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Clinical Trials in the UK / NCT06280209
Active, not recruiting Phase 1/2

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

NCT06280209 · tracked via the Priya Life Science UK tracker
Sponsor
BioMarin Pharmaceutical
Phase
Phase 1/2
Started
2024-01-03
Last updated
2026-06-25

Condition(s) studied

Duchenne Muscular Dystrophy

Investigational drug(s) / intervention(s)

BMN 351 →

BMN 351: Anti-sense Oligonucleotide BMN 351 will be administered intravenously.

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.

Eligibility

Sex
MALE
Min age
4 Years
Max age
10 Years
Healthy volunteers
No
Inclusion Criteria: * Age 4 to 10 * Diagnosis of Duchenne muscular dystrophy with a specific genetic change amenable to exon 51 skipping * Able to walk * Not requiring assistance from a ventilator to breathe * Currently on consistent doses of steroid treatment for the last 12 weeks Exclusion Criteria: * The participant will have some initial clinical labs and studies to assess baseline level of heart and lung function. * Treatment with an exon skipping therapy within 12 weeks prior to the first visit. * Any history of treatment with gene therapy

Primary outcome measure(s)

  • To 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). — Up to 97 weeks.
    The safety and tolerability of BMN 351 will be assessed based on the incidence of adverse and serious adverse events.

Trial sites (7)

FacilityCityRegionStatus
Fondazione Serena ETS - Centro Clinico NeMO Milano Milan Italy
UOC Fase I - Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore Rome Italy
Leids Universitair Medisch Centrum Leiden Netherlands
Hospital Sant Joan de Deu Barcelona Spain
Hospital Viamed Santa Angela De la Cruz Seville Spain
Yeditepe University Kosuyolu Hospital Istanbul Turkey (Türkiye)
Great Ormond Street Hospital NHS Foundation Trust London United Kingdom

More BioMarin Pharmaceutical trials in the UK

An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy Enrolling by invitation
A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH) Active, not recruiting
Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia Active, not recruiting
A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia Active, not recruiting
AAV Gene Therapy Study for Subjects with PKU Active, not recruiting

Other trials for the same condition

Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Then 6 Years Old Recruiting
A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD) Active, not recruiting
Open-Label Extension Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy Enrolling by invitation
Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients Active, not recruiting
A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy Active, not recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06280209 on ClinicalTrials.gov ↗ ← All trials in the UK