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.
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
18
Anti-sense Oligonucleotide BMN 351 will be administered intravenously
Fondazione Serena ETS - Centro Clinico NeMO Milano
Milan, Italy, Italy
UOC Fase I - Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
Rome, Italy, Italy
Leids Universitair Medisch Centrum
Leiden, Netherlands, Netherlands
Hospital Viamed Santa Angela De la Cruz
Seville, Spain, Spain
To assess the long-term safety and tolerability of BMN 351 in participants with DMD
The safety and tolerability of BMN 351 will be assessed based on the incidence of adverse and serious adverse events.
Time frame: Through study completion, at least 1 year
To evaluate the effect of BMN 351 on physical function
North Star Ambulatory Assessment (NSAA) will be assessed at the specific visits and compared to individual baseline and external controls
Time frame: Change from baseline and subsequent 24-week incremental visits
To evaluate the effect of BMN 351 on physical function
Timed 4 stair climb (4SC) will be assessed at the specified visits and compared to individual baseline and external controls
Time frame: Change from baseline and subsequent 24-week incremental visits
To evaluate the effect of BMN 351 on physical function
SV95C will be assessed at the specified visits and compared to individual baseline and external controls
Time frame: Change from baseline and subsequent 24-week incremental visits
To evaluate the effect of BMN 351 on physical function
PUL 2.0 will be assessed at the specified visits and compared to individual baseline and external controls
Time frame: Change from baseline and subsequent 24-week incremental visits
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Yeditepe University Kosuyolu Hospital
Istanbul, Turkey, Turkey (Türkiye)
Great Ormond Street Hospital NHS Foundation Trust
London, United Kingdom, United Kingdom