4 completed, 3 recruiting, 2 active not recruiting, 2 terminated. 6 Phase 3, 5 Phase 2, 2 Phase 1.
By phase and status
Follow Cemdisiran: new trials by email Literature Review of Cemdisiran trials Research Report on Cemdisiran
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix .
Trial Phase Status Sponsor Enrollment Start Updated
A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently Pozelimab, Cemdisiran - Paroxysmal Nocturnal Hemoglobinuria Phase 3 Recruiting 35 2026-06-11 2026-08-05 A Study Investigating Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Adult Participants With Geographic Atrophy Pozelimab, Cemdisiran, Placebo - Age-related Macular Degeneration (AMD), Geographic Atrophy (GA) Phase 3 Recruiting 975 2024-10-30 2026-10-13 A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works Pozelimab, Cemdisiran - Paroxysmal Nocturnal Hemoglobinuria Phase 3 Recruiting 202 2023-03-07 2026-09-28 Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Cemdisiran, Eculizumab, Pozelimab - Paroxysmal Nocturnal Hemoglobinuria Phase 3 Terminated 3 2022-10-06 2025-04-08 A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment Ravulizumab, Pozelimab, Cemdisiran - Paroxysmal Nocturnal Hemoglobinuria Phase 3 Active not recruiting 202 2022-08-01 2026-04-30 A Study to Test How Safe Pozelimab and Cemdisiran Combination Therapy and Cemdisiran Alone Are and How Well They Work in Adult Patients With Generalized Myasthenia Gravis Pozelimab + Cemdisiran, Cemdisiran, Placebo - Generalized Myasthenia Gravis Phase 3 Active not recruiting 288 2021-12-14 2026-10-28 A Study to Examine the Safety, Tolerability and Biological Effects of Single Doses of Subcutaneously and Intravenously Administered Pozelimab as Monotherapy and in Combination With Single Doses of Subcutaneously Administered Cemdisiran in Adult Japanese Healthy Volunteers Pozelimab, Cemdisiran - Healthy Phase 1 Completed 30 2021-08-04 2022-07-06 Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy Pozelimab, Cemdisiran - Paroxysmal Nocturnal Hemoglobinuria Phase 2 Completed 24 2021-07-29 2025-04-08 Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy Pozelimab, Cemdisiran - Paroxysmal Nocturnal Hemoglobinuria Phase 2 Completed 6 2021-07-08 2025-08-29 Eculizumab to Cemdisiran Switch in aHUS cemdisiran, Placebos - Atypical Hemolytic Uremic Syndrome Phase 2 Withdrawn 2021-01-01 2021-03-10 Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Pozelimab in Combination With Cemdisiran in Healthy Adult Volunteers Pozelimab, Cemdisiran - Healthy Phase 1 Completed 19 2020-11-16 2021-11-12 A Study of Cemdisiran in Adults With Immunoglobulin A Nephropathy (IgAN) Placebo, Cemdisiran - IgA Nephropathy (IgAN), Berger Disease Phase 2 Terminated 31 2019-09-30 2024-08-09 A Study of an Investigational Drug, Cemdisiran (ALN-CC5), in Patients With Atypical Hemolytic Uremic Syndrome Cemdisiran - Atypical Hemolytic Uremic Syndrome Phase 2 Withdrawn 2017-09-19 2018-10-01 Compassionate Use of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria Pozelimab, Cemdisiran - Paroxysmal Nocturnal Hemoglobinuria No longer available 2025-10-20
Conditions studied Hemoglobinuria, Paroxysmal (7) · Atypical Hemolytic Uremic Syndrome (2) · Geographic Atrophy (1) · Glomerulonephritis, IGA (1) · Hemolysis (1) · Macular Degeneration (1) · Myasthenia Gravis (1) · Renal Insufficiency (1) · Thrombocytopenia (1) · Thrombotic Microangiopathies (1)
Sponsors Regeneron Pharmaceuticals (11) · Alnylam Pharmaceuticals (2) · Mario Negri Institute for Pharmacological Research (1)
Countries United Kingdom (8) · Canada (7) · Italy (6) · Spain (6) · Poland (5) · South Korea (5) · Taiwan (5) · Brazil (4) · Hungary (4) · Malaysia (4) · Turkey (Türkiye) (4) · United States (4) · France (3) · India (3) · Japan (3)
Top investigators Nader Najafian, MD (1)
Results publications PMID 40708708 , PMID 42030965
NIH funding 2023 (4) , 2018 (2)