Actively Recruiting
A First-in-human Study of EPI-321 in Facioscapulohumeral Muscular Dystrophy
Led by Epicrispr Biotechnologies, Inc. · Updated on 2026-04-22
12
Participants Needed
7
Research Sites
364 weeks
Total Duration
On this page
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
Official Title
A First-in-human Study of EPI-321 in Facioscapulohumeral Muscular Dystrophy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 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 will not qualify if you...
- 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
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 7 locations
1
David Geffen School of Medicine at University of California, Los Angeles
Los Angeles, California, United States, 90095
Actively Recruiting
2
Rare Disease Research
Atlanta, Georgia, United States, 303329
Actively Recruiting
3
Kennedy Krieger Institute, Center for Genetic Muscle Disorders
Baltimore, Maryland, United States, 21205
Actively Recruiting
4
University of Massachusetts Chan Medical School
Worcester, Massachusetts, United States, 01605
Actively Recruiting
5
Utah Program for Inherited Neuromuscular Disorders - University of Utah
Salt Lake City, Utah, United States, 84112
Actively Recruiting
6
Royal Alfred Hospital
Sydney, New South Wales, Australia, 2050
Actively Recruiting
7
Pacific Clinical Research Network
Auckland, New Zealand, 0622
Actively Recruiting
Research Team
W
Weston Miller, M.D.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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