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NCT06450639 | RECRUITING | Duchenne Muscular Dystrophy

An Open-label Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy
Sponsor:

Hoffmann-La Roche

Brief Summary:

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 patients with Duchenne muscular dystrophy (DMD) age ≥ 8 to \< 16 years old receiving corticosteroid therapy.

Condition or disease

Duchenne Muscular Dystrophy

Intervention/treatment

Satralizumab

Phase

PHASE2

Detailed Description:

Participants will be included in two groups: those presenting with fractures at baseline and those that are fracture naive at baseline. The study will assess the potential of satralizumab to improve bone fragility and to increase muscle function. A weight tier based dose of satralizumab will be given by subcutaneous injection every 4 weeks

Study Type : INTERVENTIONAL
Estimated Enrollment : 50 participants
Masking : NONE
Primary Purpose : TREATMENT
Official Title : 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)
Actual Study Start Date : 2024-11-29
Estimated Primary Completion Date : 2026-06-30
Estimated Study Completion Date : 2027-06-30

Information not available for Arms and Intervention/treatment

Ages Eligible for Study: 8 Years to 15 Years
Sexes Eligible for Study: MALE
Accepts Healthy Volunteers:
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 \< 16 years at the time of signing Informed Consent Form
  • * Participants who are fracture-naive are additionally required to meet the following criteria
    • * 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
    • * Participants with a prior history of low-trauma fractures are additionally required to meet the following criteria
      • * Evidence of at least one prevalent vertebral compression fracture of Genant Grade 1 or higher (or radiographic signs of VF) or history of at least one low-trauma long-bone fracture (upper or lower extremity)
      • * Ambulatory (see above definition) if age ≥ 8 to \< 12.
      • * Non-ambulatory if age ≥ 12 to \<16
      • * 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, tuberculosis, 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 \> 100 kg •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 lumbar spine 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
An Open-label Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy

Location Details

NCT06450639

Please Choose a site

How to Participate

Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.

Locations

RECRUITING

United States, road cancer

Corewell Health

Grand Rapids, road cancer, United States, 49503

RECRUITING

United States, Virginia

Child(ren's) Hosp(ital) of the king's daughters

Norfolk, Virginia, United States, 23507

RECRUITING

Poland,

University Clinical Hospital in Poznań; From. Clinical Department of Child and Adolescent Neurology

Poznań, Poland, 60-355

RECRUITING

Poland,

Polish Mother's Health Center Institute; Department of Developmental Neurology and Epileptology

Łódź, Poland, 93-338