Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO) (FORZETTO)
20. mai 2026 oppdatert av: Dyne Therapeutics
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy, Safety, and Tolerability of DYNE-251 Administered Intravenously in Ambulatory Male Participants 4 to 18 Years of Age With Duchenne Muscular Dystrophy Amenable to Exon-51 Skipping
The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment rostudirsen (DYNE-251) administered intravenously (IV) every 4 weeks to ambulatory Duchenne muscular dystrophy (DMD) participants, 4 to 18 years of age, with dystrophin mutations amenable to exon 51 skipping.
Studieoversikt
Status
Rekruttering
Forhold
- Muskeldystrofier
- Muskeldystrofi, Duchenne
- Duchenne muskeldystrofi (DMD)
- Muskeldystrofi, Duchenne og Becker-typer
- Genetisk sykdom, X-koblet
- Genetisk sykdom, medfødt
- DMD
- Medfødte, arvelige og neonatale sykdommer og abnormiteter
- Muskeldystrofi (DMD)
- Muskeldystrofier (Duchenne, Becker, myotonisk dystrofi)
- Muskeldystrofi hos barn
- Muskeldystrofi, Duchenne Type
- Nevromuskulære sykdommer (NMD)
Intervensjon / Behandling
Detaljert beskrivelse
The study consists of three periods: a Screening period (up to 6 weeks), a Placebo-Controlled Period (72 weeks) and an open-label Long-Term Extension Period (96 weeks).
Studietype
Intervensjonell
Registrering (Antatt)
90
Fase
- Fase 3
Kontakter og plasseringer
Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.
Studiekontakt
- Navn: Dyne Clinical Trials
- Telefonnummer: +1-781-317-1919
- E-post: clinicaltrials@dyne-tx.com
Studiesteder
-
-
North Carolina
-
Hillsborough, North Carolina, Forente stater, 27278
- Rekruttering
- Rare Disease Research, LLC
-
Ta kontakt med:
- Hannah Nation
- Telefonnummer: 984-314-2252
- E-post: hannah.nation@rarediseaseresearch.com
-
-
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
Tar imot friske frivillige
Nei
Beskrivelse
Inclusion Criteria:
- Ambulatory male with confirmed diagnosis of DMD and with a mutation in the dystrophin gene characterized by exon deletion amenable to exon 51 skipping .
- Rise From Floor (RFF) time must be < 10 seconds for both screening assessments .
- Receiving a stable daily or weekend dosage of glucocorticoids for at least 24 weeks prior to randomization with the expectation of maintaining a stable dose during the Placebo-Controlled Period of the study (unless dose adjustment is required by weight change)
Exclusion Criteria:
- Receipt of ongoing immunosuppressive therapy (other than glucocorticoids) within 12 weeks prior to randomization
- Use of any pharmacologic treatment (other than glucocorticoids) that may have an effect on muscle strength or function within 12 weeks prior to randomization
- Any change in prophylaxis/treatment for congestive heart failure (CHF) within 12 weeks prior to randomization
- Receipt of eteplirsen within 1 week prior to randomization
- Receipt of alternative exon-skipping or dystrophin-modifying therapy or zeleciment rostudirsen within 24 weeks prior to randomization
- Receipt of givinostat within 12 weeks prior to randomization
- Receipt of gene therapy at any time
Note: Other inclusion or exclusion criteria may apply
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: Randomisert
- Intervensjonsmodell: Parallell tildeling
- Masking: Firemannsrom
Våpen og intervensjoner
Deltakergruppe / Arm |
Intervensjon / Behandling |
|---|---|
|
Eksperimentell: Placebo-Controlled Period: Zeleciment Rostudirsen (DYNE-251)
Participants will be randomized to receive zeleciment rostudirsen, once every 4 weeks (Q4W) for up to 72 weeks.
|
Administered by IV infusion
|
|
Placebo komparator: Placebo-Controlled Period: Placebo
Participants will be randomized to receive placebo, Q4W for up to 72 weeks.
|
Administreres ved IV infusjon
|
|
Eksperimentell: Open-Label Long-Term Extension Period: Zeleciment Rostudirsen (DYNE-251)
All participants who complete the Placebo-Controlled Period of the study will receive zeleciment rostudirsen administered Q4W for up to 96 weeks.
|
Administered by IV infusion
|
Hva måler studien?
