At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- BMN 351 (drug)
- How long the study runs
- Study runs about 39 months (dates as stated)
- About the drug or intervention
- BMN 351 — drug: Anti-sense Oligonucleotide BMN 351 will be administered intravenously.
- Patient visit burden
- Not specified by the sponsor
- Type of study
- Testing a treatment
- Ages
- 4 Years to 10 Years
- Who
- Male
- Number of participants
- 18
- Started
- 2024-01-03
- Last checked
- 2026-06
Plain English Summary
What is this study?
- • Testing a new treatment for duchenne muscular dystrophy
- • Phase1/Phase2 - 18 participants
- • 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
Who can take part?
- • Ages 4 Years to 10 Years
- • Diagnosed with duchenne muscular dystrophy
- • Male only
Where?
- • London - Great Ormond Street Hospital NHS Foundation Trust
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
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.
More detail
This is Phase 1/2, open-label, multi-center study consisting of 2 parts to evaluate the safety and tolerability of BMN 351 at escalating doses in participants with Duchenne Muscular Dystrophy (DMD) with genetic mutations amenable to exon 51 skipping. Participants will be assigned to one of three groups called cohorts (Cohort 1, 2 or 3). Cohort 1 participants are further divided into Cohort 1A and Cohort 1B. In Cohort 1A, 3 participants will receive increasing doses once every 2 weeks with a visit to assess safety measures collected the week after dosing prior to escalating doses of BMN 351. In part 2, the participants in cohort 1A will transition to once weekly dosing. The participants in Cohort 1B, 2, and 3 will initiate low, medium, and high doses of BMN 351 and continue once weekly dosing at that same dose. The study will enroll approximately 18 participants.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 4 Years - 10 Years
- Who can join: Male only
What the study is looking for
- ✓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
Who cannot take part
- ✗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
See the full criteria
Where Is This Study? (1 UK site)
Great Ormond Street Hospital NHS Foundation Trust
London WC1N 3JH, United Kingdom
Main Email: Research.Governance@gosh.nhs.uk
Research.Governance@gosh.nhs.uk0207 905 2700