Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors
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Phase
Phase 1/2
Status
Completed
Study type
Interventional
Design
Na · None · Treatment
Enrollment
10actual
Sites
9
Countries
Brazil, Israel, South Korea +4
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 | 2020-12-10 | actual | When the study began enrolling |
| Primary completion | 2026-04-20 | actual | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2026-04-20 | actual | The whole study's end, after follow-up |
| First posted | 2020-12-28 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-07-10 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Conditions
- Hemophilia A With Inhibitor
- Hemophilia A With Anti Factor VIII
Intervention
- Biological: Valoctocogene roxaparvovec — also filed as BMN 270 (GENEr8)
Primary outcome
what the primary-completion date above is the date OFNumber of participants with treatment-related adverse events, as assessed by Common Terminology Criteria for Adverse Events (CTCAE) v5.0 after administration of BMN 270.
measured 60 months
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