NCT06907875 · Epicrispr Biotechnologies, Inc.
A First-in-human Study of EPI-321 in Facioscapulohumeral Muscular Dystrophy
(FSHD)
What this study is about
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.
View original scientific description
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.
Interventions
BIOLOGICAL
EPI-321
EPI-321 IV Infusion
Primary outcome measures
Frequency of AEs and EPI-321 Related Adverse Reactions and Serious Adverse Reactions
Time frame: Baseline to up to 5 years.
All AEs, regardless of assessed relatedness to EPI-321, will be collected from the time of informed consent signature until the end of study participation. The Investigator is responsible for assessing the severity of an AE according to the NCI-CTCAE version 5.0.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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
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 concurrent or past medical conditions could jeopardize the safety of the participant
Where
- Los Angeles, California
- Atlanta, Georgia
- Baltimore, Maryland
- Worcester, Massachusetts
- Salt Lake City, Utah
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Apr 22, 2026 · Source of record for eligibility and locations