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)
The purpose of this study is to assess the efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of satralizumab, a humanized anti-interleukin-6 receptor (aIL-6R) monoclonal antibody, in ambulatory and non-ambulatory participants with DMD age ≥ 8 to \< 18 years old receiving corticosteroid therapy.
• 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:
‣ 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
‣ 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