An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy
23 juli 2026 bijgewerkt door: BioMarin Pharmaceutical
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
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.
Studie Overzicht
Toestand
Aanmelden op uitnodiging
Conditie
Interventie / Behandeling
Gedetailleerde beschrijving
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.
Studietype
Ingrijpend
Inschrijving (Geschat)
18
Fase
- Fase 2
Contacten en locaties
In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.
Studie Locaties
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Italy
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Milan, Italy, Italië
- Fondazione Serena ETS - Centro Clinico NeMO Milano
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Rome, Italy, Italië
- UOC Fase I - Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
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Netherlands
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Leiden, Netherlands, Nederland, 2333 ZA
- Leids Universitair Medisch Centrum
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Spain
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Seville, Spain, Spanje, 41013
- Hospital Viamed Santa Angela De la Cruz
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Turkey
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Istanbul, Turkey, Turkije (Türkiye)
- Yeditepe University Kosuyolu Hospital
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United Kingdom
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London, United Kingdom, Verenigd Koninkrijk, WC1N 3JH
- Great Ormond Street Hospital NHS Foundation Trust
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Deelname Criteria
Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.
Geschiktheidscriteria
Leeftijden die in aanmerking komen voor studie
- Kind
- Volwassen
- Oudere volwassene
Accepteert gezonde vrijwilligers
Nee
Beschrijving
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
Studie plan
Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.
Hoe is de studie opgezet?
Ontwerpdetails
- Primair doel: Behandeling
- Toewijzing: NVT
- Interventioneel model: Opdracht voor een enkele groep
- Masker: Geen (open label)
Wapens en interventies
Deelnemersgroep / Arm |
Interventie / Behandeling |
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Experimenteel: BMN 351
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.
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Anti-sense Oligonucleotide BMN 351 will be administered intravenously
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Wat meet het onderzoek?
Primaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
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To assess the long-term safety and tolerability of BMN 351 in participants with DMD
Tijdsspanne: Through study completion, at least 1 year
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The safety and tolerability of BMN 351 will be assessed based on the incidence of adverse and serious adverse events.
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Through study completion, at least 1 year
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Secundaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
|---|---|---|
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To evaluate the effect of BMN 351 on physical function
Tijdsspanne: Change from baseline and subsequent 24-week incremental visits
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Timed 4 stair climb (4SC) will be assessed at the specified visits and compared to individual baseline and external controls
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Change from baseline and subsequent 24-week incremental visits
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To evaluate the effect of BMN 351 on physical function
Tijdsspanne: Change from baseline and subsequent 24-week incremental visits
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SV95C will be assessed at the specified visits and compared to individual baseline and external controls
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Change from baseline and subsequent 24-week incremental visits
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To evaluate the effect of BMN 351 on physical function
Tijdsspanne: Change from baseline and subsequent 24-week incremental visits
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PUL 2.0 will be assessed at the specified visits and compared to individual baseline and external controls
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Change from baseline and subsequent 24-week incremental visits
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To evaluate the effect of BMN 351 on physical function
Tijdsspanne: Change from baseline and subsequent 24-week incremental visits
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North Star Ambulatory Assessment (NSAA) will be assessed at the specific visits and compared to individual baseline and external controls
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Change from baseline and subsequent 24-week incremental visits
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Andere uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
|---|---|---|
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To evaluate the trough plasma concentration of BMN 351
Tijdsspanne: Baseline, Week 13, Week 25, Week 37, Week 49
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Serial predose measurements of trough plasma PK
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Baseline, Week 13, Week 25, Week 37, Week 49
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To evaluate the immune response to BMN 351
Tijdsspanne: Change from baseline and subsequent 24-week incremental visits
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Anti-BMN 351 antibodies and Anti-dystrophin antibodies
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Change from baseline and subsequent 24-week incremental visits
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To evaluate lung function longitudinally for participants ages 7 and above
Tijdsspanne: Change from baseline to subsequent 24-week incremental visits
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Forced Expiratory Volume in 1 second (FEV1) for participants ages 7 and above
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Change from baseline to subsequent 24-week incremental visits
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To evaluate lung function longitudinally for participants ages 7 and above
Tijdsspanne: Change from baseline to subsequent 24-week incremental visits
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Forced Vital Capacity (FVC) for participants ages 7 and above
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Change from baseline to subsequent 24-week incremental visits
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To evaluate lung function longitudinally for participants ages 7 and above
Tijdsspanne: Change from baseline to subsequent 24-week incremental visits
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Peak Cough Flow (PCF) for participants ages 7 and above
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Change from baseline to subsequent 24-week incremental visits
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To evaluate lung function longitudinally for participants ages 7 and above
Tijdsspanne: Change from baseline to subsequent 24-week incremental visits
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Maximal Inspiratory Pressure (MIP) for participants ages 7 and above
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Change from baseline to subsequent 24-week incremental visits
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To evaluate lung function longitudinally for participants ages 7 and above
Tijdsspanne: Change from baseline to subsequent 24-week incremental visits
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Maximal Expiratory Pressure (MEP) for participants ages 7 and above
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Change from baseline to subsequent 24-week incremental visits
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Medewerkers en onderzoekers
Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.
Sponsor
Studie record data
Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.
Bestudeer belangrijke data
Studie start (Werkelijk)
20 april 2026
Primaire voltooiing (Geschat)
1 december 2031
Studie voltooiing (Geschat)
1 december 2031
Studieregistratiedata
Eerst ingediend
17 april 2026
Eerst ingediend dat voldeed aan de QC-criteria
1 mei 2026
Eerst geplaatst (Werkelijk)
7 mei 2026
Updates van studierecords
Laatste update geplaatst (Werkelijk)
24 juli 2026
Laatste update ingediend die voldeed aan QC-criteria
23 juli 2026
Laatst geverifieerd
1 juli 2026
Meer informatie
Termen gerelateerd aan deze studie
Aanvullende relevante MeSH-voorwaarden
Andere studie-ID-nummers
- 351-202
- 2024-511656-41-00 (Ctis)
Informatie over medicijnen en apparaten, studiedocumenten
Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel
Nee
Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct
Nee
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