A Trial to Assess Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of SFL-0821 in Adults With FSHD
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)
- Facioscapulohumeral Muscular Dystrophy (FSHD)
At a glance
- Phase
- Phase 1
- Study type
- Interventional
- Sponsor
- Soufflé Therapeutics, Inc.
- Enrolment target
- 90
- Started
- September 2026
- Main results due
- March 2028
- Study sites
- 6
- Registry updated
- 29 September 2026
Can you take part?
- Ages 18 years to 65 years.
- Open to any sex.
- You need the condition being studied — healthy volunteers are not accepted.
These are the headline rules only. Every study has a longer list, and whether you are eligible is decided by the research team at the site — never by this page.
Read the full eligibility 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.
What this study is about
In the sponsor’s own words, from the registry.
The purpose of this study is to evaluate the safety, tolerability, and preliminary efficacy of SFL-0821 in adult patients with Facioscapulohumeral Muscular Dystrophy (FSHD)
Study sites(6)
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
Mater Hospital Brisbane
South Brisbane, Queensland, Australia
Recruiting
The Alfred Hospital
Melbourne, Victoria, Australia
Not yet recruiting
Pacific Clinic Research Network
Auckland, Auckland, New Zealand
Not yet recruiting
Showing 6 of 6 sites. 1 of the 6 sites on this study is recruiting right now — a site can stop enrolling while the study as a whole is still open.
Source: ClinicalTrials.gov record NCT07798609. Trialion does not run this study, is not paid to refer anyone to it, and cannot enrol you. Eligibility is always decided by the research team at the site.
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