Assessing the effects of satralizumab in treating Duchenne Muscular Dystrophy

A Phase II Multicenter, Open-label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Satralizumab in Pediatric Patients With Duchenne Muscular Dystrophy (SHIELD DMD)

This study tests if a new medication called satralizumab can help young people with Duchenne Muscular Dystrophy who are already taking steroids by improving their muscle function and bone strength.

Quick facts

• Phase: PHASE2
• Study type: Interventional
• Enrollment: 50 (estimated)
• Ages: 8 Years to 17 Years
• Sex: Male
• Sponsor: Hoffmann-La Roche (industry)
• Drugs / interventions: satralizumab
• Locations: 15 sites (Atlanta, Georgia and 14 other locations)
• Trial ID: NCT06450639 on ClinicalTrials.gov

What this trial studies

This study evaluates the efficacy, safety, pharmacokinetics, and pharmacodynamics of satralizumab, a monoclonal antibody targeting the interleukin-6 receptor, in participants aged 8 to 18 with Duchenne Muscular Dystrophy (DMD) who are receiving corticosteroid therapy. Participants will be divided into two groups: those who are ambulatory with a history of fractures and those who are non-ambulatory or fracture naive. The treatment involves subcutaneous injections of satralizumab every four weeks, aiming to improve bone fragility and muscle function in these patients.

Who should consider this trial

Good fit: Ideal candidates are males aged 8 to 18 with a confirmed diagnosis of DMD, either ambulatory with a history of fractures or non-ambulatory.

Not a fit: Patients who are female or older than 18 years will not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could enhance bone health and muscle function in young patients with Duchenne Muscular Dystrophy.

How similar studies have performed: Other studies targeting interleukin-6 pathways have shown promise, suggesting potential for success in this approach.

Eligibility criteria

Key Inclusion Criteria:

* Signed Informed Consent Form and Signed Assent Form when appropriate
* Male at birth
* A definitive diagnosis of DMD prior to screening based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test
* Age ≥ 8 and \< 18 years at the time of signing Informed Consent Form
* Group 1 participants are required to meet the following criteria:

  - Ambulatory (defined as able to walk independently without assistive devices) with a prior history of fractures:
  1. Prior history of low-trauma fracture defined as: evidence of at least one prevalent vertebral compression fracture of Genant Grade 1 or 2 (or radiographic signs of VF) or history of at least one low-trauma long-bone fracture (upper or lower extremity) OR
  2. Non-ambulatory, characterized as being non-ambulatory for a minimum of 6 months with onset of non-ambulatory status defined as participant- or caregiver-reported age of continuous wheelchair use approximated to the nearest month, and an North Star Ambulatory Assessment (NSAA) walk score of "0" and inability to perform the 10-Meter Walk/Run (10 MWR) at the baseline visit, with or without fractures
* Group 2 participants are required to meet the following criteria:

  * Be fracture naïve, defined as: no history of prior low-trauma fractures before the baseline visit nor any radiological findings indicative of prevalent VF at the screening visit
  * Be ambulatory defined as able to walk independently without assistive devices
  * Age ≥ 8 to \< 12 years old at the time of screening
* Daily oral corticosteroids

Key Exclusion Criteria:

* Major surgery (e.g. spinal surgery) within 3 months prior to Baseline or planned surgery or procedure that would interfere with the conduct of the study for any time during this study
* Presence of any clinically significant illness
* Has serological evidence of current, chronic, or active human immunodeficiency virus (HIV), tuberculosis (TB), hepatitis C, or hepatitis B infection
* Has a symptomatic infection (e.g. upper respiratory tract infection, pneumonia, pyelonephritis, meningitis) within 4 weeks prior to baseline
* Body weight at screening \<20 or \> 100 kg
* Evidence of a severe vertebral fracture (VF) (defined as Grade 3), assessed by radiographic imaging at screening and quantified using the Genant semiquantitative method
* Treatment with prohibited therapies as defined by the protocol
* Has received a live or live attenuated virus vaccine within 6 weeks of the Baseline visit or expects to receive a vaccination during the first 3 months after Baseline.
* Has abnormal laboratory values considered clinically significant as defined by the protocol
* Any medical condition that might interfere with the evaluation of LS BMD, such as severe scoliosis or spinal fusion.
* Participant has previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect safety, make it unlikely that treatment and follow-up will be correctly completed or impair the assessment of study results, in the opinion of the investigator
* Participant has an allergy or hypersensitivity to the study medication or to any of its constituents

Other protocol defined inclusion and exclusion criteria may apply

Where this trial is running

Atlanta, Georgia and 14 other locations

• Children's Healthcare of Atlanta Center for Advanced Pediatrics — Atlanta, Georgia, United States (RECRUITING)
• University of Massachusetts Memorial Childrens Medical Center — Worcester, Massachusetts, United States (RECRUITING)
• Corewell Health — Grand Rapids, Michigan, United States (RECRUITING)
• Columbia University Medical Center — New York, New York, United States (RECRUITING)
• Neurology Rare Disease Center — Denton, Texas, United States (RECRUITING)
• Child's Hosp King's Daughters — Norfolk, Virginia, United States (RECRUITING)
• Policlinico Agostino Gemelli — Roma, Lazio, Italy (RECRUITING)
• Fondazione IRCCS Istituto Neurologico ?Carlo Besta? — Milano, Lombardia, Italy (RECRUITING)
• Uniwersyteckie Centrum Kliniczne — Gdansk, Poland (RECRUITING)
• Instytut Centrum Zdrowia Matki Polki — Lódz, Poland (RECRUITING)
• Uniwersytecki Szpital Kliniczny w Poznaniu — Poznan, Poland (RECRUITING)
• Uniwersyteckie Centrum Kliniczne WUM, Centralny Szpital Kliniczny — Warszawa, Poland (RECRUITING)
• Hospital Sant Joan De Deu — Esplugues De Llobregas, Barcelona, Spain (RECRUITING)
• Hospital U. Central de Asturias — Asturias, Spain (RECRUITING)
• Hospital Universitario la Fe — Valencia, Spain (RECRUITING)

Study contacts

• Study coordinator: Reference Study ID Number: BN45398 https://forpatients.roche.com/

Conditions: Duchenne Muscular Dystrophy, DMD, IL6, IL6R, bone, muscle, fractures, BMD, inflammation