Success Metrics

Clinical Success Rate
83.3%

Based on 5 completed trials

Completion Rate
83%(5/6)
Active Trials
1(14%)
Results Posted
100%(5 trials)
Terminated
1(14%)

Phase Distribution

Ph phase_1
3
43%
Ph phase_3
4
57%

Phase Distribution

3

Early Stage

0

Mid Stage

4

Late Stage

Phase Distribution7 total trials
Phase 1Safety & dosage
3(42.9%)
Phase 3Large-scale testing
4(57.1%)

Highest Phase Reached

Phase 3

Trial Status & Enrollment

Completion Rate

83.3%

5 of 6 finished

Non-Completion Rate

16.7%

1 ended early

Currently Active

1

trials recruiting

Total Trials

7

all time

Status Distribution
Active(1)
Completed(5)
Terminated(1)

Detailed Status

Completed5
Active, not recruiting1
Terminated1

Development Timeline

Analytics

Development Status

Total Trials
7
Active
1
Success Rate
83.3%
Most Advanced
Phase 3

Trials by Phase

Phase 13 (42.9%)
Phase 34 (57.1%)

Trials by Status

active_not_recruiting114%
completed571%
terminated114%

Recent Activity

1 active trials
Showing 5 of 7
completedphase_1

Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors

NCT04684940
active_not_recruitingphase_3

Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A

NCT06224907
completedphase_3

Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A

NCT04323098
terminatedphase_1

Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5

NCT03520712
completedphase_1

Gene Therapy Study in Severe Haemophilia A Patients (270-201)

NCT02576795
View all 7 trials

Clinical Trials (7)

Showing 7 of 7 trials
NCT04684940Phase 1

Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors

Completed
NCT06224907Phase 3

Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A

Active Not Recruiting
NCT04323098Phase 3

Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A

Completed
NCT03520712Phase 1

Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5

Terminated
NCT02576795Phase 1

Gene Therapy Study in Severe Haemophilia A Patients (270-201)

Completed
NCT03370913Phase 3

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)

Completed
NCT03392974Phase 3

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg

Completed

All 7 trials loaded

Drug Details

Intervention Type
BIOLOGICAL
Total Trials
7