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

NCT06907875 · tracked via the Priya Life Science USA tracker

Sponsor

Epicrispr Biotechnologies, Inc.

Phase

Phase 1/2

Started

2025-05-08

Last updated

2026-08-19

Condition(s) studied

Facioscapulohumeral Muscular Dystrophy

Investigational drug(s) / intervention(s)

EPI-321

EPI-321: EPI-321 IV Infusion

Study 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.

Eligibility

Sex

ALL

Min age

18 Years

Max age

75 Years

Healthy volunteers

Inclusion Criteria: * 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 Criteria: * 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

Primary outcome measure(s)

Frequency of AEs and EPI-321 Related Adverse Reactions and Serious Adverse Reactions — Baseline to up to 5 years.
All AEs, regardless of assessed relatedness to EPI-321, will be collected from the time of informed consent signature until the end of study participation. The Investigator is responsible for assessing the severity of an AE according to the NCI-CTCAE version 5.0.

Trial sites (7)

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

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06907875 on ClinicalTrials.gov ↗ ← All trials in the USA