A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy
← catalyst calendarNCT06280209 · readout in 238 d
Sponsored by BioMarin Pharmaceutical (industry) · BMRN — their whole pipeline →.
Phase
Phase 1/2
Status
Active, not recruiting
Study type
Interventional
Design
Non randomized · None · Treatment
Enrollment
18actual
Sites
7
Countries
Italy, Netherlands, Spain +2
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 | 2024-01-03 | actual | When the study began enrolling |
| Primary completion | 2027-04-14 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2027-04-14 | estimated | The whole study's end, after follow-up |
| First posted | 2024-02-28 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-06-25 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Duchenne Muscular Dystrophy
Intervention
- Drug: BMN 351
Primary outcome
what the primary-completion date above is the date OFTo evaluate and safety and tolerability of single and multiple doses of BMN 351 (incidence, severity, and dose-relationship of adverse effects and changes in laboratory parameters).
measured Up to 97 weeks.
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