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Recruiting Phase 1/2

A Trial to Assess Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of SFL-0821 in Adults With FSHD

NCT07798609 · tracked via the Priya Life Science Australia tracker
Sponsor
Soufflé Therapeutics, Inc
Phase
Phase 1/2
Started
2026-09
Last updated
2026-10-09

Condition(s) studied

Facioscapulohumeral Muscular Dystrophy (FSHD)

Investigational drug(s) / intervention(s)

SFL-0821 for injectionPlacebo

SFL-0821 for injection: single or multiple doses of SFL-0821 by intravenous (IV) infusion

Placebo: calculated volume to match active treatment by IV infusion

Study 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)

Eligibility

Sex
ALL
Min age
18 Years
Max age
65 Years
Healthy volunteers
No
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.

Primary outcome measure(s)

  • Number of participants with treatment-emergent adverse events, serious adverse events, and any other dose-limiting toxicities as assessed by CTCAE v6.0. — Part A: Up to Week 24; Part B: Up to Week 48

Trial sites (6)

FacilityCityRegionStatus
Concord Repatriation General Hospital Concord New South Wales Not Yet Recruiting
Liverpool Hospital Liverpool New South Wales Not Yet Recruiting
Wesley Research Institute Brisbane Queensland Not Yet Recruiting
Mater Hospital Brisbane South Brisbane Queensland Recruiting
The Alfred Hospital Melbourne Victoria Recruiting
Pacific Clinic Research Network Auckland Auckland Not Yet Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07798609 on ClinicalTrials.gov ↗ ← All trials in Australia