A First-in-human Study of EPI-321 in Facioscapulohumeral Muscular Dystrophy
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18-75
SponsorEpicrispr Biotechnologies, Inc.
About this trial
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
Qualifiers
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)
Disqualifiers
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
Trial design
Treatments tested in this trial
- EPI-321
Treatment groups
12 Participants
are divided into 2 treatment groupsLocations
7Australia
Royal Alfred Hospital2050, SydneyNew South Wales, Australia
New Zealand
Pacific Clinical Research Network0622, Auckland New Zealand
United States
David Geffen School of Medicine at University of California, Los Angeles90095, Los AngelesCalifornia, United States
Rare Disease Research303329, AtlantaGeorgia, United States
Sponsors and collaborators
Epicrispr Biotechnologies, Inc.
Lead sponsor
Source ClinicalTrials.gov