ClinTrial Refer

Trial Acronym
FSHD

Registration Number
NCT06907875

Scientific 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

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 check-ups for about 5 years after getting EPI-321.

- 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

- 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

Trial Location Name

Address

Status

Concord Repatriation General Hospital (Cell & Molecular)

Concord Repatriation General Hospital, Hospital Rd, Concord NSW, Australia

Recruiting

Primary Contact : Aimei Lee, SLHD-RPACMTTrials@health.nsw.gov.au , 02 9515 4860

Royal Prince Alfred Hospital (Cell & Molecular Therapies)

Royal Prince Alfred Hospital, Missenden Rd, Sydney NSW 2050, Australia

Closed

Primary Contact : Aimei Lee, SLHD-RPACMTTrials@health.nsw.gov.au , 02 9515 4860

Epicrispr Biotechnologies, Inc.

Website URL

Link Name

Link URL

ANZCTR

http://www.anzctr.org.au/TrialSearch.aspx?searchTxt=NCT06907875

Clinicaltrials.gov

https://clinicaltrials.gov/ct2/show/NCT06907875

Secondary IDs
EPI-321-02

Study Type
Interventional

Intervention Code
Treatment - Other

Phase
Phase 1/Phase 2

Minimum Age
18

Maximum Age
100

Date Registered
26/03/2025

Date Closed
N/A

Status
Open

Enrolment Type
By Specialist and/or Surgeon Referral

Mutation Status and Biomarker
Any or not stated

Is it a Cohort Trial?
No

Is this a Tele-Trial?
No

Can healthy volunteer participate?
No

Discipline

Health Condition

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