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NCT04323098 · ClinicalTrials.gov registry record · Phase 3
Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A
A Phase 3 study, sponsored by BioMarin Pharmaceutical.
- Completed
- Registry status
- Phase 3
- Development phase
- 22
- Enrollment target
NCT04323098: Completed Phase 3 study, sponsored by BioMarin Pharmaceutical.
NCT04323098 is a Phase 3 study that has completed, run by BioMarin Pharmaceutical. The registered enrollment target is 22 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (97% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04323098, a Phase 3 study, has completed, sponsored by BioMarin Pharmaceutical.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 22 participants
- Enrollment target
Study Summary
This Phase III clinical study will evaluate the safety and effectiveness of valoctocogene roxaparvovec in combination with prophylactic corticosteroids in patients with severe hemophilia A.
Primary Outcome
The change from baseline (assuming no treatment for severe hemophilia A) in FVIII activity, as measured by chromogenic substrate assay (CSA), at Week 52 (during Weeks 49 - 52) post-BMN 270 infusion. Each participant's FVIII activity level at Week 52 is defined as the median of the values obtained within the analysis window at Weeks 49-52. The baseline value will be imputed as 1 IU/dL, since there will be no washout of severe hemophilia A participants' usual FVIII prophylaxis (in order to avoid
Interventions
- BIOLOGICAL valoctocogene roxaparvovec
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 22 participants |
| Start Date | 2020-12-08 |
| Est. Completion | 2025-05-08 |
| Phase | Phase 3 |
What the finished NCT04323098 record still lists
NCT04323098 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 22 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (97% lower).
The record links to 0 conditions, and to 1 intervention - of which valoctocogene roxaparvovec is the first listed.
NCT04323098 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT04323098 about?
NCT04323098 is a clinical study titled "Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A". This Phase III clinical study will evaluate the safety and effectiveness of valoctocogene roxaparvovec in combination with prophylactic corticosteroids in patients with severe hemophilia A.
What is the current status of trial NCT04323098?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 22 participants. The study started on 2020-12-08. Estimated completion is 2025-05-08.
What interventions are being tested in trial NCT04323098?
The interventions under investigation include: valoctocogene roxaparvovec (BIOLOGICAL).
Who is sponsoring clinical trial NCT04323098?
This trial is sponsored by BioMarin Pharmaceutical, which has 80 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT04323098's enrollment target sits among peer trials
22 1953rd of 2000 higher than 48 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 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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