Paroxysmal Nocturnal Hemoglobinuria, PNH
44
12
13
20
Key Insights
Highlights
Success Rate
80% trial completion
Published Results
18 trials with published results (41%)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 37/100
11.4%
5 terminated out of 44 trials
80.0%
-6.6% vs benchmark
18%
8 trials in Phase 3/4
90%
18 of 20 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 20 completed trials
Clinical Trials (44)
A Multicenter, Randomized, Open-Label, Positive Controlled Phase III Study to Evaluate the Efficacy and Safety of VSA012 Injection in Participants With Paroxysmal Nocturnal Hemoglobinuria Who Are Naive to Complement Inhibitor Therapy
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
To Evaluate Safety, Tolerability, PK/PD & Food Effect of Single/Multiple Oral TUL321 Capsules in Healthy Participants.
Efficacy and Safety of Lanoracopan in the Treatment of PNH
Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients
A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Factor B, in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria With Persistent Anemia on a C5 Inhibitor
A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria
Safety, Tolerability, PK and PD of ADX-038 in Healthy Participants and Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients
Phase Ib Clinical Study to Evaluate the Safety and Tolerability of VSA012 Injection in Paroxysmal Nocturnal Hemoglobinuria
Study of Ultomiris® (Ravulizumab) Safety in Pregnancy
A Phase I Clinical Trial to Evaluate CMS-D017 Following Single and Multiple Doses in Healthy Participants
National Longitudinal Cohort of Hematological Diseases
SLN12140 in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China
A Study of Single and Multiple Doses of LP-005 in Healthy Adult Participants
Managed Access Programs for LNP023, Iptacopan
Investigation of Pharmacokinetics,Safety,and Pharmacodynamics of HSK39297 in Subjects With Hepatic Impairment
Hematological Disorders in EHPVO Patients
Study of Safety and Efficacy of MY008211A in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
A Study to Evaluate the Efficacy and Safety of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria（PNH）