Global PNH Patient Registry
- Status
- RECRUITING
- Phase
- N/A
Clinical Trials Related to Paroxysmal Nocturnal Hemoglobinuria
Related studies linked through entity extraction aliases.
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
- Status
- RECRUITING
- Phase
- PHASE3
Long-term Safety of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria
- Status
- TERMINATED
- Phase
- PHASE2
Study of NM8074 in Adult PNH Patients With Inadequate Response to Soliris
- Status
- NOT_YET_RECRUITING
- Phase
- PHASE2
Study of Efficacy and Safety of NM8074 in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy
- Status
- NOT_YET_RECRUITING
- Phase
- PHASE2
A Study of Single-dose MY008211A in Healthy Adults
- Status
- COMPLETED
- Phase
- PHASE1
Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan.
- Status
- COMPLETED
- Phase
- PHASE3
To Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Escalating Single and Multiple Doses of KP104
- Status
- COMPLETED
- Phase
- PHASE1
To Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- PHASE2
A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- PHASE3
Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria
- Status
- COMPLETED
- Phase
- N/A
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
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- PHASE3
Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria
- Status
- TERMINATED
- Phase
- PHASE3
Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)
- Status
- COMPLETED
- Phase
- PHASE2
Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy
- Status
- COMPLETED
- Phase
- PHASE2
Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy
- Status
- COMPLETED
- Phase
- PHASE2
Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria
- Status
- ACTIVE_NOT_RECRUITING
- Phase
- PHASE3
A Long-Term Safety Study of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Status
- TERMINATED
- Phase
- PHASE2
A Safety and Immunogenicity Study in Long-term Treatment of Eculizumab (JSC "GENERIUM", Russian Federation)
- Status
- COMPLETED
- Phase
- PHASE3
Prospective Observational Study of Long-term Pathogenic Treatment of Elizaria®
- Status
- COMPLETED
- Phase
- N/A