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Experimental gene 'Silencer' therapy enters human testing for rare muscle disease

NCT ID NCT06907875

Disease control Sponsor: Epicrispr Biotechnologies, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Aug 20, 2026 · Updated 4 times

Summary

This early-phase trial is testing EPI-321, a one-time gene therapy designed to silence the faulty gene that causes facioscapulohumeral muscular dystrophy (FSHD). The study will enroll 12 adults with FSHD Type 1 to see if the treatment is safe and tolerable, and whether it shows any signs of working. Participants receive a single IV dose and are monitored for about 5 years.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
EPI-321 (a gene therapy that aims to silence the faulty gene causing FSHD)
What this could lead to
If it works, this could point toward a treatment that stops or slows muscle damage in FSHD, a rare muscular dystrophy.
What could go wrong
This is a very early, small trial (12 people) focused on safety, not proof of effectiveness. The therapy is new and may not work or could cause side effects.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 12 people

The number the study aims to enrol. It can still change while the study runs.

Started

May 2025

Expected to finish

Apr 2032

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 75 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

facioscapulohumeral muscular dystrophy facioscapulohumeral muscular dystrophy 1 muscular dystrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Facioscapulohumeral muscular dystrophy

Contacts and locations

Locations

  • David Geffen School of Medicine at University of California, Los Angeles

    Los Angeles, California, 90095, United States

  • Kennedy Krieger Institute, Center for Genetic Muscle Disorders

    Baltimore, Maryland, 21205, United States

  • Pacific Clinical Research Network

    Auckland, New Zealand, 0622, New Zealand

  • Rare Disease Research

    Atlanta, Georgia, 303329, United States

  • Royal Alfred Hospital

    Sydney, New South Wales, 2050, Australia

  • University of Massachusetts Chan Medical School

    Worcester, Massachusetts, 01605, United States

  • Utah Program for Inherited Neuromuscular Disorders - University of Utah

    Salt Lake City, Utah, 84112, United States

More trials for these conditions

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