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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 oppdatert av: 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.

Studieoversikt

Status

Påmelding etter invitasjon

Forhold

Intervensjon / Behandling

Detaljert beskrivelse

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

Intervensjonell

Registrering (Antatt)

18

Fase

  • Fase 2

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiesteder

  • Italia
    • Italy
      • Milan, Italy, Italia
        • Fondazione Serena ETS - Centro Clinico NeMO Milano
      • Rome, Italy, Italia
        • UOC Fase I - Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
  • Nederland
    • Netherlands
      • Leiden, Netherlands, Nederland, 2333 ZA
        • Leids Universitair Medisch Centrum
  • Spania
    • Spain
      • Seville, Spain, Spania, 41013
        • Hospital Viamed Santa Angela De la Cruz
  • Storbritannia
    • United Kingdom
      • London, United Kingdom, Storbritannia, WC1N 3JH
        • Great Ormond Street Hospital NHS Foundation Trust
  • Tyrkia (Türkiye)
    • Turkey
      • Istanbul, Turkey, Tyrkia (Türkiye)
        • Yeditepe University Kosuyolu Hospital

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

  • Barn
  • Voksen
  • Eldre voksen

Tar imot friske frivillige

Nei

Beskrivelse

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

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: N/A
  • Intervensjonsmodell: Enkeltgruppeoppdrag
  • Masking: Ingen (Open Label)

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: 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.
Legemiddel: BMN 351
Anti-sense Oligonucleotide BMN 351 will be administered intravenously

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
To assess the long-term safety and tolerability of BMN 351 in participants with DMD
Tidsramme: 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.
Through study completion, at least 1 year

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
To evaluate the effect of BMN 351 on physical function
Tidsramme: Change from baseline and subsequent 24-week incremental visits
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
Tidsramme: Change from baseline and subsequent 24-week incremental visits
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
Tidsramme: Change from baseline and subsequent 24-week incremental visits
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
To evaluate the effect of BMN 351 on physical function
Tidsramme: 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
Change from baseline and subsequent 24-week incremental visits

Andre resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
To evaluate the trough plasma concentration of BMN 351
Tidsramme: Baseline, Week 13, Week 25, Week 37, Week 49
Serial predose measurements of trough plasma PK
Baseline, Week 13, Week 25, Week 37, Week 49
To evaluate the immune response to BMN 351
Tidsramme: Change from baseline and subsequent 24-week incremental visits
Anti-BMN 351 antibodies and Anti-dystrophin antibodies
Change from baseline and subsequent 24-week incremental visits
To evaluate lung function longitudinally for participants ages 7 and above
Tidsramme: Change from baseline to subsequent 24-week incremental visits
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
Tidsramme: Change from baseline to subsequent 24-week incremental visits
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
Tidsramme: Change from baseline to subsequent 24-week incremental visits
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
Tidsramme: Change from baseline to subsequent 24-week incremental visits
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
Tidsramme: Change from baseline to subsequent 24-week incremental visits
Maximal Expiratory Pressure (MEP) for participants ages 7 and above
Change from baseline to subsequent 24-week incremental visits

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

BioMarin Pharmaceutical

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Faktiske)

20. april 2026

Primær fullføring (Antatt)

1. desember 2031

Studiet fullført (Antatt)

1. desember 2031

Datoer for studieregistrering

Først innsendt

17. april 2026

Først innsendt som oppfylte QC-kriteriene

1. mai 2026

Først lagt ut (Faktiske)

7. mai 2026

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

24. juli 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

23. juli 2026

Sist bekreftet

1. juli 2026

Mer informasjon

Begreper knyttet til denne studien

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • 351-202
  • 2024-511656-41-00 (Ctis)

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Nei

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

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