A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema
← catalyst calendarNCT05121376 · readout ≤ 834 d
Sponsored by BioMarin Pharmaceutical (industry) · BMRN — their whole pipeline →.
Phase
Phase 1/2
Status
Active, not recruiting
Study type
Interventional
Design
Na · None · Treatment
Enrollment
44estimated
Sites
16
Countries
Australia, Spain, United States
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 | 2022-02-15 | actual | When the study began enrolling |
| Primary completion | Nov 2028 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | Nov 2028 | estimated | The whole study's end, after follow-up |
| First posted | 2021-11-16 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2024-05-16 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Conditions
- Hereditary Angioedema
- HAE
Interventions
- Genetic: Dose 1 of BMN 331
- Genetic: Dose 2 of BMN 331
- Genetic: Dose 3 of BMN 331
- Genetic: Dose 4 of BMN 331
- Genetic: Dose 5 of BMN 331
- Genetic: Dose 6 of BMN 331
- Genetic: Dose 7 of BMN 331
Primary outcome
what the primary-completion date above is the date OFNumber of participants with treatment-emergent adverse events following a single IV administration of BMN 331
measured At 5 years
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