Status:
RECRUITING
A First-in-human Study of EPI-321 in Facioscapulohumeral Muscular Dystrophy
Lead Sponsor:
Epicrispr Biotechnologies, Inc.
Conditions:
Facioscapulohumeral Muscular Dystrophy
Eligibility:
All Genders
18-75 years
Phase:
PHASE1
PHASE2
Brief 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 facioscapulo...
Detailed Description
EPI-321 is an investigational drug product comprising a recombinant adeno-associated viral vector, serotype rh74 (AAVrh74), for the delivery of genetic material encoding an epigenetic editor designed ...
Eligibility Criteria
Inclusion
- 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
Exclusion
- 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
Key Trial Info
Start Date :
May 8 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
April 30 2032
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT06907875
Start Date
May 8 2025
End Date
April 30 2032
Last Update
December 16 2025
Active Locations (6)
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1
David Geffen School of Medicine at University of California, Los Angeles
Los Angeles, California, United States, 90095
2
Rare Disease Research
Atlanta, Georgia, United States, 303329
3
University of Massachusetts Chan Medical School
Worcester, Massachusetts, United States, 01605
4
Utah Program for Inherited Neuromuscular Disorders - University of Utah
Salt Lake City, Utah, United States, 84112