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 groupsSponsors and collaborators
Source ClinicalTrials.gov