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)

Hoffmann-La Roche43 sites in 6 countries30 target enrollmentApril 4, 2025

ConditionsDuchenne Muscular Dystrophy

InterventionsSatralizumab

DrugsSatralizumab

Overview

Phase: Phase 2
Intervention: Satralizumab
Conditions: Duchenne Muscular Dystrophy
Sponsor: Hoffmann-La Roche
Enrollment: 30
Locations: 43
Primary Endpoint: Group 2: Change From Baseline to Week 52 in Lumbar Spine (LS) Bone Mineral Density (BMD) Z-score Measured by Dual-energy X-ray Absorptiometry (DEXA)
Status: Active, not recruiting
Last Updated: 23 days ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

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 participants with DMD age ≥ 8 to < 18 years old receiving corticosteroid therapy.

Detailed Description

Participants will be included in two groups: ambulatory participants with fractures and non-ambulatory participants with or without a history of fractures (Group 1) and ambulatory participants who are fracture naive (Group 2) 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 (SC) injection every 4 weeks (Q4W).

Registry

clinicaltrials.gov

Start Date

April 4, 2025

End Date

November 18, 2026

Last Updated

23 days ago

Study Type

Interventional

Study Design

Single Group

Sex

Male

Investigators

Sponsor

Hoffmann-La Roche

Responsible Party

Sponsor

Eligibility Criteria

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:
•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

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.

Arms & Interventions

Satralizumab

Participants will receive satralizumab SC injection on Day 1, Weeks 2 and 4 (loading doses) and then Q4W from Weeks 8 to 104 (maintenance doses) until the study completion visit.

Intervention: Satralizumab

Outcomes

Primary Outcomes

Group 2: Change From Baseline to Week 52 in Lumbar Spine (LS) Bone Mineral Density (BMD) Z-score Measured by Dual-energy X-ray Absorptiometry (DEXA)

Time Frame: Baseline to Week 52

BMD of the LS is measured using DEXA

Secondary Outcomes

All Participants: Change From Baseline to Weeks 24, 52, and 104 in LS BMD Z-score Measured by DEXA(Baseline to Week 24, Week 52 and Week 104)
Group 2: Change From Baseline to Weeks 24 and 104 in LS BMD Z-score Measured by DEXA(Baseline to Week 24 and Week 104)
Group 2: Change From Baseline to Weeks 24, 52 and 104 in Total Body Less Head Bone Mineral Density (TBLH BMD) Z-score Measured by DEXA(Baseline to Week 24, Week 52 and Week 104)
Group 2: Change From Baseline to Weeks 24, 52 and 104 in Total Hip BMD Z-score Measured by DEXA(Baseline to Week 24, Week 52 and Week 104)
Group 2: Change From Baseline to Weeks 12, 24 and 52 in Circulating Bone Metabolism Biomarkers(Baseline to Week 12, Week 24 and Week 52)
All Participants: Change From Baseline to Weeks 12, 24 and 52 in Circulating Bone Metabolism Biomarkers(Baseline to Week 12, Week 24, and Week 52)
All Participants: Mean Number Per Participants of New Low-trauma Long-bone or Vertebral Fractures (VF)(Baseline to Week 52 and Week 104)
All Participants: Percentage of Participants With New Low-trauma Long-bone or VF(Baseline to Week 52 and Week 104)
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)(Up to approximately 30 Months)
Percentage of Participants With Serious Adverse Events (SAEs)(Up to approximately 30 Months)
Percentage of Participants With Adverse Events of Special Interest (AESIs)(Up to approximately 30 Months)
Observed Serum Concentration of Satralizumab at Specified Trough Timepoints up to Week 104(Up to Week 104)
Apparent Clearance of Satralizumab(Up to Week 104)
Apparent Volume of Distribution of Satralizumab(Up to Week 104)
Area Under the Concentration-time Curve of Satralizumab(Up to Week 104)
Percentage of Participants With Anti-drug Antibodies (ADAs) at Baseline and During the Study(Up to approximately 30 Months)