Safety and early efficacy test of SFL-0821 in adults with FSHD
Official title: A Randomized, Double-Blind, Placebo-Controlled, Single and Multiple Ascending Dose Trial to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of SFL-0821 in Adults With Facioscapulohumeral Muscular Dystrophy (FSHD)
Run by Soufflé Therapeutics, Inc. · NCT07798609 · Data updated 2026-11-12
This study will test whether SFL-0821 is safe and tolerable and whether it shows early signs of benefit in adults with Facioscapulohumeral Muscular Dystrophy (FSHD).
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Ages | 18 Years to 65 Years |
| Sex | All |
| Enrollment | 90 (estimated) |
| Sponsor | Soufflé Therapeutics, Inc. Industry-sponsored |
| Locations | 6 sites (Concord, New South Wales and 5 other locations) |
| Status | Recruiting |
| Started | September 2026 |
| Main results expected | March 2028 |
| Study ends | April 2028 |
| Registry record updated | October 30, 2026 |
| Trial ID | NCT07798609 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Adults with genetically confirmed FSHD, mild-to-moderate disease severity, able to walk 10 meters with minimal assistance, and having a biopsy-eligible lower-extremity muscle.
✕Probably not a fit
Patients with more advanced disease, non-ambulatory status, or those not meeting biopsy eligibility may not benefit from the treatment.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This is a two-phase interventional trial evaluating SFL-0821 versus placebo in adults with FSHD. The study will assess safety, tolerability, pharmacokinetics and pharmacodynamics, with preliminary signals of efficacy. Participants must have a confirmed FSHD diagnosis, a specific baseline disease severity, ambulatory ability, and a biopsyable leg muscle selected by MRI. Exclusion criteria include conditions that may confound results or pose additional risk, recent muscle biopsies, or prior investigational FSHD therapies. The trial is led by Soufflé Therapeutics, Inc. and conducted at three sites in Australia.
Why it matters
Potential benefit: If successful, participants may experience improved muscle function or slowed disease progression related to SFL-0821 treatment.
How similar studies have gone: Previous studies in similar gene-targeted approaches for muscular dystrophy have shown safety signals and hints of efficacy, but this exact approach is exploratory in FSHD.
Who can join: the full rules
Show full inclusion / exclusion criteria
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.
Where it's running
-
Mater Hospital Brisbane
South Brisbane, Queensland, Australia Recruiting -
The Alfred Hospital
Melbourne, Victoria, Australia Recruiting -
Pacific Clinic Research Network
Auckland, Auckland, New Zealand Recruiting -
Concord Repatriation General Hospital
Concord, New South Wales, Australia Not yet recruiting -
Liverpool Hospital
Liverpool, New South Wales, Australia Not yet recruiting -
Wesley Research Institute
Brisbane, Queensland, Australia Not yet recruiting
Study contacts
- Study coordinator: Soufflé Therapeutics, Inc.
- Email: SFL0821clinicaltrials@souffletx.com
- Phone: 1 617-458-7581
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.