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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
EU CTIS ID: 2023-506737-30-00
What this study is testing
To assess the safety and tolerability of BMN 351 at different dose levels in participants with DMD
- Phase I and Phase II (Integrated)- First administration to humans
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1. Is male and age 4 through 10 years at Screening.
- 2. Clinical diagnosis of Duchenne muscular dystrophy in the opinion of the investigator resulting from a documented dystrophin mutation in the DMD gene amenable to exon 51 skipping as reviewed by a central genetic counselor.
- 3. Ambulatory at Screening, defined as able to walk independently without assistive devices and complete the timed 10 meter walk/run test in 8 seconds or less.
- 4. Not currently daytime ventilator dependent and not expected to need daytime mechanical or noninvasive ventilation within the next year in the opinion of the investigator.
- 5. Currently receiving treatment with oral corticosteroids or vamorolone, on a stable dose for at least 12 weeks prior to Baseline, and must remain on a consistent dose/dose regimen throughout the study except for modifications to accommodate changes in weight.
- 6. Normal urinalysis at Screening (trace protein allowed).
You likely can't join if
- 1. For children 7 years of age or older, forced expiratory volume (FEV1) < 60% of predicted.
- 2. Current or history of liver or renal disease.
- 3. Left ventricular ejection fraction (LVEF) < 55% based on an ECHO performed within 3 months prior to the Baseline (Day 1) visit.
- 4. Mean QT interval corrected with Fridericia’s method (QTcF) ≥ 450 msec on the Screening electrocardiogram (ECG) conducted in triplicate.
- 5. Platelet count of < 150 x 10^9/L at Screening.
- 6. Renal function laboratory parameters outside of prespecified values as defined per protocol.
See the full eligibility criteria
- 1. Is male and age 4 through 10 years at Screening.
- 2. Clinical diagnosis of Duchenne muscular dystrophy in the opinion of the investigator resulting from a documented dystrophin mutation in the DMD gene amenable to exon 51 skipping as reviewed by a central genetic counselor.
- 3. Ambulatory at Screening, defined as able to walk independently without assistive devices and complete the timed 10 meter walk/run test in 8 seconds or less.
- 4. Not currently daytime ventilator dependent and not expected to need daytime mechanical or noninvasive ventilation within the next year in the opinion of the investigator.
- 5. Currently receiving treatment with oral corticosteroids or vamorolone, on a stable dose for at least 12 weeks prior to Baseline, and must remain on a consistent dose/dose regimen throughout the study except for modifications to accommodate changes in weight.
- 6. Normal urinalysis at Screening (trace protein allowed).
- 1. For children 7 years of age or older, forced expiratory volume (FEV1) < 60% of predicted.
- 2. Current or history of liver or renal disease.
- 3. Left ventricular ejection fraction (LVEF) < 55% based on an ECHO performed within 3 months prior to the Baseline (Day 1) visit.
- 4. Mean QT interval corrected with Fridericia’s method (QTcF) ≥ 450 msec on the Screening electrocardiogram (ECG) conducted in triplicate.
- 5. Platelet count of < 150 x 10^9/L at Screening.
- 6. Renal function laboratory parameters outside of prespecified values as defined per protocol.
- 7. Treatment with any exon skipping therapy within 12 weeks prior to Baseline (Day 1) or with any gene therapy for the treatment of DMD at any time.
The study team makes the final eligibility decision.
Where it's taking place
- Turkey
- United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Turkey; United Kingdom. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.