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NCT04684940 · ClinicalTrials.gov registry record · Phase 1
Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors
A Phase 1 study of Hemophilia A With Inhibitor and Hemophilia A With Anti Factor VIII, sponsored by BioMarin Pharmaceutical.
- Active
- Registry status
- Phase 1
- Development phase
- 10
- Enrollment target
- 9
- Study locations
NCT04684940: Active Phase 1 study of Hemophilia A With Inhibitor and Hemophilia A With Anti Factor VIII, sponsored by BioMarin Pharmaceutical.
NCT04684940 is a Phase 1 study of Hemophilia A With Inhibitor and Hemophilia A With Anti Factor VIII that is active but no longer recruiting, run by BioMarin Pharmaceutical. The registered enrollment target is 10 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (83% lower). The trial reports 9 study locations across 2 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04684940, a Phase 1 study of Hemophilia A With Inhibitor and Hemophilia A With Anti Factor VIII, is active but no longer recruiting, sponsored by BioMarin Pharmaceutical.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 10 participants
- Enrollment target
- 9
- Study locations
Study Summary
This Phase I/II clinical study will evaluate the safety and efficacy of valoctocogene roxaparvovec in patients with severe haemophilia A and inhibitors to FVIII. Part A of the study will involve subjects who have active inhibitors to FVIII, and Part B involving subjects with a prior history of inhibitors.
Conditions Studied
Interventions
- BIOLOGICAL Valoctocogene roxaparvovec
Study Locations (9)
Other
- Hemocentro Da UNICAMP - Campinas
- Arthur De Siqueira Cavalcanti Hematology State Institute - Rio de Janeiro
- Chaim Sheba Medical Center - Ramat Gan
- Kyung Hee University Hospital at Gangdong - Seoul
- Kaohsiung Medical University - Chung-Ho Memorial Hospital - Kaohsiung City
- Taichung Veterans General Hospital - Taichung
- National Taiwan University Hospital - Taipei
- Ege University School of Medicine - Izmir
California
- Children's Hospital Los Angeles - Los Angeles
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 2020-12-10 |
| Est. Completion | 2029-04 |
| Phase | Phase 1 |
What the registry record for NCT04684940 still lists
NCT04684940 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 10 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (83% lower).
The record links to 2 conditions, with Hemophilia A With Inhibitor appearing as the primary indexed condition, and to 1 intervention - of which Valoctocogene roxaparvovec is the first listed.
NCT04684940 lists 9 locations in 2 states (Other, California).
Frequently Asked Questions
What is clinical trial NCT04684940 about?
NCT04684940 is a clinical study titled "Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors". This Phase I/II clinical study will evaluate the safety and efficacy of valoctocogene roxaparvovec in patients with severe haemophilia A and inhibitors to FVIII. Part A of the study will involve subjects who have active inhibitors to FVIII, and Part B involving subjects with a prior history of inhib...
What is the current status of trial NCT04684940?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 10 participants. The study started on 2020-12-10. Estimated completion is 2029-04.
What conditions does trial NCT04684940 study?
This clinical trial studies the following conditions: Hemophilia A With Inhibitor, Hemophilia A With Anti Factor VIII.
What interventions are being tested in trial NCT04684940?
The interventions under investigation include: Valoctocogene roxaparvovec (BIOLOGICAL).
Who is sponsoring clinical trial NCT04684940?
This trial is sponsored by BioMarin Pharmaceutical, which has 80 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04684940 being conducted?
This trial has 9 study locations across California. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT04684940's enrollment target sits among peer trials
10 1913th of 2000 higher than 46 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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