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)
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
90
single or multiple doses of SFL-0821 by intravenous (IV) infusion
calculated volume to match active treatment by IV infusion
Concord Repatriation General Hospital
Concord, New South Wales, Australia
Liverpool Hospital
Liverpool, New South Wales, Australia
Wesley Research Institute
Brisbane, Queensland, Australia
Mater Hospital Brisbane
South Brisbane, Queensland, Australia
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
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The Alfred Hospital
Melbourne, Victoria, Australia
Pacific Clinic Research Network
Auckland, Auckland, New Zealand
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