Satralizumab for Muscular Dystrophy
(REINFORCE Trial)

Recruiting in Palo Alto (17 mi)

+1 other location

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Centre Hospitalier Universitaire de Nice

Must not be taking: IL-6 inhibitors

Disqualifiers: Diabetes, Cancer, Severe autoimmune, others

Prior Safety Data

Approved in 5 Jurisdictions

Trial Summary

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

The trial requires that if you are on medications or supplements that affect muscle function, you must be on a stable dose for at least 3 months before starting the study and continue on that stable dose throughout the study.

Research Team

Eligibility Criteria

This trial is for adults with FSHD1, a type of muscular dystrophy. Participants should have evidence of muscle weakness and meet specific clinical criteria. Those with other forms of muscular dystrophy or conditions that might interfere with the study are excluded.

Inclusion Criteria

Willing to maintain same level of exercise (frequency and intensity) during the study

For female patients of childbearing potential: use adequate contraception during the treatment period and/or until treatment discontinuation

Patient affiliated to a European social security system (Nice center only)

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Exclusion Criteria

Any known hypersensitivity to satralizumab or any of its components and/or history of severe allergic reaction to a biologic agent (e.g., shock, anaphylactic reactions)

I have not received any live vaccines in the last 6 weeks.

I do not have any major health issues that could interfere with the study.

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Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Double Blind Treatment

Participants receive either satralizumab or placebo to evaluate efficacy and safety

48 weeks

Regular visits for assessments

Open-label Extension

Participants may continue receiving satralizumab to further assess long-term effects

48 weeks

Regular visits for assessments

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

Satralizumab (Monoclonal Antibodies)

Trial OverviewThe study tests Satralizumab, an IL-6 receptor blocker thought to reduce inflammation in muscles affected by FSHD1. It's compared against a placebo in a randomized, double-blind setup to assess its effectiveness and safety.

Participant Groups

2Treatment groups

Experimental Treatment

Placebo Group

Group I: Group of patients who receive satralizumabExperimental Treatment1 Intervention

Group II: Group of patients who receive placeboPlacebo Group1 Intervention

Satralizumab is already approved in Canada, Japan, Switzerland for the following indications: