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Enrolling by invitation NCT07573631

An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy

Phase II Interventional Duchenne Muscular Dystrophy (DMD)

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: BMN 351.
Who it may be relevant to
Registry conditions: Duchenne Muscular Dystrophy (DMD). Basic parameters: from 4 years · Male.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Italy, Netherlands, Spain, Turkey (Türkiye), United Kingdom
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of Weekly Intravenous Infusions of BMN 351 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

Overview

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 effect of BMN 351 on physical function, in participants with DMD who participated in the 351-201 study.

Detailed description

This Phase 2, multi-center, open-label extension study is designed to assess the long-term safety, tolerability, and functional efficacy of weekly intravenous doses of BMN 351 administered to participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping in the Phase 1/2 study, 351-201. Up to 18 participants ages 4 through 23 at baseline will enroll in the trial after completing 351-201.

The first visit for this study is the same as the final visit of 351-201. To be eligible for this study, potential participants must satisfy the eligibility criteria described in the protocol.

Interventions

  • Drug BMN 351
    Anti-sense Oligonucleotide BMN 351 will be administered intravenously

Primary outcome measures

  • To assess the long-term safety and tolerability of BMN 351 in participants with DMD [Time frame: Through study completion, at least 1 year]
Secondary outcome measures (4)
  • To evaluate the effect of BMN 351 on physical function [Time frame: Change from baseline and subsequent 24-week incremental visits]
  • To evaluate the effect of BMN 351 on physical function [Time frame: Change from baseline and subsequent 24-week incremental visits]
  • To evaluate the effect of BMN 351 on physical function [Time frame: Change from baseline and subsequent 24-week incremental visits]
  • To evaluate the effect of BMN 351 on physical function [Time frame: Change from baseline and subsequent 24-week incremental visits]

Eligibility criteria

Inclusion criteria

  • Participants must have completed 351-201 without permanent discontinuation of the investigational medicinal product (IMP) or withdrawal from the study
  • Currently receiving treatment with oral corticosteroids, on a stable dose regimen during 351-201, and must remain on a consistent dose regimen throughout 351-202 or 351-203 except for modifications to accommodate changes in weight
  • Transition to the equivalent dose of vamorolone is permitted in 351-202 where approved in participating countries.
  • Willing and able to adhere to the study visit schedule and other protocol requirements
  • Willing to use contraception (sexually mature males) throughout the study and for 90 days after the final dose, if sexually active
  • Contraceptive use by males should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
  • Willing and able to provide written assent (if required by local regulations or the IRB/IEC) after the nature of the study has been explained and prior to performance of any research-related procedure
  • Willing and able to provide written, signed informed consent as parent or guardian after the nature of the study has been explained and prior to performance of any research-related procedure

Exclusion criteria

  • Have known coagulation disorder
  • Are taking any prohibited medications
  • any approved exon skipping therapy within 12 weeks prior to baseline or with any gene therapy for the treatment of DMD at any time
  • anti-coagulants, anti-thrombotics, or anti-platelet agents
  • immunosuppressants

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

Italy · 2 centers
  • Fondazione Serena ETS - Centro Clinico NeMO Milano — Milan
  • UOC Fase I - Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica — Rome
Netherlands · 1 center
  • Leids Universitair Medisch Centrum — Leiden
Spain · 1 center
  • Hospital Viamed Santa Angela De la Cruz — Seville
Turkey (Türkiye) · 1 center
  • Yeditepe University Kosuyolu Hospital — Istanbul
United Kingdom · 1 center
  • Great Ormond Street Hospital NHS Foundation Trust — London

Identifiers

NCT: NCT07573631 · 351-202 · 2024-511656-41-00

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