An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of Weekly Intravenous Infusions of BMN 351 in Participants with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

Status

Not yet recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2024-511656-41-00

Enrollment

Registered

2026-03-16

Start date

Unknown

Completion date

Unknown

Last updated

2026-03-16

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Duchenne Muscular Dystrophy

Brief summary

Incidence of adverse events/SAEs/AESI, physical examination, safety laboratory test parameters, ECG parameters, echocardiogram

Detailed description

NSAA (total score and selected individual components, i.e., timed 10MWRT and TTR) at baseline and subsequent Q24W visits, Timed 4SC at baseline and subsequent Q24W visits, SV95C (4-week monitoring periods preceding baseline and subsequent Q24W visits), PUL 2.0 at baseline and subsequent Q24W visits

Interventions

DRUGExon 51 specific phosphorothioate oligonucleotide

Eligibility

Sex/Gender

Male

Age

0 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
Incidence of adverse events/SAEs/AESI, physical examination, safety laboratory test parameters, ECG parameters, echocardiogram	—

Secondary

Measure	Time frame
NSAA (total score and selected individual components, i.e., timed 10MWRT and TTR) at baseline and subsequent Q24W visits, Timed 4SC at baseline and subsequent Q24W visits, SV95C (4-week monitoring periods preceding baseline and subsequent Q24W visits), PUL 2.0 at baseline and subsequent Q24W visits	—

Outcome results

None listed

Source: EU CTIS · Data processed: Mar 17, 2026