Active, Not Recruiting
A Phase 12 Open-label Dose-escalation Study to Evaluate the Safety, Tolerability, and Biological Activity of EPI-321, an AAVrh74-delivered Epigenetic Editing Therapy in Adult FSHD Patients
Led by Epicrispr Biotechnologies, Inc. · Updated on 2026-07-20
12
Participants Needed
7
Research Sites
251 weeks
Total Duration
AI-Summary
What this Trial Is About
The goal of this clinical trial is to learn how safe and tolerable EPI-321 is and whether there may be early signs it is working in male or female adult (18 to 75 years) participants with facioscapulohumeral muscular dystrophy (FSHD) Type 1 condition. The main questions it aims to answer are: How safe is EPI-321 and how well can people handle it over time? How does EPI-321 interact with its target and does it show early signs of working? Participants will receive a single dose of EPI-321 through a vein while being closely watched in a hospital and visit the clinic regularly for tests and checkups for about 5 years after getting EPI-321.
CONDITIONS
Brief Title
A First-in-human Study of EPI-321 in Facioscapulohumeral Muscular Dystrophy
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