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OPGx-RDH12 · RDH12

Safety and Tolerability of Subretinal OPGx-RDH12-1001 for LCA-Associated Inherited Retinal Degeneration (LCA-IRD)

Ensayo de Opus Genetics, Inc. en Leber Congenital Amaurosis · Leber Congenital Amaurosis (LCA) (del registro, en inglés).

Fase

Fase 1/2

Estado

Aún sin reclutar

Participantes

10

previstos

Centros

3

Fin del objetivo primario

dic 2032

estimado por el promotor

Qué significa cada fase y cada estado.

Estudio de intervención, de un solo grupo, abierto. Comenzó en sep 2026.

Qué mide

Number of dose-limiting toxicity (DLT) events at the proposed doses (del registro, en inglés) (5 años)

Cómo lo describe el promotor

En el documentoEn inglés, del registroThis study is an early-stage clinical trial (Phase 1b/2a) testing a gene therapy called OPGx-RDH12 for people with Leber Congenital Amaurosis (LCA) caused by mutations in the RDH12 gene, a rare genetic eye disease that leads to severe vision loss. The treatment is delivered as a one-time injection (300 µL) into the retina (subretinal space) of the worse-seeing eye, using a method similar to approved gene therapies like Luxturna. The study is designed to evaluate safety and effectiveness at two dose levels (1E11 and 3E11 viral genomes per eye) in small groups of 5 participants. Each group begins cautiously with 2 adults (age ≥18), treated at least one month apart, followed by FDA review before allowing adolescents (ages 12-17) to participate. An independent monitoring committee (IDMC) oversees safety throughout. After 3 adolescents are treated and followed for 3 months, the committee reviews all data to decide whether to move to a higher dose. However, if the lower dose (1E11 vg/eye) shows strong effectiveness in the first group, the study may expand by treating more adolescents at that same dose instead of increasing it further.

Más ensayos de IRD

Todos los de IRD, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT07681778), actualizada en jul 2026.