RECRUITING

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

Conditions

Duchenne Muscular Dystrophy DMD, IL6, IL6R, bone, muscle, fractures, BMD, inflammation

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

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)

Quick Facts

Study Start:2025-02-15

Study Completion:2027-11-15

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06450639

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:8 Years to 17 Years

Sexes Eligible for Study:MALE

Accepts Healthy Volunteers:No

Standard Ages:CHILD

Inclusion Criteria	Exclusion 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: 1. 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 2. 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 * Be ambulatory defined as able to walk independently without assistive devices * Age ≥ 8 to \< 12 years old at the time of screening * Daily oral corticosteroids	* 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. * 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

Inclusion Criteria

Exclusion 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:
1. 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
2. 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
* Be ambulatory defined as able to walk independently without assistive devices
* Age ≥ 8 to \< 12 years old at the time of screening
* Daily oral corticosteroids

* 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.
* 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

Contacts and Locations

Study Contact

Reference Study ID Number: BN45398 https://forpatients.roche.com/

CONTACT

888-662-6728 (U.S.)

global-roche-genentech-trials@gene.com

Global Medical Information:

CONTACT

global-roche-genentech-trials@gene.com

Principal Investigator

Clinical Trials

STUDY_DIRECTOR

Hoffmann-La Roche

Study Locations (Sites)

Corewell Health

Grand Rapids, Michigan, 49503

United States

Neurology Rare Disease Center

Denton, Texas, 76208

United States

Child's Hosp King's Daughters

Norfolk, Virginia, 23507

United States

Collaborators and Investigators

Sponsor: Hoffmann-La Roche

Clinical Trials, STUDY_DIRECTOR, Hoffmann-La Roche

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-02-15

Study Completion Date2027-11-15

Study Record Updates

Study Start Date2025-02-15

Study Completion Date2027-11-15

Terms related to this study

Keywords Provided by Researchers

DMD, IL6, IL6R, bone, muscle, fractures, BMD, inflammation

Additional Relevant MeSH Terms

Duchenne Muscular Dystrophy