Facioscapulohumeral Muscular Dystrophy (FSHD)Phase 1/2NCT07798609
A Trial to Assess Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of SFL-0821 in Adults With FSHD
A Phase 1/2 study testing SFL-0821 for injection in people with Facioscapulohumeral Muscular Dystrophy (FSHD), run by Soufflé Therapeutics, Inc. at 6 sites in 2 countries.
Trial summary
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) Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Facioscapulohumeral Muscular Dystrophy (FSHD)
- What is being tested?
- SFL-0821 for injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 90 participants
- Where
- 6 sitesin 2 countries
- Study dates
- Sep 2026to Apr 2028
- Phase
- Phase 1/2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
6 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
6 sites in 2 countries
Australia5 sites, 5 recruiting
- Not yet recruiting
Wesley Research Institute
Brisbane, Queensland, 4066
Robert Henderson, MBBS (Hons), PhD, FRACP, robert.henderson@health.qld.gov.au
- Not yet recruiting
Concord Repatriation General Hospital
Concord, New South Wales, 2139
Katrina Morris, MBBS (Hons), PhD, FRACP, katrina.morris1@health.nsw.gov.au
- Not yet recruiting
Liverpool Hospital
Liverpool, New South Wales, 2170
Suzanne Hodgkinson, MBBS, PhD, FRACP, s.hodgkinson@unsw.edu.au
- Recruiting
The Alfred Hospital
Melbourne, Victoria, 3004
Edrich Rodrigues, MBBS, PhD, E.Rodrigues@alfred.org.au
- Recruiting
Mater Hospital Brisbane
South Brisbane, Queensland, 4101
Cullen O'Gorman, M.B.B.S., Ph.D., Cullen.OGorman@health.qld.gov.au
New Zealand1 site, 1 recruiting
- Recruiting
Pacific Clinic Research Network
Auckland, Auckland, 0622
Miriam Rodrigues, miriam.rodrigues@tewhatuora.govt.nz
About this study
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)
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)
- Being tested
- SFL-0821 for injection, Placebo
- Medical subject headings
- Muscular Dystrophy, Facioscapulohumeral
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SFL-0821 for injectionDrug
single or multiple doses of SFL-0821 by intravenous (IV) infusion
PlaceboDrug
calculated volume to match active treatment by IV infusion
| Study group | Role | What they receive |
|---|---|---|
| SFL-0821 | Experimental | SFL-0821 for Injection Receives: Drug: SFL-0821 for injection |
| Placebo | Placebo comparator | Placebo for Injection Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with treatment-emergent adverse events, serious adverse events, and any other dose-limiting toxicities as assessed by CTCAE v6.0.
Measured: Part A: Up to Week 24; Part B: Up to Week 48
Secondary outcomes (12)
Pharmacokinetics of SFL-0821: Maximum Observed Plasma Concentration (Cmax)
Measured: Part A and B: through 24 hours post dose (all cohorts)
Pharmacokinetics of SFL-0821: Elimination half-life (T1/2 [h])
Measured: Part A and B: through 24 hours post dose (all cohorts)
Pharmacokinetics of SFL-0821: Area under the concentration-time curve (AUC)
Measured: Part A and B: through 24 hours post dose (all cohorts)
DUX4-regulated gene expression in skeletal muscle
Measured: Part A: Up to Week 24; Part B: Up to Week 48
Incidence of anti-drug antibodies (ADAs), and if positive, of neutralizing ADAs
Measured: 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
Measured: 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).
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Part B: Up to Week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Sep 2026
- Study startSep 2026 (estimated)
- Primary completionMar 2028 (estimated)
- Study completionApr 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Facioscapulohumeral Muscular Dystrophy (FSHD)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 90 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Soufflé Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07798609
- Sponsor's protocol ID
- SFL0821-101
- Status last verified
- Oct 2026
- First submitted
- 17 Aug 2026
- Last updated
- 23 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No