Primære resultatmål
Resultatmål |
Tidsramme |
|---|---|
|
Rise From Floor (RFF) velocity
Tidsramme: Baseline, Week 73
|
Baseline, Week 73
|
Sekundære resultatmål
Resultatmål |
Tidsramme |
|---|---|
|
RFF (Rise From Floor) velocity
Tidsramme: Baseline, up to Week 169
|
Baseline, up to Week 169
|
|
Stride Velocity 95th Percentile (SV95C)
Tidsramme: Baseline, Week 73, up to Week 169
|
Baseline, Week 73, up to Week 169
|
|
North Star Ambulatory Assessment (NSAA) Total Score
Tidsramme: Baseline, Week 73, up to Week 169
|
Baseline, Week 73, up to Week 169
|
|
10-Meter Walk/Run (10MWR) Velocity
Tidsramme: Baseline, Week 73, up to Week 169
|
Baseline, Week 73, up to Week 169
|
|
4-Stair Climb (4SC) velocity
Tidsramme: Baseline, Week 73, up to Week 169
|
Baseline, Week 73, up to Week 169
|
|
Functional Composite score
Tidsramme: Baseline, Week 73, up to Week 169
|
Baseline, Week 73, up to Week 169
|
|
Forced Vital Capacity (FVC)
Tidsramme: Baseline, Week 73, up to Week 169
|
Baseline, Week 73, up to Week 169
|
|
Patient Global Impression of Severity (PGI-S)
Tidsramme: Baseline, Week 73, up to Week 169
|
Baseline, Week 73, up to Week 169
|
|
Outcome of Patient Global Impression of Change (PGI-C)
Tidsramme: Week 73, up to Week 169
|
Week 73, up to Week 169
|
|
Blood Creatine Kinase (CK) levels
Tidsramme: Baseline, Week 73, up to Week 169
|
Baseline, Week 73, up to Week 169
|
|
Incidence of participants With Treatment-Emergent Adverse Events (TEAEs)
Tidsramme: Through study completion, up to Week 173
|
Through study completion, up to Week 173
|
|
Maximum Observed Plasma Drug Concentration of DYNE-251 (Cmax)
Tidsramme: Through study completion, up to Week 169
|
Through study completion, up to Week 169
|
|
Time to Maximum Observed Plasma Drug Concentration of DYNE-251 (tmax)
Tidsramme: Through study completion, up to Week 169
|
Through study completion, up to Week 169
|
|
Area Under the Plasma Drug Concentration-Time Curve From Time 0 to the Last Quantifiable Concentration of DYNE-251 in Plasma (AUC-tlast)
Tidsramme: Through study completion, up to Week 169
|
Through study completion, up to Week 169
|
|
Area Under the Plasma Drug Concentration Versus Time Curve From Time 0 (Dosing) Extrapolated to Time Infinity of DYNE-251 (AUC∞)
Tidsramme: Through study completion, up to Week 169
|
Through study completion, up to Week 169
|
|
Apparent Terminal Phase Elimination Rate Constant of DYNE-251 in Plasma (λz)
Tidsramme: Through study completion, up to Week 169
|
Through study completion, up to Week 169
|
|
Apparent Terminal Elimination Half-Life of DYNE-251 in Plasma (t½)
Tidsramme: Through study completion, up to Week 169
|
Through study completion, up to Week 169
|
|
Total Body Clearance (CL) of DYNE-251
Tidsramme: Through study completion, up to Week 169
|
Through study completion, up to Week 169
|
|
Volume of Distribution at the Terminal Phase of DYNE-251 in Plasma (Vz)
Tidsramme: Through study completion, up to Week 169
|
Through study completion, up to Week 169
|
|
Volume of Distribution at Steady State of DYNE-251 in Plasma (Vss)
Tidsramme: Through study completion, up to Week 169
|
Through study completion, up to Week 169
|
|
Incidence of Participants With Antidrug Antibodies (ADAs)
Tidsramme: Through study completion, up to Week 169
|
Through study completion, up to Week 169
|
Samarbeidspartnere og etterforskere
Det er her du vil finne personer og organisasjoner som er involvert i denne studien.
Sponsor
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 (Antatt)
1. juni 2026
Primær fullføring (Antatt)
1. desember 2030
Studiet fullført (Antatt)
1. oktober 2032
Datoer for studieregistrering
Først innsendt
20. mai 2026
Først innsendt som oppfylte QC-kriteriene
20. mai 2026
Først lagt ut (Faktiske)
27. mai 2026
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
27. mai 2026
Siste oppdatering sendt inn som oppfylte QC-kriteriene
20. mai 2026
Sist bekreftet
1. mai 2026
Mer informasjon
Begreper knyttet til denne studien
Nøkkelord
Ytterligere relevante MeSH-vilkår
- Muskel- og skjelettsykdommer
- Sykdommer i nervesystemet
- Muskelsykdommer
- Nevrodegenerative sykdommer
- Heredodegenerative lidelser, nervesystemet
- Muskellidelser, atrofisk
- Myotoniske lidelser
- Genetiske sykdommer, medfødte
- Muskeldystrofier
- Myotonisk dystrofi
- Muskeldystrofi, Duchenne
- Genetiske sykdommer, X-linked
- Nevromuskulære sykdommer
- Medfødte, arvelige og neonatale sykdommer og abnormiteter
Andre studie-ID-numre
- DYNE251-DMD-301
- 2025-524096-23-00 (Ctis)
Plan for individuelle deltakerdata (IPD)
Planlegger du å dele individuelle deltakerdata (IPD)?
NEI
Legemiddel- og utstyrsinformasjon, studiedokumenter
Studerer et amerikansk FDA-regulert medikamentprodukt
Ja
Studerer et amerikansk FDA-regulert enhetsprodukt
Nei
Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .