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| ID | Type | Description | Link |
|---|---|---|---|
| 2024-511656-41-00 | EU Trial (CTIS) Number |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| BMN 351 | Experimental | Participants from 351-201 enrolling in 351-202 will initially receive BMN 351 at the dose level at which they completed 351-201. Once all 351-201 participants have completed their Week 25 visit in 351-202, available safety and PD data will be analyzed and a single optimal dose level will be selected for 351-202. All participants from 351-201 will transition to that dose level at their next applicable visit. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| BMN 351 | Drug | Anti-sense Oligonucleotide BMN 351 will be administered intravenously |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Through study completion, at least 1 year |
| Measure | Description | Time Frame |
|---|---|---|
| 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 contraols | Change from baseline and subsequent 24-week incremental visits |
| To evaluate the effect of BMN 351 on physical function |
| Measure | Description | Time Frame |
|---|---|---|
| To evaluate the trough plasma concentration of BMN 351 | Serial predose measurements of trough plasma PK | Baseline, Week 13, Week 25, Week 37, Week 49 |
| To evaluate the immune response to BMN 351 | Anti-BMN 351 antibodies and Anti-dystrophin antibodies |
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
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
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
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Fondazione Serena ETS - Centro Clinico NeMO Milano | Milan | Italy | ||||
| UOC Fase I - Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore |
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Open-Label Extension study available to eligible participants from the 351-201 study
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Timed 4 stair climb (4SC) will be assessed at the specified visits and compared to individual baseline and external controls |
| 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 | 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 | Change from baseline and subsequent 24-week incremental visits |
| Change from baseline and subsequent 24-week incremental visits |
| To evaluate lung function longitudinally for participants ages 7 and above | Forced Expiratory Volume in 1 second (FEV1) for participants ages 7 and above | Change from baseline to subsequent 24-week incremental visits |
| To evaluate lung function longitudinally for participants ages 7 and above | Forced Vital Capacity (FVC) for participants ages 7 and above | Change from baseline to subsequent 24-week incremental visits |
| To evaluate lung function longitudinally for participants ages 7 and above | Peak Cough Flow (PCF) for participants ages 7 and above | Change from baseline to subsequent 24-week incremental visits |
| To evaluate lung function longitudinally for participants ages 7 and above | Maximal Inspiratory Pressure (MIP) for participants ages 7 and above | Change from baseline to subsequent 24-week incremental visits |
| To evaluate lung function longitudinally for participants ages 7 and above | Maximal Expiratory Pressure (MEP) for participants ages 7 and above | Change from baseline to subsequent 24-week incremental visits |
| Rome |
| Italy |
| Leids Universitair Medisch Centrum | Leiden | 2333 ZA | Netherlands |
| Hospital Sant Joan de Deu | Barcelona | 08950 | Spain |
| Hospital Viamed Santa Angela De la Cruz | Seville | 41013 | Spain |
| Yeditepe University Kosuyolu Hospital | Istanbul | Turkey | Turkey (Türkiye) |
| Great Ormond Street Hospital NHS Foundation Trust | London | WC1N 3JH | United Kingdom |
| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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