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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

50 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-04-04

Study Completion Date

2027-11-15

Brief Summary

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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.

Detailed Description

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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 naive (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

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Duchenne Muscular Dystrophy

Keywords

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

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Satralizumab

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.

Group Type EXPERIMENTAL

Satralizumab

Intervention Type 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.

Interventions

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

Intervention Type DRUG

Eligibility Criteria

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Inclusion 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:

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

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, 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

Minimum Eligible Age

8 Years

Maximum Eligible Age

17 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Clinical Trials

Role: STUDY_DIRECTOR

Hoffmann-La Roche

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

Role: CONTACT

Phone: 888-662-6728 (U.S.)

Email: [email protected]

Global Medical Information:

Role: CONTACT

Email: [email protected]

Other Identifiers

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2024-512383-65-00

Identifier Type: CTIS

Identifier Source: secondary_id

BN45398

Identifier Type: -

Identifier Source: org_study_id

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

Study Results

Basic Information

Brief Summary

Detailed Description

Conditions

Keywords

Study Design

Study Groups

Satralizumab

Satralizumab

Interventions

Satralizumab

Eligibility Criteria

Inclusion Criteria

Exclusion Criteria

Sponsors

Hoffmann-La Roche

Responsible Party

Principal Investigators

Clinical Trials

Locations

Arkansas Children's Hospital

University of California Davis Medical Center

Children's Healthcare of Atlanta Center for Advanced Pediatrics

University of Massachusetts Memorial Childrens Medical Center

Corewell Health

Billings Clinic Research Center

Columbia University Medical Center

Neurology Rare Disease Center

Child's Hosp King's Daughters

The Children's Hospital at Westmead

Queensland Children's Hospital

Perth Children's Hospital

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

Policlinico Agostino Gemelli

IRCCS Istituto G. Gaslini

Fondazione IRCCS Istituto Neurologico ?Carlo Besta?

Uniwersyteckie Centrum Kliniczne

Instytut Centrum Zdrowia Matki Polki

Uniwersytecki Szpital Kliniczny w Poznaniu

Uniwersyteckie Centrum Kliniczne WUM, Centralny Szpital Kliniczny

Hospital Sant Joan De Deu

Hospital U. Central de Asturias

Hospital Universitario Torrecardenas;Servicio de Neurologia

Hospital Universitario la Fe

Ivano-Frankivsk Regional Children Clinical Hospital

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

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

Lvivska oblasna tsentralna likarnia

Countries

Central Contacts

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

Global Medical Information:

Other Identifiers

2024-512383-65-00

BN45398