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Can an experimental infusion slow muscle loss in FSHD?

NCT ID NCT07798609

Disease control Sponsor: Soufflé Therapeutics, 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 This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Sep 01, 2026 · Last updated Sep 04, 2026 · Updated 3 times

Summary

This trial tests an experimental drug called SFL-0821 in adults with facioscapulohumeral muscular dystrophy (FSHD), a genetic condition that causes progressive muscle weakness. Researchers give the drug by intravenous infusion, either once or multiple times, and compare it against a placebo. The main goals are to check the drug's safety and tolerability, and to look for early signs that it might help. Participants must have a confirmed FSHD diagnosis, be able to walk a short distance, and have a leg muscle available for biopsy.

What this could mean

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

Active substance
SFL-0821, an experimental drug given by intravenous infusion
What this could lead to
If SFL-0821 proves safe and shows promise, it could become a first treatment to slow or stop muscle loss in FSHD, a disease with no approved therapies.
What could go wrong
This is an early-phase trial with a small number of participants, so the drug may not work or may cause side effects. The main goal is safety, not proof of benefit.

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 90 people

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

Expected to start

Sep 2026

An estimate. Start dates often move.

Expected to finish

Apr 2028

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 65 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: * Genetically confirmed diagnosis of FSHD 1 or 2 * FSHD Clinical Severity Score ≥1.5 to ≤4 at screening (5-point Ricci scale) * Ambulatory and able to walk 10 meters between ≥5 and ≤14 seconds (only assistive devices allowed are single cane or splints for ankle-foot orthoses) * Must have eligible lower extremity muscle for biopsy as determined from MRI by a central reader Exclusion Criteria: * History of any illness or any clinical condition that, in the opinion of the Investigator, might confound the results of the study or pose an additional risk in administering investigational product to the participant. * History of biopsy of the same muscle within 30 days of the Screening biopsy or planning to undergo any non-study muscle biopsies over the duration of the study * Treatment with an oligonucleotide, gene therapy, or other experimental therapies for FSHD.

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Conditions

The condition(s) this trial relates to.

facioscapulohumeral muscular dystrophy

As listed by the trial registrant

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

Facioscapulohumeral muscular dystrophy (FSHD)

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    6 sites in 2 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Concord Repatriation General Hospital

    Concord, New South Wales, 2139, Australia

  • Liverpool Hospital

    Liverpool, New South Wales, 2170, Australia

  • Mater Hospital Brisbane

    South Brisbane, Queensland, 4101, Australia

  • Pacific Clinic Research Network

    Auckland, Auckland, 0622, New Zealand

  • The Alfred Hospital

    Melbourne, Victoria, 3004, Australia

  • Wesley Research Institute

    Brisbane, Queensland, 4066, Australia

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