A Phase 1/2 interventional study of SFL-0821 for injection and Placebo in Facioscapulohumeral Muscular Dystrophy (FSHD), sponsored by Soufflé Therapeutics, Inc.. Recruiting at 6 sites in 2 countries. Open to participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2026-10-30.
Sponsored by Soufflé Therapeutics, Inc. · Phase 1/2, Interventional, and Treatment
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)
79 studies on the registry are indexed under Muscular Dystrophy, Facioscapulohumeral; 26 are open to participants now.
This study's planned enrollment of 90 is above the median of 48 across 49 interventional studies indexed under Muscular Dystrophy, Facioscapulohumeral.
Browse Muscular Dystrophy, Facioscapulohumeral studies →This is the only study on the registry with Soufflé Therapeutics, Inc. as lead sponsor.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
SFL-0821 for Injection
Drug: SFL-0821 for injection
Placebo for Injection
Drug: Placebo
single or multiple doses of SFL-0821 by intravenous (IV) infusion
calculated volume to match active treatment by IV infusion
Number of participants with treatment-emergent adverse events, serious adverse events, and any other dose-limiting toxicities as assessed by CTCAE v6.0.
Time frame: Part A: Up to Week 24; Part B: Up to Week 48
Pharmacokinetics of SFL-0821: Maximum Observed Plasma Concentration (Cmax)
Time frame: Part A and B: through 24 hours post dose (all cohorts)
Pharmacokinetics of SFL-0821: Elimination half-life (T1/2 [h])
Time frame: Part A and B: through 24 hours post dose (all cohorts)
Pharmacokinetics of SFL-0821: Area under the concentration-time curve (AUC)
Time frame: Part A and B: through 24 hours post dose (all cohorts)
DUX4-regulated gene expression in skeletal muscle
Time frame: Part A: Up to Week 24; Part B: Up to Week 48
Incidence of anti-drug antibodies (ADAs), and if positive, of neutralizing ADAs
Time frame: Part A: Up to Week 24; Part B: Up to Week 48
Change from Baseline in quantitative skeletal muscle magnetic resonance imaging (MRI) at the cohort specified timepoints after dosing
Time frame: Part B: Up to Week 48
Change from Baseline to Week 48 in 10-Meter Walk-Run-Test (10MWRT)
Assessment used to measure gait speed and functional mobility over a short distance (10 meters).
Time frame: Part B: Up to Week 48
Change from Baseline to Week 48 in Timed Up-and-Go (TUG) Test
Assessment of functional mobility by measuring the time required to stand up from a chair, walk 3 meters, turn, return, and sit down.
Time frame: Part B: Up to Week 48
Change from Baseline to Week 48 in 4-Step Stair Climb Test
Assessment of the time to ascend and descend 4 stair steps.
Time frame: Part B: Up to Week 48
Change from Baseline to Week 48 in Sit-to-Stand Test
Assessment of the number of full stands completed in the allotted time.
Time frame: Part B: Up to Week 48
Change from Baseline to Week 48 in Reachable Workspace (RWS)
Assessment of upper extremity function, including shoulder and proximal arm.
Time frame: Part B: Up to Week 48
Change from Baseline to Week 48 in quantitative muscle strength testing
Assessment of the skeletal muscle strength in the upper and lower limbs bilaterally.
Time frame: Part B: Up to Week 48
Plan to share: No
No publications or documents are linked to this record.
Start is estimated for Sep 2026; primary completion for Mar 2028, 18 months later.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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Muscular Dystrophy, Facioscapulohumeral →