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)
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
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.
Sequential
SFL-0821 for injection
Drugsingle or multiple doses of SFL-0821 by intravenous (IV) infusion
Placebo
Drugcalculated 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.
Pharmacokinetics of SFL-0821: Maximum Observed Plasma Concentration (Cmax)
Pharmacokinetics of SFL-0821: Elimination half-life (T1/2 [h])
Pharmacokinetics of SFL-0821: Area under the concentration-time curve (AUC)
DUX4-regulated gene expression in skeletal muscle
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