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

Part of paid clinical trials in Los Angeles, California.

Sponsor
Epicrispr Biotechnologies, Inc.
Study ID
NCT06907875
Phase
PHASE1/PHASE2
Status
Recruiting
Apply to this trial

Conditions

  • Facioscapulohumeral Muscular Dystrophy

Eligibility Criteria

Sex
ALL
Age
18 Years - 75 Years
Healthy Volunteers
Not accepted

Interventions

  • EPI-321 — BIOLOGICAL
    EPI-321 IV Infusion

Study Details

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.

Key Dates

First listed
Apr 2, 2025
Start date
May 8, 2025
Status verified
Mar 2026
Primary completion
Mar 31, 2031
Completion
Apr 30, 2032

Study Design

Enrollment
12 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: EPI-321 Cohort 1 Single IV Dose
    Single IV infusion of a target dose of 2x10\^13 vg/kg
  • Experimental: EPI-321 Cohort 2 Single IV Dose
    Single IV infusion of a target dose of 4x10\^13 vg/kg

Primary Outcome Measure

Frequency of AEs and EPI-321 Related Adverse Reactions and Serious Adverse Reactions [ Time Frame: Baseline to up to 5 years. ]

Central Contacts

Locations (5)

FacilityCityStateZIPSite coordinators
David Geffen School of Medicine at University of California, Los AngelesLos AngelesCalifornia90095
Brenden Roberts
[email protected]
310-825-3264
Perry Shieh, MD, PhD (PRINCIPAL_INVESTIGATOR)
Rare Disease ResearchAtlantaGeorgia303329
Laura Sutton
[email protected]
470-600-9134
Renata Shih, MD (PRINCIPAL_INVESTIGATOR)
Kennedy Krieger Institute, Center for Genetic Muscle DisordersBaltimoreMaryland21205
Demaya Starkes
[email protected]
443-923-3832
Doris G Leung, MD, PhD (PRINCIPAL_INVESTIGATOR)
University of Massachusetts Chan Medical SchoolWorcesterMassachusetts01605
Catherine Douthwright
[email protected]
508-865-1524
[email protected]
Utah Program for Inherited Neuromuscular Disorders - University of UtahSalt Lake CityUtah84112
Ryan Kennington
[email protected]
801-587-0833
Russell Butterfield, MD, PhD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Los Angeles, CA

By research site
David Geffen School of Medicine at University of California, Los Angeles· Los Angeles, CARare Disease Research· Atlanta, GAKennedy Krieger Institute, Center for Genetic Muscle Disorders· Baltimore, MDUniversity of Massachusetts Chan Medical School· Worcester, MAUtah Program for Inherited Neuromuscular Disorders - University of Utah· Salt Lake City, UT

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