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delandistrogene moxeparvovec

A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)

Ensayo de Sarepta Therapeutics, Inc. en Distrofia muscular de Duchenne.

Más ensayos de: Distrofia muscular de Duchenne.

Fase

Fase 1/2

Estado

Completado

Participantes

41

previstos

Centros

2

Fin del objetivo primario

dic 2020

fecha real

Qué significa cada fase y cada estado.

Estudio de intervención, aleatorizado, cuádruple ciego. Comenzó en dic 2018.

Qué mide

Change From Baseline at Week 12 in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression as Measured by Western Blot Adjusted by Muscle Content (Baseline, Week 12 (Part 1)) (del registro, en inglés)

Cómo lo describe el promotor

En el documentoEn inglés, del registroThe purpose of this study is to evaluate the safety and efficacy of exogenous gene transfer in DMD participants by measuring biological and clinical endpoints in three parts: two 48-week randomized, double-blinded, placebo-controlled periods (Part 1 and Part 2), and an open-label follow-up period (Part 3). Participants who are randomized to placebo in Part 1 will have the opportunity for treatment with delandistrogene moxeparvovec in Part 2. In order to provide a uniform approach to monitoring long-term safety and efficacy in participants who received SRP-9001 in a clinical trial, the Sponsor has amended Study Completion for this study to occur at Week 130. Therefore, participants have transitioned and will complete the remainder of the Part 3 follow up visits in a long-term extension study, SRP-9001-305 (NCT05967351).

Más ensayos de SRPT

Todos los de SRPT, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT03769116), actualizada en nov 2024.