Duchenne Muscular Dystrophy (DMD)
Conditions
Brief summary
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
DRUGBMN 351
Anti-sense Oligonucleotide BMN 351 will be administered intravenously
Sponsors
BioMarin Pharmaceutical
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Open-Label Extension study available to eligible participants from the 351-201 study
Eligibility
Sex/Gender
MALE
Age
4 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| To assess the long-term safety and tolerability of BMN 351 in participants with DMD | Through study completion, at least 1 year | The safety and tolerability of BMN 351 will be assessed based on the incidence of adverse and serious adverse events. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| To evaluate the effect of BMN 351 on physical function | Change from baseline and subsequent 24-week incremental visits | North Star Ambulatory Assessment (NSAA) will be assessed at the specific visits and compared to individual baseline and external controls |
Countries
Italy, Netherlands, Spain, Turkey (Türkiye), United Kingdom
Outcome results
None listed