Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A
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Phase
Phase 3
Status
Active, not recruiting
Study type
Interventional
Design
Na · None · Treatment
Enrollment
6estimated
Sites
4
Country
Japan
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2023-12-25 | actual | When the study began enrolling |
| Primary completion | 2025-04-16 | actual | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | Mar 2029 | estimated | The whole study's end, after follow-up |
| First posted | 2024-01-25 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-03-19 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Hemophilia A
Intervention
- Biological: Valoctocogene roxaparvovec — also filed as BMN 270, ROCTAVIAN
Primary outcome
what the primary-completion date above is the date OFChange in the human coagulation factor VIII (hFVIII) activity, as measured by chromogenic substrate assay, during Weeks 49 to 52 post BMN 270 infusion from Baseline.
measured 52 Weeks
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