RecruitingPhase 1Phase 2NCT06907875

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

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

Sponsor

Epicrispr Biotechnologies, Inc.

Enrollment

12 participants

Start Date

May 8, 2025

Study Type

INTERVENTIONAL

Conditions

facioscapulohumeral muscular dystrophy

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

Min Age: 18 YearsMax Age: 75 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a biological treatment called EPI-321 for people with facioscapulohumeral muscular dystrophy. The study is currently recruiting participants at 7 locations.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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