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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06907875




Registration number
NCT06907875
Ethics application status
Date submitted
26/03/2025
Date registered
2/04/2025

Titles & IDs
Public title
A First-in-human Study of EPI-321 in Facioscapulohumeral Muscular Dystrophy
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
Secondary ID [1] 0 0
EPI-321-02
Universal Trial Number (UTN)
Trial acronym
FSHD
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Facioscapulohumeral Muscular Dystrophy 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Neurological 0 0 0 0
Other neurological disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - EPI-321

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


Treatment: Other: EPI-321
EPI-321 IV Infusion
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Frequency of AEs and EPI-321 Related Adverse Reactions and Serious Adverse Reactions
Timepoint [1] 0 0
Baseline to up to 5 years.
Secondary outcome [1] 0 0
Vector Copy Number
Timepoint [1] 0 0
Baseline, 3 and 12 months
Secondary outcome [2] 0 0
EPI-321 Cargo Transcriptional Activity
Timepoint [2] 0 0
Baseline, 3 and 12 months
Secondary outcome [3] 0 0
DUX4 Expression
Timepoint [3] 0 0
Baseline, 3 and 12 months
Secondary outcome [4] 0 0
Methylation Status
Timepoint [4] 0 0
Baseline, 3 and 12 months

Eligibility
Key inclusion criteria
* 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
Minimum age
18 Years
Maximum age
75 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* 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 body mass >90 kg
* Has a concurrent or past medical conditions could jeopardize the safety of the participant

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
8/05/2025
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
30/04/2032
Actual
Sample size
Target
9
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Georgia
Country [2] 0 0
New Zealand
State/province [2] 0 0
Auckland

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Epicrispr Biotechnologies, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Weston Miller, M.D.
Address 0 0
Country 0 0
Phone 0 0
888-562-4123
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT06907875