Clinical Trial

A First-in-human Study of EPI-321 in Facioscapulohumeral Muscular Dystrophy

Study acronym: FSHD
Active, Not Recruiting Phase 1/2
View on ClinicalTrials.gov →
Summary
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.
Protocol Amendment History 2 changes
critical Trial status changed: Recruiting → Active, Not Recruiting 2026-07-21
notable Primary completion moved earlier: 2031-03-31 -> 2027-07-07 2026-07-21
Trial Details
NCT Number NCT06907875
Lead Sponsor Epicrispr Biotechnologies, Inc.
Conditions Facioscapulohumeral Muscular Dystrophy
Enrollment 12 participants
Start Date 2025-05-08
Primary Completion 2027-07-07 (estimated)
Study Completion 2032-04-30 (estimated)
Updated on ClinicalTrials.gov 2026-07-20