A Phase 1/2, Open-Label, Dose Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Multiple Intravenous Doses of BMN 351 in Participants with Duchenne Muscular Dystrophy

Status

Recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2023-506737-30-00

Acronym

351-201

Enrollment

Registered

2023-11-30

Start date

2024-03-08

Completion date

Unknown

Last updated

2026-01-15

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

1. Incidence of adverse events/SAEs/AESI, physical examination, safety laboratory test parameters, ECG parameters, echocardiography, 2. Physical examination, 3. Safety laboratory test parameters, 4. ECG parameters

Detailed description

1. BMN 351 plasma PK, urine PK and concentration in muscle

Interventions

DRUGExon 51 specific phosphorothioate oligonucleotide

Eligibility

Sex/Gender

Male

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
1. Incidence of adverse events/SAEs/AESI, physical examination, safety laboratory test parameters, ECG parameters, echocardiography, 2. Physical examination, 3. Safety laboratory test parameters, 4. ECG parameters	—

Secondary

Measure	Time frame
1. BMN 351 plasma PK, urine PK and concentration in muscle	—

Countries

Italy, Netherlands, Spain

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026