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NCT Number: NCT07798609

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

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Key information

Conditions

Facioscapulohumeral Muscular Dystrophy (FSHD) Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Muscular Dystrophy, Facioscapulohumeral Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Concord Repatriation General Hospital, Concord, New South Wales, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

SFL-0821 for injection

Drug

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

Placebo

Drug

calculated volume to match active treatment by IV infusion

Primary outcomes

  1. 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

Secondary outcomes

  1. Pharmacokinetics of SFL-0821: Maximum Observed Plasma Concentration (Cmax)

    Time frame: Part A and B: through 24 hours post dose (all cohorts)

  2. Pharmacokinetics of SFL-0821: Elimination half-life (T1/2 [h])

    Time frame: Part A and B: through 24 hours post dose (all cohorts)

  3. Pharmacokinetics of SFL-0821: Area under the concentration-time curve (AUC)

    Time frame: Part A and B: through 24 hours post dose (all cohorts)

  4. DUX4-regulated gene expression in skeletal muscle

    Time frame: Part A: Up to Week 24; Part B: Up to Week 48

  5. 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

  6. 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

  7. Change from Baseline to Week 48 in 10-Meter Walk-Run-Test (10MWRT)

    Time frame: Part B: Up to Week 48

    Assessment used to measure gait speed and functional mobility over a short distance (10 meters).

  8. Change from Baseline to Week 48 in Timed Up-and-Go (TUG) Test

    Time frame: Part B: Up to Week 48

    Assessment of functional mobility by measuring the time required to stand up from a chair, walk 3 meters, turn, return, and sit down.

  9. Change from Baseline to Week 48 in 4-Step Stair Climb Test

    Time frame: Part B: Up to Week 48

    Assessment of the time to ascend and descend 4 stair steps.

  10. Change from Baseline to Week 48 in Sit-to-Stand Test

    Time frame: Part B: Up to Week 48

    Assessment of the number of full stands completed in the allotted time.

Study contacts

Contact information is provided by the study sponsor or research team.

Soufflé Therapeutics, Inc.

CONTACT

[email protected]

1 617-458-7581

Sponsors and collaborators

Lead sponsor

Soufflé Therapeutics, Inc.

Industry

Registry information

Official study 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)

Important dates

Study start
2026
Primary completion
2028
Study completion
2028
First posted
Sep 1, 2026
Registry last updated
Sep 1, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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