoglobulin A nephropathy (IgAN);Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: cemdisiran Product Code: ALN-CC5 INN or Proposed INN: cemdisiran Other descriptive name: CEMDISIRAN | Alnylam Pharmaceuticals Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Phase 2 | United States;France;Philippines;Taiwan;Canada;Spain;Malaysia;Singapore;United Kingdom;Sweden | ||
| 2 | EUCTR2018-002716-27-SE (EUCTR) | 28/05/2019 | 06/02/2019 | A Phase 2 Study of Cemdisiran in Adult Patients with IgA Nephropathy | A Phase 2, Randomized, Double-blind, Placebo-controlled Study of Cemdisiran in Adult Patients with IgA Nephropathy - A Phase 2 Study of Cemdisiran in Adult Patients with IgA Nephropathy | Immunoglobulin A nephropathy (IgAN);Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: cemdisiran Product Code: ALN-CC5 INN or Proposed INN: cemdisiran Other descriptive name: CEMDISIRAN | Alnylam Pharmaceuticals Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Phase 2 | France;United States;Philippines;Taiwan;Canada;Spain;Malaysia;Singapore;United Kingdom;Sweden | ||
| 3 | NCT03841448 (ClinicalTrials.gov) | April 24, 2019 | 13/2/2019 | A Study of Cemdisiran in Adults With Immunoglobulin A Nephropathy (IgAN) | A Phase 2, Randomized, Double-blind, Placebo-controlled Study of Cemdisiran in Adult Patients With IgA Nephropathy | IgA Nephropathy (IgAN);Berger Disease;Glomerulonephritis, IgA | Drug: Placebo;Drug: Cemdisiran | Alnylam Pharmaceuticals | NULL | Active, not recruiting | 18 Years | 65 Years | All | 31 | Phase 2 | Canada;France;Malaysia;Philippines;Singapore;Spain;Sweden;Taiwan;United Kingdom;United States |
| 4 | EUCTR2018-002716-27-ES (EUCTR) | 08/04/2019 | 12/04/2019 | A Phase 2 Study of Cemdisiran in Adult Patients with IgA Nephropathy | A Phase 2, Randomized, Double-blind, Placebo-controlled Study of Cemdisiran in Adult Patients with IgA Nephropathy - A Phase 2 Study of Cemdisiran in Adult Patients with IgA Nephropathy | Immunoglobulin A nephropathy (IgAN);Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: cemdisiran Product Code: ALN-CC5 INN or Proposed INN: cemdisiran Other descriptive name: CEMDISIRAN | Alnylam Pharmaceuticals Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Philippines;Taiwan;Spain;United Kingdom;France;Canada;Malaysia;Singapore;Australia;Germany;Sweden;Korea, Republic of |
109. Atypical hemolytic uremic syndrome
Clinical trials : 114 / Drugs : 36 - (DrugBank : 7) / Drug target genes : 3 - Drug target pathways : 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 | NCT03999840 (ClinicalTrials.gov) | January 2021 | 25/6/2019 | Eculizumab to Cemdisiran Switch in aHUS | A Phase II, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Safety and Efficacy of Cemdisiran (ALN-CC5) Following Withdrawal of Chronic Eculizumab Therapy in Patients With Atypical HUS at High Risk of Recurrence | Atypical Hemolytic Uremic Syndrome | Drug: cemdisiran;Drug: Placebos | Mario Negri Institute for Pharmacological Research | Alnylam Pharmaceuticals | Withdrawn | 12 Years | N/A | All | 0 | Phase 2 | Italy |
| 2 | EUCTR2018-004382-13-IT (EUCTR) | 28/11/2019 | 25/05/2021 | Safety and efficacy of Cemdisiran in athypical hemolytic uremic syndrome | A PHASE II, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED TRIAL TO EVALUATE THE SAFETY AND EFFICACY OF CEMDISIRAN (ALN-CC5) FOLLOWING WITHDRAWAL OF CHRONIC ECULIZUMAB THERAPY IN PATIENTS WITH ATYPICAL HUS AT HIGH RISK OF RECURRENCE - Eculizumab to Cemdisiran switch in aHUS | Atypical Hemolytic Uremic Syndrome 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: ALN-CC5 Product Code: [ALN-62643] INN or Proposed INN: Cemdisiran | IRCCS- ISTITUTO DI RICERCHE FARMACOLOGICHE MARIO NEGRI | NULL | Not Recruiting | Female: yes Male: yes | 12 | Phase 2 | Italy | ||
