Calendario›SRPT›SRP-1001 for Injection

SRP-1001 for Injection

Study of SRP-1001 in Adult and Adolescent Participants With Facioscapulohumeral Muscular Dystrophy Type 1

Ensayo de Sarepta Therapeutics, Inc. en Muscular Dystrophy, Facioscapulohumeral (del registro, en inglés).

Fase

Fase 1/2

Estado

Reclutando pacientes

Participantes

60

previstos

Centros

16

Fin del objetivo primario

dic 2028

estimado por el promotor

Qué significa cada fase y cada estado.

Estudio de intervención, aleatorizado, cuádruple ciego. Comenzó en jun 2024.

Qué mide

Number of Participants With Treatment-Emergent Adverse Events Over Time Through End of Study (Part 1: Up to Day 90; Part 2: Up to Day 360) (del registro, en inglés)

Cómo lo describe el promotor

En el documentoEn inglés, del registroThe purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of SRP-1001 in participants with facioscapulohumeral muscular dystrophy Type 1 (FSHD1). In Part 1 of the study, participants will receive one dose of SRP-1001 or placebo. In Part 2 of the study, participants will receive 4 doses of SRP-1001 or placebo. Participants who complete Part 1 will have the option to re-screen and re-randomize into Part 2. All participants will undergo pre- and post-dose magnetic imaging resonance (MRI)-guided muscle biopsies (a total of 2 biopsies). Participants who complete Part 1 and enroll in Part 2 will be required to undergo an additional screening biopsy. Participants completing Part 1 or Part 2 may have the option to continue to receive drug in an open-label extension study or may be eligible to participate in later-stage clinical studies.

Más ensayos de SRPT

Todos los de SRPT, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT06131983), actualizada en ago 2026.