A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD)

Launched by HOFFMANN-LA ROCHE · Jun 5, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a medication called satralizumab to see how well it works and if it is safe for boys with Duchenne muscular dystrophy (DMD), a condition that affects muscle strength. The trial is open to boys aged 8 to 16 years who are currently receiving corticosteroid treatment. Participants will include both those who can walk independently (ambulatory) and those who cannot (non-ambulatory). To join the study, boys must have a confirmed diagnosis of DMD and meet certain health criteria, such as not having a history of bone fractures or certain other medical conditions.

If a boy is eligible and chooses to participate, he will receive the study medication and be monitored closely by the research team. The trial will help researchers understand how satralizumab affects muscle function and overall health in boys with DMD. It's important to note that participants cannot have certain health issues or recent surgeries that could interfere with the study. This trial is currently recruiting participants, and it aims to contribute valuable information about potential new treatments for DMD.

Gender

MALE

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