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

September 3, 2026 updated by: Hoffmann-La Roche

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)

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 aged ≥ 8 to < 18 years old receiving corticosteroid therapy.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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-naïve (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).

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
30

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Contact Backup

Study Locations

  • Denmark
      • København Ø, Denmark, 2100
        • Rigshospitalet;Klinik for Børn og Unge med Hjerne- og Nervesygdomme
  • Italy
    • Lazio
      • Rome, Lazio, Italy, 00168
        • Policlinico Agostino Gemelli
    • Lombardy
      • Milan, Lombardy, Italy, 20133
        • Fondazione IRCCS Istituto Neurologico ?Carlo Besta?
  • Poland
      • Gdansk, Poland, 80-952
        • Uniwersyteckie Centrum Kliniczne
      • Lodz, Poland, 93-338
        • Instytut Centrum Zdrowia Matki Polki
      • Warsaw, Poland, 02-097
        • Uniwersyteckie Centrum Kliniczne WUM, Centralny Szpital Kliniczny
  • Spain
      • Almería, Spain, 04009
        • Hospital Universitario Torrecardenas;Servicio de Neurologia
      • Valencia, Spain, 46026
        • Hospital Universitario La Fe
    • Barcelona
      • Esplugues de Llobregas, Barcelona, Spain, 08950
        • Hospital Sant Joan de Déu
    • Principality of Asturias
      • Asturias, Principality of Asturias, Spain, 33011
        • Hospital U. Central de Asturias
  • Ukraine
      • Kyiv, Ukraine, 01135
        • Ohmatdyt - National Specialized children's hospital of MoH of Ukraine
      • Lviv, Ukraine, 79010
        • Lvivska oblasna tsentralna likarnia
  • United States
    • Arkansas
      • Little Rock, Arkansas, United States, 72202
        • Arkansas Children's Hospital
    • California
      • Sacramento, California, United States, 95817
        • University of California Davis Medical Center
    • Georgia
      • Atlanta, Georgia, United States, 30329
        • Children's Healthcare of Atlanta Center for Advanced Pediatrics
    • Michigan
      • Grand Rapids, Michigan, United States, 49503
        • Corewell Health
    • Texas
      • Flower Mound, Texas, United States, 75028
        • Neurology Rare Disease Center
    • Virginia
      • Norfolk, Virginia, United States, 23507
        • Child's Hosp King's Daughters

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.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Description

Key Inclusion Criteria:

  • Signed Informed Consent Form (ICF) 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 ICF
  • 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: a) 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 vertebral fractures [VF]) or history of at least one low-trauma long-bone fracture (upper or lower extremity) or b) 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

Key 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 virus (HCV), or hepatitis B virus (HBV) 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 kilograms (kg)
  • Evidence of a severe 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 live or live attenuated virus vaccine during the study
  • 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. Other protocol defined inclusion and exclusion criteria may apply

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: 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 until the study completion (maintenance doses).
Drug: Satralizumab
Satralizumab will be administered as SC injection in the abdominal or femoral region on Day 1, Weeks 2 and 4 (loading doses) and then Q4W from Week 8 until study completion (maintenance doses).
Other Names:
  • ENSPRYNG®; RO5333787

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Group 2: Change From Baseline to Week 24 in Lumbar Spine (LS) Bone Mineral Density (BMD) Z-score Measured by Dual-energy X-ray Absorptiometry (DEXA)
Time Frame: Baseline up to Week 24
BMD of the LS is measured using DEXA.
Baseline up to Week 24

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
All Participants: Change From Baseline to Week 24 in LS BMD Z-score Measured by DEXA
Time Frame: Baseline up to Week 24
Baseline up to Week 24
Group 2: Change From Baseline to Week 24 in Total Body Less Head (TBLH) BMD Z-score Measured by DEXA
Time Frame: Baseline up to Week 24
Baseline up to Week 24
Group 2: Change From Baseline to Week 24 in Total Hip BMD Z-score Measured by DEXA
Time Frame: Baseline up to Week 24
Baseline up to Week 24
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)
Time Frame: Up to 90 weeks
An adverse event (AE) is any untoward medical occurrence in a clinical study participant temporally associated with the use of a study treatment, whether or not considered related to the study treatment.
Up to 90 weeks
Percentage of Participants With Serious Adverse Events (SAEs)
Time Frame: Up to 90 weeks
An SAE is defined as any untoward medical occurrence that, at any dose: results in death; is life threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent disability or incapacity; is a congenital anomaly or birth defect; is medically significant.
Up to 90 weeks
Percentage of Participants With Adverse Events of Special Interest (AESIs)
Time Frame: Up to 90 weeks
Up to 90 weeks
Observed Serum Concentration of Satralizumab at Specified Trough Timepoints up to Study End
Time Frame: Up to 90 weeks
Up to 90 weeks
Apparent Clearance (CL) of Satralizumab
Time Frame: Up to 90 weeks
CL is a quantitative measure of the rate at which a drug substance is removed from the blood.
Up to 90 weeks
Apparent Volume of Distribution (Vd) of Satralizumab
Time Frame: Up to 90 weeks
Vd is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of a drug.
Up to 90 weeks
Area Under the Concentration-time Curve (AUC) of Satralizumab
Time Frame: Up to 90 weeks
AUC from time zero to the last quantifiable concentration of satralizumab in plasma.
Up to 90 weeks
Percentage of Participants With Anti-drug Antibodies (ADAs) to Satralizumab at Baseline and During the Study
Time Frame: Up to 90 weeks
Up to 90 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Hoffmann-La Roche

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Clinical Trials, 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 Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
April 4, 2025

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
November 18, 2026

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
November 18, 2026

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
June 5, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 5, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
June 10, 2024

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
September 4, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 3, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
September 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • BN45398
  • 2024-512383-65-00 (Ctis: EU CT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data_sharing

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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