Active, not recruiting
Phase 2
A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD)
Condition(s) studied
Duchenne Muscular Dystrophy
Investigational drug(s) / intervention(s)
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).
Study 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.
Eligibility
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
Primary outcome measure(s)
- 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) — Baseline up to Week 24
BMD of the LS is measured using DEXA.
Trial sites (18)
| Facility | City | Region | Status |
| Arkansas Children's Hospital |
Little Rock |
Arkansas |
|
| University of California Davis Medical Center |
Sacramento |
California |
|
| Children's Healthcare of Atlanta Center for Advanced Pediatrics |
Atlanta |
Georgia |
|
| Corewell Health |
Grand Rapids |
Michigan |
|
| Neurology Rare Disease Center |
Flower Mound |
Texas |
|
| Child's Hosp King's Daughters |
Norfolk |
Virginia |
|
| Rigshospitalet;Klinik for Børn og Unge med Hjerne- og Nervesygdomme |
København Ø |
Denmark |
|
| Policlinico Agostino Gemelli |
Rome |
Lazio |
|
| Fondazione IRCCS Istituto Neurologico ?Carlo Besta? |
Milan |
Lombardy |
|
| Uniwersyteckie Centrum Kliniczne |
Gdansk |
Poland |
|
| Instytut Centrum Zdrowia Matki Polki |
Lodz |
Poland |
|
| Uniwersyteckie Centrum Kliniczne WUM, Centralny Szpital Kliniczny |
Warsaw |
Poland |
|
| Hospital Sant Joan De Deu |
Esplugues de Llobregas |
Barcelona |
|
| Hospital U. Central de Asturias |
Asturias |
Principality of Asturias |
|
| Hospital Universitario Torrecardenas;Servicio de Neurologia |
Almería |
Spain |
|
| Hospital Universitario la Fe |
Valencia |
Spain |
|
| Ohmatdyt - National Specialized children's hospital of MoH of Ukraine |
Kyiv |
Ukraine |
|
| Lvivska oblasna tsentralna likarnia |
Lviv |
Ukraine |
|
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