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

Sex: All

Ages: 18 Years – 75 Years

Healthy volunteers: No

Phase: PHASE1, PHASE2

Enrollment: 12

Sponsor: Epicrispr Biotechnologies, Inc.

Location: David Geffen School of Medicine at University of California, Los Angeles Los Angeles California

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 Criteria

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

Interested in this study? View the official listing for contact and enrollment details.

View on ClinicalTrials.gov

Source: ClinicalTrials.gov (NCT06907875). StuddyBuddy aggregates publicly available trial information.