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RECRUITINGPhase 2INTERVENTIONAL

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

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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.

Who May Be Eligible (Plain English)

Key Who May Qualify: - Signed willing to sign a consent form 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 willing to sign a consent form 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 Key Who Should NOT Join This Trial: - 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 ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
Key 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 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, 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 Other protocol defined inclusion and exclusion criteria may apply

Treatments Being Tested

DRUG

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.

Locations (20)

Arkansas Children's Hospital
Little Rock, Arkansas, United States
University of California Davis Medical Center
Sacramento, California, United States
Children's Healthcare of Atlanta Center for Advanced Pediatrics
Atlanta, Georgia, United States
University of Massachusetts Memorial Childrens Medical Center
Worcester, Massachusetts, United States
Corewell Health
Grand Rapids, Michigan, United States
Billings Clinic Research Center
Billings, Montana, United States
Columbia University Medical Center
New York, New York, United States
Neurology Rare Disease Center
Flower Mound, Texas, United States
Child's Hosp King's Daughters
Norfolk, Virginia, United States
Rigshospitalet;Klinik for Børn og Unge med Hjerne- og Nervesygdomme
København Ø, Denmark
Policlinico Agostino Gemelli
Rome, Lazio, Italy
IRCCS Istituto G. Gaslini
Genoa, Liguria, Italy
Fondazione IRCCS Istituto Neurologico ?Carlo Besta?
Milan, Lombardy, Italy
Uniwersyteckie Centrum Kliniczne
Gdansk, Poland
Instytut Centrum Zdrowia Matki Polki
Lodz, Poland
Uniwersytecki Szpital Kliniczny w Poznaniu
Późna, Poland
Uniwersyteckie Centrum Kliniczne WUM, Centralny Szpital Kliniczny
Warsaw, Poland
Hospital Sant Joan De Deu
Esplugues de Llobregas, Barcelona, Spain
Hospital U. Central de Asturias
Asturias, Principality of Asturias, Spain
Hospital Universitario Torrecardenas;Servicio de Neurologia
Almería, Spain