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drugset / Trial / NCT05514249

Treatment of a Single Patient With CRD-TMH-001

NCT05514249 ↗

Phase 1 Unknown 1 enrolled Cure Rare Disease, Inc University of Massachusetts, Worcester · collab
NaSingle-groupOpen-labelTreatment

Summary

The study is a single patient study intended to understand the effects of a gene-editing therapeutic to treat a rare mutation of Duchenne muscular dystrophy.

Timeline

Start
2022-08-31
Primary completion
2023-09
Completion
2023-09

Drugs

EvaluationDrugModalityDoseRoute
Subject CRD-TMH-001 Unknown — Intravenous

Indications

Duchenne muscular dystrophy