Calendario›SRPT›A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, and LGMD2A/R1 ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice
A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, and LGMD2A/R1 ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice
Ensayo de Sarepta Therapeutics, Inc. en Limb-girdle Muscular Dystrophy (del registro, en inglés).
- Fase
- no aplica
- Estado
- En marcha, sin reclutar
- Participantes
- 205
- Centros
- 26
- Fin del objetivo primario
- sep 2030
previstos
estimado por el promotor
Qué significa cada fase y cada estado.
Estudio observacional. Comenzó en abr 2021.
Qué mide
NSAD Total Score (Baseline up to Month 60) (del registro, en inglés)
Cómo lo describe el promotor
En el documentoEn inglés, del registroThis study will follow participants who are screened and confirmed with a genetic diagnosis of Limb-girdle muscular dystrophy type 2E (LGMD2E/R4), Limb-girdle muscular dystrophy type 2D (LGMD2D/R3), Limb-girdle muscular dystrophy type 2C (LGMD2C/R5), or Limb-girdle muscular dystrophy type 2A (LGMD2A/R1). These enrolled participants will be followed to evaluate mobility and pulmonary function for up to 5 years after enrollment for participants with LGMD2C/R5, LGMD2D/R3, and LGMD2E/R4 with a North Star Assessment for Dysferlinopathy (NSAD) ≥ 25 at Baseline, up to 3 years for participants with LGMD2C/R5, LGMD2D/R3, and LGMD2E/R4 with a NSAD \< 25 at Baseline, and up to 3 years for participants with LGMD2A/R1. Additional participant data will be collected from the time the individual began experiencing LGMD symptoms to the present.
Más ensayos de SRPT
| Ensayo | Fase | Estado | Lectura de resultados |
|---|---|---|---|
| SRP-9003 · EMERGENE | Fase 3 | En marcha, sin reclutar | mar 2025 (real) |
| Eteplirsen · MIS51ON | Fase 3 | En marcha, sin reclutar | oct 2026 (prevista) |
| SRP-1003 IV Infusion | Fase 1/2 | Reclutando pacientes | dic 2026 (prevista) |
| delandistrogene moxeparvovec · ENVISION | Fase 3 | En marcha, sin reclutar | may 2027 (prevista) |
| ELEVIDYS · ENHANCE | Fase 4 | Aún sin reclutar | ago 2027 (prevista) |
| SRP-1005 | Fase 1 | Reclutando pacientes | nov 2027 (prevista) |
Todos los de SRPT, en su ficha.
Ficha completa en ClinicalTrials.gov (NCT04475926), actualizada en nov 2025.