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A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy

NCT06280209 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 18

Last updated 2026-04-08

No results posted yet for this study

Summary

The purpose of this study is to test the safety and tolerability of BMN 351 in participants with Duchenne Muscular Dystrophy (DMD) with a genetic mutation amenable to exon 51 skipping.

Conditions

Interventions

DRUG

BMN 351

Anti-sense Oligonucleotide BMN 351 will be administered intravenously.

Sponsors & Collaborators

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
4 Years
Max Age
10 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-01-03
Primary Completion
2026-09-30
Completion
2026-09-30

Countries

  • Canada
  • Italy
  • Netherlands
  • Spain
  • Turkey (Türkiye)
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06280209 on ClinicalTrials.gov