NCT06450639

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

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)

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 30 (actual)
• Sponsor: Hoffmann-La Roche · Industry
• Sex: Male
• Age: 8 Years – 17 Years
• Healthy volunteers: Not accepted

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).

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	Satralizumab	Satralizumab will be administered SC in the abdominal or femoral region on Day 1, Weeks 2 and 4 (loading doses) and then Q4W from Weeks 8 to 104 (maintenance doses) until the study completion visit.

Timeline

• Start date: 2025-04-04
• Primary completion: 2026-08-26
• Completion: 2026-11-18
• First posted: 2024-06-10
• Last updated: 2026-04-09

Locations

22 sites across 6 countries: United States, Denmark, Italy, Poland, Spain, Ukraine

Regulatory

• FDA-regulated drug study