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

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: Active, not recruiting
• Conditions: Duchenne Muscular Dystrophy
• Intervention / Treatment: Drug: Satralizumab

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: Interventional
• Enrollment (Actual): 30
• Phase: Phase 2

Contacts and Locations

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 Deu
  • 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
  • Kharkiv Governorate
    • Ivano-Frankivsk, Kharkiv Governorate, Ukraine, 76018
      • Ivano-Frankivsk Regional Children Clinical Hospital
• 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

Eligibility Criteria

• 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

How is the study designed?

Design Details

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

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
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

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)	BMD of the LS is measured using DEXA.	Baseline up to Week 24

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
All Participants: Change From Baseline to Week 24 in LS BMD Z-score Measured by DEXA		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		Baseline up to Week 24
Group 2: Change From Baseline to Week 24 in Total Hip BMD Z-score Measured by DEXA		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.	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.	Up to 90 weeks
Percentage of Participants With Adverse Events of Special Interest (AESIs)		Up to 90 weeks
Observed Serum Concentration of Satralizumab at Specified Trough Timepoints up to Study End		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.	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.	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.	Up to 90 weeks
Percentage of Participants With Anti-drug Antibodies (ADAs) to Satralizumab at Baseline and During the Study		Up to 90 weeks

Collaborators and Investigators

• Sponsor: Hoffmann-La Roche
• Investigators: Study Director: Clinical Trials, Hoffmann-La Roche

Study record dates

Study Major Dates

• Study Start (Actual): April 4, 2025
• Primary Completion (Estimated): August 26, 2026
• Study Completion (Estimated): November 18, 2026

Study Registration Dates

• First Submitted: June 5, 2024
• First Submitted That Met QC Criteria: June 5, 2024
• First Posted (Actual): June 10, 2024

Study Record Updates

• Last Update Posted (Actual): May 15, 2026
• Last Update Submitted That Met QC Criteria: May 13, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: DMD, IL6, IL6R, bone, muscle, fractures, BMD, inflammation
• Additional Relevant MeSH Terms: Musculoskeletal Diseases; Nervous System Diseases; Muscular Diseases; Wounds and Injuries; Pathologic Processes; Neuromuscular Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Muscular Disorders, Atrophic; Muscular Dystrophies; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Pathological Conditions, Signs and Symptoms; Fractures, Bone; Inflammation; Muscular Dystrophy, Duchenne; satralizumab
• Other Study ID 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