Duchenne Muscular DystrophyPhase 2SHIELD DMDNCT06450639
A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD)
A Phase 2 study testing Satralizumab in people with Duchenne Muscular Dystrophy, run by Hoffmann-La Roche at 18 sites in 6 countries.
Trial 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 aged ≥ 8 to < 18 years old receiving corticosteroid therapy. Read more about this study
- Who is it for?
- Ages 8 to 17 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Satralizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 18 sitesin 6 countries
- Study dates
- 4 Apr 2025to 18 Nov 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 to 17 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
18 sites in 6 countries.
18 sites in 6 countries
United States6 sites
Children's Healthcare of Atlanta Center for Advanced Pediatrics
Atlanta, Georgia, 30329
Neurology Rare Disease Center
Flower Mound, Texas, 75028
Corewell Health
Grand Rapids, Michigan, 49503
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
Child's Hosp King's Daughters
Norfolk, Virginia, 23507
University of California Davis Medical Center
Sacramento, California, 95817
Spain4 sites
Hospital Universitario Torrecardenas;Servicio de Neurologia
Almería, 04009
Hospital U. Central de Asturias
Asturias, Principality of Asturias, 33011
Hospital Sant Joan De Deu
Esplugues de Llobregas, Barcelona, 08950
Hospital Universitario la Fe
Valencia, 46026
Poland3 sites
Uniwersyteckie Centrum Kliniczne
Gdansk, 80-952
Instytut Centrum Zdrowia Matki Polki
Lodz, 93-338
Uniwersyteckie Centrum Kliniczne WUM, Centralny Szpital Kliniczny
Warsaw, 02-097
Italy2 sites
Fondazione IRCCS Istituto Neurologico ?Carlo Besta?
Milan, Lombardy, 20133
Policlinico Agostino Gemelli
Rome, Lazio, 00168
Ukraine2 sites
Ohmatdyt - National Specialized children's hospital of MoH of Ukraine
Kyiv, 01135
Lvivska oblasna tsentralna likarnia
Lviv, 79010
Denmark1 site
Rigshospitalet;Klinik for Børn og Unge med Hjerne- og Nervesygdomme
København Ø, 2100
About this study
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)
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.
Read the 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).
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Satralizumab
- Keywords
- DMD, IL6, IL6R, bone, muscle, fractures, BMD, inflammation
- Medical subject headings
- Muscular Dystrophy, Duchenne, Fractures, Bone, Inflammation
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SatralizumabDrug
Also called ENSPRYNG®; RO5333787
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).
| Study group | Role | What they receive |
|---|---|---|
| Satralizumab | Experimental | 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). Receives: Drug: Satralizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
BMD of the LS is measured using DEXA.
Measured: Baseline up to Week 24
Secondary outcomes (11)
All Participants: Change From Baseline to Week 24 in LS BMD Z-score Measured by DEXA
Measured: 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
Measured: Baseline up to Week 24
Group 2: Change From Baseline to Week 24 in Total Hip BMD Z-score Measured by DEXA
Measured: Baseline up to Week 24
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)
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.
Measured: Up to 90 weeks
Percentage of Participants With Serious Adverse Events (SAEs)
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.
Measured: Up to 90 weeks
Percentage of Participants With Adverse Events of Special Interest (AESIs)
Measured: Up to 90 weeks
Observed Serum Concentration of Satralizumab at Specified Trough Timepoints up to Study End
Measured: Up to 90 weeks
Apparent Clearance (CL) of Satralizumab
CL is a quantitative measure of the rate at which a drug substance is removed from the blood.
Measured: Up to 90 weeks
Apparent Volume of Distribution (Vd) of Satralizumab
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.
Measured: Up to 90 weeks
Area Under the Concentration-time Curve (AUC) of Satralizumab
AUC from time zero to the last quantifiable concentration of satralizumab in plasma.
Measured: Up to 90 weeks
Percentage of Participants With Anti-drug Antibodies (ADAs) to Satralizumab at Baseline and During the Study
Measured: Up to 90 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jun 2024
- Study start4 Apr 2025
- Primary completion18 Nov 2026 (estimated)
- Study completion18 Nov 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- More trials run by Hoffmann-La RocheThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hoffmann-La Roche
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Hoffmann-La Roche
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06450639
- Sponsor's protocol ID
- BN45398
- Other IDs
- 2024-512383-65-00
- Acronym
- SHIELD DMD
- Status last verified
- Sep 2026
- First submitted
- 5 Jun 2024
- Last updated
- 4 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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
Study at a glance
From the registry record, last updated by the sponsor on 4 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT06450639
- Acronym / sponsor ID
- SHIELD DMD / BN45398
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- DMD, IL6, IL6R, bone, muscle, fractures, BMD, inflammation
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Hoffmann-La RocheIndustry (pharma, biotech, devices)
- Study start
- 4 Apr 2025 (actual)
- Primary completion
- 18 Nov 2026 (estimated)
- Study completion
- 18 Nov 2026 (estimated)
- First posted
- 10 Jun 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Satralizumab | Experimental | Drug: SatralizumabSatralizumab 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). |
Who may qualify
- Ages
- 8 to 17 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (12 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SatralizumabAlso: ENSPRYNG®; RO5333787Satralizumab 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). | Drug | Satralizumab |
Primary outcome
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.
11 secondary outcomes
All Participants: Change From Baseline to Week 24 in LS BMD Z-score Measured by DEXA
Time frame: 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
Group 2: Change From Baseline to Week 24 in Total Hip BMD Z-score Measured by DEXA
Time frame: Baseline up to Week 24
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)
Time frame: Up to 90 weeks
Percentage of Participants With Serious Adverse Events (SAEs)
Time frame: Up to 90 weeks
Percentage of Participants With Adverse Events of Special Interest (AESIs)
Time frame: Up to 90 weeks
Observed Serum Concentration of Satralizumab at Specified Trough Timepoints up to Study End
Time frame: Up to 90 weeks
Apparent Clearance (CL) of Satralizumab
Time frame: Up to 90 weeks
Apparent Volume of Distribution (Vd) of Satralizumab
Time frame: Up to 90 weeks
Area Under the Concentration-time Curve (AUC) of Satralizumab
Time frame: 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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Sep 2026.
- Individual participant dataSharing planned: Yes. 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
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Trials study director
Hoffmann-La Roche
18 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Satralizumab in people with Duchenne Muscular Dystrophy, run by Hoffmann-La Roche at 18 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 4 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites in United States, Spain, Poland, Italy, Ukraine and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Sep 2026.