Key Insights

Highlights

Success Rate

87% trial completion (above average)

Published Results

96 trials with published results (26%)

Research Maturity

218 completed trials (59% of total)

Clinical Risk Assessment

Based on trial outcomes

Low Risk

Score: 17/100

Termination Rate

8.9%

33 terminated out of 369 trials

Success Rate

86.9%

+0.3% vs benchmark

Late-Stage Pipeline

32%

117 trials in Phase 3/4

Results Transparency

44%

96 of 218 completed with results

Key Signals

96 with results87% success33 terminated

Data Visualizations

Phase Distribution

222Total
Not Applicable (36)
Early P 1 (3)
P 1 (45)
P 2 (21)
P 3 (73)
P 4 (44)

Trial Status

Completed218
Recruiting40
Unknown39
Terminated33
Active Not Recruiting17
Not Yet Recruiting10

Trial Success Rate

86.9%

Benchmark: 86.5%

Based on 218 completed trials

Clinical Trials (369)

Showing 20 of 20 trials
NCT05932914Not Yet RecruitingPrimary

Liver Biopsy Following Gene Therapy For Hemophilia

NCT04644575Phase 3Active Not RecruitingPrimary

Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A

NCT07226206Phase 1RecruitingPrimary

A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A

NCT03818763Phase 1Active Not RecruitingPrimary

Gene Therapy Trial for Platelet Derived Factor VIII Production in Hemophilia A

NCT07692217Not Yet RecruitingPrimary

TG-INSIGHT With Joint POCUS, Hemostatic Potential in Patients With Severe Hemophilia A on Novel Replacement and Substitution FVIII Therapies

NCT07416526Phase 3RecruitingPrimary

A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A

NCT07416604Phase 3RecruitingPrimary

A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People With Hemophilia A

NCT03588299Phase 1Active Not RecruitingPrimary

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"

NCT05987449Phase 1RecruitingPrimary

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A

NCT05022459RecruitingPrimary

Prevention of Bleeding in Patients With Moderate and Severe Hemophilia A Playing Sports: A Comparison Between Factor VIII and Emicizumab Prophylaxis

NCT05147662Phase 3Completed

A Study to Learn How Safe the Study Treatment BAY94-9027 is and How it Affects the Body in Previously Treated Children Aged 7 to Less Than 12 Years With Severe Hemophilia A, a Genetic Bleeding Disorder That is Caused by the Lack of a Protein Called Clotting Factor 8 (FVIII) in the Blood

NCT06349473Phase 1Recruiting

A Study of Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Two Participants Groups (Part A: Healthy Participants, and Part B: Participants With Hemophilia A or Hemophilia B or Factor VII Deficiency)

NCT05145127Phase 3RecruitingPrimary

Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors

NCT05643560Active Not RecruitingPrimary

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.

NCT03655223Active Not Recruiting

Early Check: Expanded Screening in Newborns

NCT03932201Active Not RecruitingPrimary

Evaluating Effectiveness and Long Term Safety of Damoctocog Alfa Pegol in Patients, Who Have Been Diagnosed With Hemophilia A

NCT04461639Active Not RecruitingPrimary

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

NCT06940830Phase 4RecruitingPrimary

Long-term Study Evaluating Joint Health in People With Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog Alfa

NCT07088458Not Yet RecruitingPrimary

An Observational Study to Learn More About How Well Damoctocog Alfa Pegol Works in Previously Treated Children With Hemophilia A

NCT06579144Phase 1CompletedPrimary

Pharmacokinetic Comparison of Efanesoctocog Alfa vs Other EHL-rFVIII Products in Participants With Severe Haemophilia A

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