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A First-in-human Study of EPI-321 in Facioscapulohumeral Muscular Dystrophy
Sponsor: Epicrispr Biotechnologies, Inc.
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.
Official title: A Phase 1/2 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
Key Details
Gender
All
Age Range
18 Years - 75 Years
Study Type
INTERVENTIONAL
Enrollment
12
Start Date
2025-05-08
Completion Date
2032-04-30
Last Updated
2026-03-31
Healthy Volunteers
No
Conditions
Interventions
EPI-321
EPI-321 IV Infusion
Locations (7)
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