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

NCT06450639 | Active Not Recruiting Phase 2 FDA Drug

• 2 other identifiers: BN453982024-512383-65-00
• Study Type: interventional
• Target: 30
• Locations: 6 countries
• Sites: 22

Brief 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 age ≥ 8 to \< 18 years old receiving corticosteroid therapy.

Conditions

Duchenne Muscular Dystrophy

Keywords

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

Outcome Measures

Primary Outcomes (1)

• Group 2: Change From Baseline to Week 52 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 to Week 52

Secondary Outcomes (16)

• All Participants: Change From Baseline to Weeks 24, 52, and 104 in LS BMD Z-score Measured by DEXA

  Baseline to Week 24, Week 52 and Week 104

• Group 2: Change From Baseline to Weeks 24 and 104 in LS BMD Z-score Measured by DEXA

  Baseline to Week 24 and Week 104

• Group 2: Change From Baseline to Weeks 24, 52 and 104 in Total Body Less Head Bone Mineral Density (TBLH BMD) Z-score Measured by DEXA

  Baseline to Week 24, Week 52 and Week 104

• Group 2: Change From Baseline to Weeks 24, 52 and 104 in Total Hip BMD Z-score Measured by DEXA

  Baseline to Week 24, Week 52 and Week 104

• Group 2: Change From Baseline to Weeks 12, 24 and 52 in Circulating Bone Metabolism Biomarkers

  Baseline to Week 12, Week 24 and Week 52

Study Arms (1)

Satralizumab

EXPERIMENTAL

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.

Drug: Satralizumab

Interventions

Satralizumab DRUG

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 to 104 (maintenance doses) until the study completion visit.

Satralizumab

Eligibility Criteria

• Age: 8 Years - 17 Years
• Gender Eligibility Details: Male participants
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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:
• \- Ambulatory (defined as able to walk independently without assistive devices) with a prior history of fractures:
• 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
• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Hoffmann-La Roche: lead

Study Sites (22)

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

Location

University of California Davis Medical Center

Sacramento, California, 95817, United States

Location

Children's Healthcare of Atlanta Center for Advanced Pediatrics

Atlanta, Georgia, 30329, United States

Location

Corewell Health

Grand Rapids, Michigan, 49503, United States

Location

Neurology Rare Disease Center

Flower Mound, Texas, 75028, United States

Location

Child's Hosp King's Daughters

Norfolk, Virginia, 23507, United States

Location

Rigshospitalet;Klinik for Børn og Unge med Hjerne- og Nervesygdomme

København Ø, 2100, Denmark

Location

Policlinico Agostino Gemelli

Rome, Lazio, 00168, Italy

Location

IRCCS Istituto G. Gaslini

Genoa, Liguria, 16147, Italy

Location

Fondazione IRCCS Istituto Neurologico ?Carlo Besta?

Milan, Lombardy, 20133, Italy

Location

Uniwersyteckie Centrum Kliniczne

Gdansk, 80-952, Poland

Location

Instytut Centrum Zdrowia Matki Polki

Lodz, 93-338, Poland

Location

Uniwersytecki Szpital Kliniczny w Poznaniu

Późna, 60-355, Poland

Location

Uniwersyteckie Centrum Kliniczne WUM, Centralny Szpital Kliniczny

Warsaw, 02-097, Poland

Location

Hospital Sant Joan De Deu

Esplugues de Llobregas, Barcelona, 08950, Spain

Location

Hospital U. Central de Asturias

Asturias, Principality of Asturias, 33011, Spain

Location

Hospital Universitario Torrecardenas;Servicio de Neurologia

Almería, 04009, Spain

Location

Hospital Universitario la Fe

Valencia, 46026, Spain

Location

Ivano-Frankivsk Regional Children Clinical Hospital

Ivano-Frankivsk, Kharkiv Governorate, 76018, Ukraine

Location

Ukrainian medical rehabilitation center for children with organic lesions of the nervous system

Kyiv, KIEV Governorate, 04209, Ukraine

Location

Ohmatdyt - National Specialized children's hospital of MoH of Ukraine

Kyiv, 01135, Ukraine

Location

Lvivska oblasna tsentralna likarnia

Lviv, 79010, Ukraine

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne; Fractures, Bone; Inflammation

Interventions

satralizumab

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Wounds and Injuries; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Clinical Trials

  Hoffmann-La Roche

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 5, 2024
• First Posted: June 10, 2024
• Study Start: April 4, 2025
• Primary Completion (Estimated): August 26, 2026
• Study Completion (Estimated): November 18, 2026
• Last Updated: April 9, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share

Locations

IT (3); DK (1); US (6); UA (4); ES (4); PL (4)