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

Running, not enrolling · Phase 1/Phase 2

Conditions studied: Facioscapulohumeral Muscular Dystrophy

In brief

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.

Key facts

Study ID

NCT06907875

Run by

Epicrispr Biotechnologies, Inc.

People needed

12

Starts

2025-05-08

Expected to finish

2032-04-30

Last updated by the study team

2026-07-20

Who can join

Age: 18 and older, up to 75. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Able and willing to provide informed consent
• Male or female 18 to 75 years of age
• Clinical diagnosis of FSHD with genetic Type 1
• FSHD Ricci clinical severity score 2 to 4 (on 5-point scale)
• Has adequate liver function
• Has adequate kidney function

You may not qualify if…

• Has an anti-AAVrh74 total binding antibody titer > 1:400
• Requires a walker or wheelchair for ambulation
• Pregnant and/or breastfeeding at baseline or is planning to become pregnant during the first 12 months following EPI-321 administration
• Has FSHD Type 2
• Has a concurrent or past medical conditions could jeopardize the safety of the participant

Where it is running

• David Geffen School of Medicine at University of California, Los Angeles — Los Angeles, California, United States
• Rare Disease Research — Atlanta, Georgia, United States
• Kennedy Krieger Institute, Center for Genetic Muscle Disorders — Baltimore, Maryland, United States
• University of Massachusetts Chan Medical School — Worcester, Massachusetts, United States
• Utah Program for Inherited Neuromuscular Disorders - University of Utah — Salt Lake City, Utah, United States
• Royal Alfred Hospital — Sydney, New South Wales, Australia
• Pacific Clinical Research Network — Auckland, New Zealand, New Zealand

Full record on ClinicalTrials.gov

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