NCT06907875

Brief 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.

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
12

participants targeted

Target at below P25 for phase_1

Timeline
73mo left

Started May 2025

Longer than P75 for phase_1

Geographic Reach
3 countries

7 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Progress14%
May 2025Apr 2032

First Submitted

Initial submission to the registry

March 26, 2025

Completed
7 days until next milestone

First Posted

Study publicly available on registry

April 2, 2025

Completed
1 month until next milestone

Study Start

First participant enrolled

May 8, 2025

Completed
5.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 31, 2031

Expected
1.1 years until next milestone

Study Completion

Last participant's last visit for all outcomes

April 30, 2032

Last Updated

April 22, 2026

Status Verified

March 1, 2026

Enrollment Period

5.9 years

First QC Date

March 26, 2025

Last Update Submit

April 20, 2026

Conditions

Facioscapulohumeral Muscular Dystrophy

Keywords

Facioscapulohumeral Muscular DystrophyEPI-321Muscular Dystrophy

Outcome Measures

Primary Outcomes (1)

  • Frequency of AEs and EPI-321 Related Adverse Reactions and Serious Adverse Reactions

    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.

    Baseline to up to 5 years.

Secondary Outcomes (4)

  • Vector Copy Number

    Baseline, 3 and 12 months

  • EPI-321 Cargo Transcriptional Activity

    Baseline, 3 and 12 months

  • DUX4 Expression

    Baseline, 3 and 12 months

  • Methylation Status

    Baseline, 3 and 12 months

Study Arms (2)

EPI-321 Cohort 1 Single IV Dose

EXPERIMENTAL

Single IV infusion of a target dose of 2x10\^13 vg/kg

Biological: EPI-321

EPI-321 Cohort 2 Single IV Dose

EXPERIMENTAL

Single IV infusion of a target dose of 4x10\^13 vg/kg

Biological: EPI-321

Interventions

EPI-321BIOLOGICAL

EPI-321 IV Infusion

EPI-321 Cohort 1 Single IV DoseEPI-321 Cohort 2 Single IV Dose

Eligibility Criteria

Age18 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (7)

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

Los Angeles, California, 90095, United States

RECRUITING

Rare Disease Research

Atlanta, Georgia, 303329, United States

RECRUITING

Kennedy Krieger Institute, Center for Genetic Muscle Disorders

Baltimore, Maryland, 21205, United States

RECRUITING

University of Massachusetts Chan Medical School

Worcester, Massachusetts, 01605, United States

RECRUITING

Utah Program for Inherited Neuromuscular Disorders - University of Utah

Salt Lake City, Utah, 84112, United States

RECRUITING

Royal Alfred Hospital

Sydney, New South Wales, 2050, Australia

RECRUITING

Pacific Clinical Research Network

Auckland, 0622, New Zealand

RECRUITING

MeSH Terms

Conditions

Muscular Dystrophy, FacioscapulohumeralMuscular Dystrophies

Condition Hierarchy (Ancestors)

Muscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Weston Miller, M.D.

CONTACT

888-562-4123epic.clinicaltrial@epic-bio.com

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Model Details: Dose escalation with two dose levels
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 26, 2025

First Posted

April 2, 2025

Study Start

May 8, 2025

Primary Completion (Estimated)

March 31, 2031

Study Completion (Estimated)

April 30, 2032

Last Updated

April 22, 2026

Record last verified: 2026-03

Data Sharing

IPD Sharing
Will not share

Locations

AU(1)NZ(1)US(5)