Paroxysmal Nocturnal Hemoglobinuria (PNH)
41
11
12
20
Key Insights
Highlights
Success Rate
80% trial completion
Published Results
18 trials with published results (44%)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 37/100
12.2%
5 terminated out of 41 trials
80.0%
-6.5% vs benchmark
17%
7 trials in Phase 3/4
90%
18 of 20 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 20 completed trials
Clinical Trials (41)
Phase Ib Clinical Study to Evaluate the Safety and Tolerability of VSA012 Injection in Paroxysmal Nocturnal Hemoglobinuria
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
Safety, Tolerability, PK and PD of ADX-038 in Healthy Participants and Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients
REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria
Study of Ultomiris® (Ravulizumab) Safety in Pregnancy
Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients
A Phase I Clinical Trial to Evaluate CMS-D017 Following Single and Multiple Doses in Healthy Participants
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
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）
A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
An Extension Clinical Study of BCD-148 for the Treatment of Patients With PNH Previously Treated in Clinical Study No. BCD-148-2/NOCTURN
Coversin in PNH in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms
Long-term Safety and Tolerability of MY008211A Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria