RECRUITING

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

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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

Quick Facts

Study Start:2025-05-08
Study Completion:2032-04-30
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06907875

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years to 75 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Able and willing to provide informed consent
  2. * Male or female 18 to 75 years of age
  3. * Clinical diagnosis of FSHD with genetic Type 1
  4. * FSHD Ricci clinical severity score 2 to 4 (on 5-point scale)
  5. * Has adequate liver function
  6. * Has adequate kidney function
  1. * Has an anti-AAVrh74 total binding antibody titer \> 1:400
  2. * Requires a walker or wheelchair for ambulation
  3. * Pregnant and/or breastfeeding at baseline or is planning to become pregnant during the first 12 months following EPI-321 administration
  4. * Has FSHD Type 2
  5. * Has a body mass \>90 kg
  6. * Has a concurrent or past medical conditions could jeopardize the safety of the participant

Contacts and Locations

Study Contact

Weston Miller, M.D.
CONTACT
888-562-4123
epic.clinicaltrial@epic-bio.com

Study Locations (Sites)

Rare Disease Research
Atlanta, Georgia, 303329
United States

Collaborators and Investigators

Sponsor: Epicrispr Biotechnologies, Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-05-08
Study Completion Date2032-04-30

Study Record Updates

Study Start Date2025-05-08
Study Completion Date2032-04-30

Terms related to this study

Keywords Provided by Researchers

  • Facioscapulohumeral Muscular Dystrophy
  • EPI-321
  • Muscular Dystrophy

Additional Relevant MeSH Terms

  • Facioscapulohumeral Muscular Dystrophy