Facioscapulohumeral muscular dystrophy (FSHD) is characterized by clinical diversity, with FSHD1 being the most common form. It is associated with a toxic gain of function of the Double homeobox 4 (DUX4) gene, leading to muscle cell death and weakness. Despite the lack of approved treatments, recent studies highlight inflammation's role in early FSHD progression, triggered by inappropriate DUX4 expression. In understanding inflammation's pivotal role in FSHD, a study assessed serum cytokines in 100 adult FSHD1 patients. Out of the 20 cytokines examined, 10 showed significantly altered expression levels compared to healthy controls of similar age and sex. FSHD1 patients exhibited heightened levels of inflammatory cytokines and diminished anti-inflammatory cytokines, signaling chronic inflammation. Notably, Interleukin-6 (IL-6) emerged as a promising disease activity biomarker, displaying robust correlations with established clinical severity and functional scores. Given the pathological significance of inflammation and the correlation of IL-6 levels with disease severity, the ReInForce study will explore the satralizumab, an IL6-receptor (IL6-R) antagonist, for its efficacy in specifically reducing muscle and systemic inflammation. By antagonizing IL-6R downstream signaling, satralizumab holds promise in mitigating inflammation and potentially curtailing fibrofatty degeneration in FSHD.
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The study will evaluate the efficacy of satralizumab compared to placebo, measured by the change during the Double Blind (DB) period (week 0 to week 48) on whole body muscle MRI.
Timeframe: From baseline to week 48
The study will evaluate the efficacy of satralizumab compared to placebo, measured by the change during the Double Blind (DB) period (week 0 to week 48) on RICCI clinical severity scale score.
Timeframe: From baseline to week 48
The study will evaluate the efficacy of satralizumab compared to placebo, measured by the change during the Double Blind (DB) period (week 0 to week 48) on Reachable Work Space (RWS) results with and without weights.
Timeframe: From baseline to week 48
The study will evaluate the efficacy of satralizumab compared to placebo, measured by the change during the Double Blind (DB) period (week 0 to week 48) on muscle strength, determined by quantitative isometric dynamometry
Timeframe: From baseline to week 48
The study will evaluate the efficacy of satralizumab compared to placebo, measured by the change during the Double Blind (DB) period (week 0 to week 48) on FSHD-Composite Outcome Measure (FSHD-COM) total score, sub-scale scores and individual items.
Timeframe: From baseline to week 48
The study will evaluate the efficacy of satralizumab compared to placebo, measured by the change during the Double Blind (DB) period (week 0 to week 48) on FSHD-Rasch-built overall disability scale (FSHD-RODS) total score
Timeframe: From baseline to week 48
The study will evaluate the efficacy of satralizumab compared to placebo, measured by the proportion of participants with an improvement in the FSHD Patient Global Impression of Change scale (FSHD-PGIC) during the Double Blind period (week 0 to week 48)
Timeframe: From baseline to week 48
The study will evaluate the efficacy of satralizumab compared to placebo, measured by the proportion of participants with an improvement in FSHD Clinical Global Impressions of Change scale (FSHD-CGIC) during the Double Blind period (week 0 to week 48)
Timeframe: From baseline to week 48
The study will evaluate the efficacy of satralizumab compared to placebo, measured by the change during the Double Blind (DB) period (week 0 to week 48) on the number of falls reported.
Timeframe: From baseline to week 48
The study will evaluate the efficacy of satralizumab compared to placebo, measured by the change during the Double Blind (DB) period (week 0 to week 48) on Neuromuscular Disease Independence Scale-Upper Limb Module (NMDIS-ULM) total score
Timeframe: From baseline to week 48
The study will evaluate the efficacy of satralizumab compared to placebo, measured by the change during the Double Blind (DB) period (week 0 to week 48) on Neuromuscular Disease Independence Scale-Ambulatory (NMDIS-Amb) total score.
Timeframe: From baseline to week 48
The study will evaluate the efficacy of satralizumab compared to placebo, measured by the change during the Double Blind (DB) period (week 0 to week 48) on inflammation, and specifically on molecular biomarkers associated with inflammation.
Timeframe: From baseline to week 48
The study will evaluate the safety and tolerability of satralizumab compared to placebo, measured by the type, frequency, severity, seriousness, and relationship of Adverse Events to satralizumab during the Double Blind (DB) period (week 0 to week 48).
Timeframe: From baseline to week 48
The study will evaluate the safety and tolerability of satralizumab compared to placebo, measured by the incidence of Adverse events of special interest (AESIs) and selected AEs during the Double Blind (DB) period (week 0 to week 48).
Timeframe: From baseline to week 48
The study will evaluate the safety and tolerability of satralizumab compared to placebo, measured by the number of subjects discontinuing study drug due to an AE during the Double Blind (DB) period (week 0 to week 48).
Timeframe: From baseline to week 48
The study will evaluate safety and tolerability of satralizumab compared to placebo, measured by frequency of clinically significant changes from baseline in laboratory test, vital signs, and physical examination results during the Double Blind period
Timeframe: From baseline to week 48