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Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO) (FORZETTO)

20. maj 2026 opdateret af: 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.

Studieoversigt

Status

Rekruttering

Betingelser

Intervention / Behandling

Detaljeret 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).

Undersøgelsestype

Interventionel

Tilmelding (Anslået)

90

Fase

  • Fase 3

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiekontakt

Studiesteder

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

  • Barn
  • Voksen

Tager imod sunde frivillige

Ingen

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

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Behandling
  • Tildeling: Randomiseret
  • Interventionel model: Parallel tildeling
  • Maskning: Firedobbelt

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: 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.
Medicin: Zeleciment Rostudirsen (DYNE-251)
Administered by IV infusion
Placebo komparator: Placebo-Controlled Period: Placebo
Participants will be randomized to receive placebo, Q4W for up to 72 weeks.
Medicin: Placebo
Administreret ved IV infusion
Eksperimentel: 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.
Medicin: Zeleciment Rostudirsen (DYNE-251)
Administered by IV infusion

Hvad måler undersøgelsen?

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

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

Dyne Therapeutics

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Anslået)

1. juni 2026

Primær færdiggørelse (Anslået)

1. december 2030

Studieafslutning (Anslået)

1. oktober 2032

Datoer for studieregistrering

Først indsendt

20. maj 2026

Først indsendt, der opfyldte QC-kriterier

20. maj 2026

Først opslået (Faktiske)

27. maj 2026

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

27. maj 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

20. maj 2026

Sidst verificeret

1. maj 2026

Mere information

Begreber relateret til denne undersøgelse

Nøgleord

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • DYNE251-DMD-301
  • 2025-524096-23-00 (Ctis)

Plan for individuelle deltagerdata (IPD)

Planlægger du at dele individuelle deltagerdata (IPD)?

INGEN

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

Studerer et amerikansk FDA-reguleret lægemiddelprodukt

Ja

Studerer et amerikansk FDA-reguleret enhedsprodukt

Ingen

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