Satralizumab for Duchenne Muscular Dystrophy

(SHIELD DMD Trial)

Recruiting at 41 trial locations

Overseen By Global Medical Information:

Age: < 18

Sex: Male

Trial Phase: Phase 2

Sponsor: Hoffmann-La Roche

Must be taking: Corticosteroids

Disqualifiers: HIV, Tuberculosis, Hepatitis B, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 5 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 4

Duration: 104 weeks

50 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the safety and effectiveness of satralizumab, a medication targeting the immune system, in individuals with Duchenne Muscular Dystrophy (DMD) who are already on corticosteroid therapy. The focus includes both those who can walk independently and those who use wheelchairs. Suitable participants are boys aged 8 to 17 diagnosed with DMD, taking daily corticosteroids, and either have experienced fractures or have no history of fractures. As a Phase 2 trial, the research measures how well the treatment works in an initial, smaller group of people.

Do I have to stop taking my current medications for the trial?

The trial requires participants to be on daily oral corticosteroids, but it does not specify if you need to stop other medications. It's best to discuss your current medications with the trial team to get a clear answer.

Is there any evidence suggesting that satralizumab is likely to be safe for humans?

Research has shown that satralizumab has been tested to assess its safety in humans. This treatment is a monoclonal antibody designed to block a protein that can cause inflammation. Earlier studies found that satralizumab was generally well-tolerated, with most participants not experiencing serious side effects. Some reported mild issues like headaches or injection site reactions. Since this trial is in Phase 2, satralizumab has already demonstrated some safety in humans. However, further testing is needed to confirm its safety for individuals with Duchenne Muscular Dystrophy. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Duchenne Muscular Dystrophy?

Satralizumab is unique because it offers a new approach to treating Duchenne Muscular Dystrophy (DMD) by targeting the interleukin-6 (IL-6) receptor, which plays a role in inflammation. Most existing treatments for DMD, like corticosteroids, focus on reducing inflammation broadly but can have significant side effects over time. Satralizumab's targeted action may provide more effective inflammation control with potentially fewer side effects. Researchers are excited about its potential to improve muscle function and slow disease progression in a way that current therapies do not.

What evidence suggests that satralizumab might be an effective treatment for Duchenne Muscular Dystrophy?

Research has shown that satralizumab, a drug targeting a specific part of the immune system, may improve muscle function and bone strength in people with Duchenne Muscular Dystrophy (DMD). Studies have found that satralizumab reduces inflammation by blocking the interleukin-6 receptor, which contributes to muscle damage and weakness in DMD. Early results suggest that this reduction in inflammation might protect muscles and bones, potentially leading to better movement and stronger bones. This promising evidence supports the idea that satralizumab could benefit people with DMD. Participants in this trial will receive satralizumab as part of the study's experimental treatment arm. ^{1 2 4 6 7}

Who Is on the Research Team?

Clinical Trials

Principal Investigator

Hoffmann-La Roche

Are You a Good Fit for This Trial?

This trial is for boys aged 8 to less than 16 with Duchenne Muscular Dystrophy (DMD) who can walk or not and are on corticosteroid therapy. Details about specific inclusion and exclusion criteria are not provided, but typically these would outline health requirements and any medications or conditions that might interfere with the study.

Inclusion Criteria

Participants with a prior history of low-trauma fractures are additionally required to meet the following criteria:

I have a confirmed diagnosis of DMD based on genetic testing.

Signed Informed Consent Form and Signed Assent Form when appropriate

Exclusion Criteria

Has serological evidence of current, chronic, or active human immunodeficiency virus, tuberculosis, hepatitis C, or hepatitis B infection

I haven't had major surgery in the last 3 months and don't plan any that could affect this study.

I have a serious health condition.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive satralizumab SC injection on Day 1, Weeks 2 and 4 (loading doses) and then Q4W from Weeks 8 to 104 (maintenance doses)

104 weeks

In-person visits every 4 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension (optional)

Participants may opt into continuation of treatment long-term

Long-term

What Are the Treatments Tested in This Trial?

Interventions

• Satralizumab

Trial Overview The trial is testing Satralizumab, an antibody targeting the IL-6 receptor, which may affect inflammation. It aims to see how well it works (efficacy), its safety profile, how the body processes it (pharmacokinetics), and how it affects the body's response (pharmacodynamics).

How Is the Trial Designed?

Group I: Satralizumab Experimental Treatment 1 Intervention

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.

Satralizumab is already approved in United States, European Union, Canada, Japan, Switzerland for the following indications:

Approved in United States as Enspryng for:

• Neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive

Approved in European Union as Enspryng for:

• Neuromyelitis optica spectrum disorder (NMOSD)

Approved in Canada as Enspryng for:

• Neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive

Approved in Japan as Enspryng for:

• Neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive

Approved in Switzerland as Enspryng for:

• Neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive

Who Is Running the Clinical Trial?

Hoffmann-La Roche

Lead Sponsor

Trials

2,482

Recruited

1,107,000+

Headquarters

Basel, Switzerland

Known For

Precision medicine

Top Products

Avastin, Herceptin, Rituxan, Accu-Chek

Dr. Levi Garraway

Hoffmann-La Roche

Chief Medical Officer since 2019

MD from the University of Basel

Dr. Thomas Schinecker

Hoffmann-La Roche

Chief Executive Officer since 2023

PhD in Molecular Biology from New York University

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06450639

NCT06450639 | A Study to Assess the Efficacy and Safety ... 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 ...

2. neurologylive.com neurologylive.com/view/shield-dmd-evaluate-therapeutic-potential-il-6-receptor-satralizumab-duchenne-muscular-dystrophy

SHIELD-DMD to Evaluate Therapeutic Potential of IL-6 ... SHIELD-DMD trial explores IL-6R inhibition with satralizumab to improve bone and muscle health in DMD patients. · Participants are divided by age ...

3. parentprojectmd.org parentprojectmd.org/clinical-trial/a-study-to-assess-the-efficacy-and-safety-of-satralizumab-in-duchenne-muscular-dystrophy-dmd/

A Study to Assess the Efficacy and Safety of Satralizumab ... The study will assess the potential of satralizumab to improve bone fragility and to increase muscle function.

4. trialx.com trialx.com/clinical-trials/listings/293869/a-study-to-assess-the-efficacy-and-safety-of-satralizumab-in-duchenne-muscular-dystrophy-dmd/

A Study to Assess the Efficacy and Safety of Satralizumab ... Overview. The purpose of this study is to assess the efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of satralizumab, ...

5. forpatients.roche.com forpatients.roche.com/en/trials/muscle-and-peripheral-nerve-disease/dmd/an-open-label-study-to-assess-the-efficacy-and-safety-o-12382.html

Clinical Study / satralizumab RO5333787 - ForPatients - Roche This study is testing a medicine called satralizumab (RO5333787) to see if it can help with the muscle and bone issues of people living with DMD.

6. genentech-clinicaltrials.com genentech-clinicaltrials.com/en/trials/muscle-and-peripheral-nerve-disease/dmd/an-open-label-study-to-assess-the-efficacy-and-safety-o-12382.html

Clinical Study / satralizumab RO5333787 | ForPatients-Roche This study is testing a medicine called satralizumab (RO5333787) to see if it can help with the muscle and bone issues of people living with DMD.

7. centerwatch.com centerwatch.com/clinical-trials/listings/NCT06450639/a-study-to-assess-the-efficacy-and-safety-of-satralizumab-in-duchenne-muscular-dystrophy-dmd

A Study to Assess the Efficacy and Safety of Satralizumab ... The purpose of this study is to assess the efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of satralizumab, a humanized ...