NCT06222827

Study to Evaluate the Efficacy and Safety of Satralizumab in FSHD1

A Bicentric, Randomized, Double Blind, Placebo-controlled Pilot Study to Evaluate the Efficacy and Safety of Satralizumab in FSHD1

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 46 (actual)
• Sponsor: Centre Hospitalier Universitaire de Nice · Academic / Other
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• Facioscapulohumeral Muscular Dystrophy 1

Interventions

Type	Name	Description
DRUG	Satralizumab Prefilled Syringe	Satralizumab is supplied as prefilled syringe with 1 millimeter of solution for subcutaneous injection corresponding to 120mg of satralizumab. During the first period (double blind period), satralizumab will be administered at weeks 0, 2, 4 and every 4 weeks until week 48. During the second period (open label period), satralizumab will be administered at weeks 48, 50, 52 and every 4 weeks until week 96.
DRUG	Placebo Comparator	Placebo prefilled syringe is identical in composition to satralizumab, but does not contain the satralizumab active agent. It is identical in appearance and packaging to satralizumab. During the first period (double blind period), placebo will be administered at weeks 0, 2, 4 and every 4 weeks until week 48. During the second period (open label period), satralizumab will be administered at weeks 48, 50, 52 and every 4 weeks until week 96.

Timeline

• Start date: 2024-01-24
• Primary completion: 2027-03-01
• Completion: 2027-03-01
• First posted: 2024-01-25
• Last updated: 2025-03-26

Locations

2 sites across 2 countries: Canada, France