BMN 351 for Duchenne Muscular Dystrophy
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to test BMN 351, an experimental treatment for Duchenne Muscular Dystrophy (DMD), specifically targeting those with a genetic change that allows exon 51 skipping. The primary goal is to assess the treatment's safety and tolerability. Participants will receive varying doses, either once every two weeks or weekly, based on their group. The trial seeks boys aged 4 to 10 who can walk, do not require a ventilator, and have maintained a stable dose of steroids for the past 12 weeks. As a Phase 1/Phase 2 trial, this research focuses on understanding the treatment's effects and measuring its initial effectiveness in a smaller group.
Is there any evidence suggesting that BMN 351 is likely to be safe for humans?
Research has shown that BMN 351 was safe in early studies, with participants tolerating the treatment well. Initial results have been positive regarding safety, suggesting that BMN 351 might be safe for individuals with Duchenne Muscular Dystrophy (DMD), particularly those with a specific genetic mutation treatable by skipping exon 51. However, it is important to remember that this treatment remains under study, and ongoing research will provide more information about its safety.12345
Why do researchers think this study treatment might be promising for Duchenne Muscular Dystrophy?
Unlike the current treatments for Duchenne Muscular Dystrophy, which often involve corticosteroids to slow muscle degeneration, BMN 351 targets the disease differently. This new treatment is designed to address the genetic causes of the disease by modulating splicing of the dystrophin gene. Researchers are excited because BMN 351 offers a potential for more direct intervention at the genetic level, which could lead to more effective and longer-lasting results. Additionally, its varied dosing regimens, including high, medium, and low doses, allow for personalized treatment plans that could optimize outcomes for different patients.
What evidence suggests that BMN 351 might be an effective treatment for Duchenne Muscular Dystrophy?
Studies have shown that BMN 351 can increase levels of dystrophin, a crucial protein missing in boys with Duchenne Muscular Dystrophy (DMD). Increasing dystrophin may potentially slow the disease's progression. In tests with mice, BMN 351 improved heart and leg muscle conditions. Early trial results with boys have demonstrated that the treatment is safe and increases dystrophin levels. This trial will evaluate BMN 351 across different dosing regimens, including single and multiple ascending doses, to further assess its safety and effectiveness. These findings suggest that BMN 351 could be a helpful treatment for those with the exon 51 skipping mutation in DMD.23467
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive BMN 351 at escalating doses in different cohorts with varying dosing schedules
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- BMN 351
How Is the Trial Designed?
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Treatment groups
Experimental Treatment
BMN 351 high dose will be administered once weekly for up to 48 weeks
BMN 351 medium dose will be administered once weekly for up to 73 weeks
BMN 351 low dose will be administered once weekly for up to 97 weeks
Cohort 1A will consist of both a single ascending dose (SAD) part and a multiple ascending dose (MAD). BMN 351 will be administered once every 2 weeks during the SAD portion of the study for up to 8 weeks and once weekly during the MAD portion for up to 89 weeks.
Find a Clinic Near You
Who Is Running the Clinical Trial?
BioMarin Pharmaceutical
Lead Sponsor
Alexander Hardy
BioMarin Pharmaceutical
Chief Executive Officer since 2023
MBA from INSEAD
Greg Friberg
BioMarin Pharmaceutical
Chief Medical Officer
MD from New York Medical College
Citations
BMN 351-Induced Exon Skipping and Dystrophin ...
Compared with vehicle-treated hDMDdel52/mdx mice, BMN 351 ameliorated DMD-related histopathologic changes in the gastrocnemius muscle and heart.
An Open-Label Extension Study to Evaluate the Long-term ...
This open-label extension study aims to evaluate the long-term safety and tolerability of weekly BMN 351 infusions, as well as to assess the ...
Initial results from an open-label phase 1/2 study of BMN ...
Interim analyses demonstrated acceptable safety and positive dystrophin changes in boys with DMD receiving BMN 351 ≤9mg/kg. PS chemistry-based PK/PD modeling ...
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clinicaltrials.biomarin.com
clinicaltrials.biomarin.com/clinical-trial/a-phase-1-2-study-to-assess-the-safety-tolerability-pharmacokinetics-and-pharmacodynamics-of-bmn-351-in-participants-with-duchenne-muscular-dystrophy/A Phase 1/2 Study to Assess the Safety, Tolerability ...
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 ...
BMN 351
BMN 351 is an exon skipping therapy for those amenable to exon 51 skipping. Status: A Phase 1/2 trial is actively recruiting Ex-US.
NCT06280209 | A Phase 1/2 Study to Assess the Safety, ...
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 ...
A Phase 1/2 Study to Assess the Safety, Tolerability ...
The purpose of this study is to test the safety and tolerability of BMN 351 in participants with Duchenne Muscular Dystrophy (DMD) with a ...
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