Active — Not RecruitingPhase 2

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

United States30 participantsStarted 2025-04-04

Plain-language 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.

Who can participate

Age range8 Years – 17 Years

SexMALE

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Inclusion criteria

✓. 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

What they're measuring

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)

Timeframe: Baseline to Week 52

Trial details

NCT IDNCT06450639

SponsorHoffmann-La Roche

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2026-08-26

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