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PM577a

A Study of the Safety and Efficacy of Prime Editing (PM577) in Participants With Wilson Disease (WD)

Ensayo de Prime Medicine, Inc. en Wilson Disease · Wilson's Disease · Wilsons Disease (del registro, en inglés).

Fase

Fase 1/2

Estado

Reclutando pacientes

Participantes

42

previstos

Centros

4

Fin del objetivo primario

nov 2028

estimado por el promotor

Qué significa cada fase y cada estado.

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

Lo que ha cambiado

Movimientos registrados desde que PivotalWire vigila este ensayo. Un retraso no se anuncia: se ve comparando, y esto es la comparación ya hecha.

Qué mide

Safety and tolerability of PM577a. Quantified by frequency and severity of treatment emergent adverse events (TEAEs). (Post-infusion through Week 48) (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, biological activity, and initial efficacy of PM577a, an investigational Prime Editing therapy, in adults and adolescents with Wilson disease (WD). Wilson disease is caused by changes (mutations) in the ATP7B gene that prevent the body from removing excess copper normally. PM577a is designed to precisely correct one of the most common disease-causing ATP7B mutations (p.H1069Q) in liver cells with the goal of restoring normal copper metabolism. This is the first study of PM577a in people. Participants will receive a single intravenous (IV) infusion of PM577a and will be monitored closely to evaluate safety, how the body responds to treatment, whether copper metabolism improves, and whether treatment may improve signs and symptoms of Wilson disease.

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Todos los de PRME, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT07748403), actualizada en sep 2026.