| 3 | EUCTR2017-001082-24-SE (EUCTR) | 20/11/2017 | 27/09/2017 | An international study at different study sites providing a drug called cemdisiran (ALN-CC5) to patients with Atypical Hemolytic Uremic Syndrome by a subcutaneous injection | A Phase 2, Open Label, Multicenter Study of ALN-CC5 Administered Subcutaneously in Adult Patients with Atypical Hemolytic Uremic Syndrome | Atypical Hemolytic Uremic Syndrome 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: ALN-CC5 Product Code: ALN-CC5 INN or Proposed INN: cemdisiran | Alnylam Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 24 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | Serbia;Estonia;Lithuania;India;Canada;Macedonia, the former Yugoslav Republic of;Romania;Albania;Georgia;Latvia;Moldova, Republic of;Sweden;Bosnia and Herzegovina | ||
| 4 | NCT03303313 (ClinicalTrials.gov) | September 19, 2017 | 25/9/2017 | A Study of an Investigational Drug, Cemdisiran (ALN-CC5), in Patients With Atypical Hemolytic Uremic Syndrome | A Phase 2, Open Label, Multicenter Study of ALN-CC5 Administered Subcutaneously in Adult Patients With Atypical Hemolytic Uremic Syndrome | Atypical Hemolytic Uremic Syndrome | Drug: Cemdisiran | Alnylam Pharmaceuticals | NULL | Withdrawn | 18 Years | N/A | All | 0 | Phase 2 | Bosnia and Herzegovina;Canada;Estonia;Georgia;Latvia;Lithuania;Macedonia, The Former Yugoslav Republic of;Moldova, Republic of;Serbia;Sweden |
| 5 | EUCTR2017-001082-24-EE (EUCTR) | 15/09/2017 | 16/08/2017 | An international study at different study sites providing a drug called cemdisiran (ALN-CC5) to patients with Atypical Hemolytic Uremic Syndrome by a subcutaneous injection | A Phase 2, Open Label, Multicenter Study of ALN-CC5 Administered Subcutaneously in Adult Patients with Atypical Hemolytic Uremic Syndrome | Atypical Hemolytic Uremic Syndrome 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: ALN-CC5 Product Code: ALN-CC5 INN or Proposed INN: cemdisiran | Alnylam Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 24 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | Serbia;Estonia;Lithuania;India;Canada;Macedonia, the former Yugoslav Republic of;Romania;Albania;Georgia;Latvia;Moldova, Republic of;Sweden;Bosnia and Herzegovina | ||
| 6 | EUCTR2017-001082-24-LT (EUCTR) | 23/08/2017 | 27/06/2017 | An international study at different study sites providing a drug called cemdisiran (ALN-CC5) to patients with Atypical Hemolytic Uremic Syndrome by a subcutaneous injection | A Phase 2, Open Label, Multicenter Study of ALN-CC5 Administered Subcutaneously in Adult Patients with Atypical Hemolytic Uremic Syndrome | Atypical Hemolytic Uremic Syndrome 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: ALN-CC5 Product Code: ALN-CC5 INN or Proposed INN: cemdisiran | Alnylam Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 24 | Phase 2 | Serbia;Estonia;Lithuania;India;Canada;Macedonia, the former Yugoslav Republic of;Romania;Albania;Georgia;Latvia;Moldova, Republic of;Sweden;Bosnia and Herzegovina | ||
| 7 | EUCTR2017-001082-24-LV (EUCTR) | 18/08/2017 | 20/06/2017 | An international study at different study sites providing a drug called cemdisiran (ALN-CC5) to patients with Atypical Hemolytic Uremic Syndrome by a subcutaneous injection | A Phase 2, Open Label, Multicenter Study of ALN-CC5 Administered Subcutaneously in Adult Patients with Atypical Hemolytic Uremic Syndrome | Atypical Hemolytic Uremic Syndrome 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: ALN-CC5 Product Code: ALN-CC5 INN or Proposed INN: cemdisiran | Alnylam Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 24 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | Serbia;Estonia;Macedonia, the former Yugoslav Republic of;Canada;Romania;Lithuania;Georgia;Albania;Latvia;Moldova, Republic of;Sweden;Bosnia and Herzegovina |