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AAV5-RPGR

Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR)

Ensayo de MeiraGTx Holdings plc en X-Linked Retinitis Pigmentosa (del registro, en inglés).

Fase

Fase 1/2

Estado

Completado

Participantes

49

previstos

Centros

5

Fin del objetivo primario

nov 2021

fecha real

Qué significa cada fase y cada estado.

Estudio de intervención, aleatorizado, abierto. Comenzó en jul 2017.

Qué mide

Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 9 Weeks Following Administration, at Least Possibly Related to the Advanced Therapy Investigational Medicinal Product (ATIMP), Not Surgery Alone. (del registro, en inglés) (9 semanas)

Cómo lo describe el promotor

En el documentoEn inglés, del registroPhase 1 of the study is a dose escalation of the subretinal administration of AAV5-hRKp.RPGR vector to assess the safety of this vector in participants with XLRP caused by mutations in RPGR. Participants enrolled in Phase 1 were assigned to a dose group based on when they enrolled (i.e., sequential assignment). Phase 2 of the study is a cohort expansion of the subretinal administration of AAV5-hRKp.RPGR vector to assess the safety and efficacy of this vector in participants with XLRP caused by mutations in RPGR. Participants enrolled in Phase 2 were randomized to immediate or deferred treatment.

Más ensayos de MGTX

Todos los de MGTX, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT03252847), actualizada en dic 2024.