Hemophilia A
365
40
54
217
Key Insights
Highlights
Success Rate
87% trial completion (above average)
Published Results
96 trials with published results (26%)
Research Maturity
217 completed trials (59% of total)
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 17/100
8.8%
32 terminated out of 365 trials
87.1%
+0.6% vs benchmark
32%
117 trials in Phase 3/4
44%
96 of 217 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 217 completed trials
Clinical Trials (365)
Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively
Synovial Proliferation on Routine Ultrasound: Active or Inactive?
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis
A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A
Lentiviral FVIII Gene Therapy
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
ATHNdataset Registry
Searching Patterns In the Robustness of Immunological FVIII Tolerance
Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a "Shuttle"
A Study to Learn More About the Safety of Damoctocog-alfa-pegol When Used in Routine Medical Care in Korean Participants With Hemophilia A
An Observational Study to Learn More About How Well Damoctocog Alfa Pegol Works in Previously Treated Children With Hemophilia A
Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A
An Observational Study Called JOIHA to Learn More About How Well the Treatment With Jivi Works to Prevent Problems With Joints in Adults With Hemophilia A.
Joint Health, Balance and Quality of Life in Adults With Hemophilia A
Evaluating Effectiveness and Long Term Safety of Damoctocog Alfa Pegol in Patients, Who Have Been Diagnosed With Hemophilia A
Study to Learn More About the Safety of Drug Jivi Over a Long Period of Time in Previously Treated Patients With Hemophilia A (Bleeding Disorder Resulting From a Lack of FVIII) Who Are Receiving Jivi Regularly at Their Treating Doctors to Prevent Bleeding
Liver Biopsy Following Gene Therapy For Hemophilia
Efficacy, Safety, and Pharmacokinetic Study of Prophylactic Emicizumab Versus No Prophylaxis in Hemophilia A Participants