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)

Status: Recruiting
Location: See all (18) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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.

Eligibility
Participation Requirements
Sex: Male
Minimum Age: 8
Maximum Age: 17
Healthy Volunteers: f
View:

• 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

Locations
United States
Arkansas
Arkansas Children's Hospital
RECRUITING
Little Rock
Georgia
Children's Healthcare of Atlanta Center for Advanced Pediatrics
RECRUITING
Atlanta
Massachusetts
University of Massachusetts Memorial Childrens Medical Center
RECRUITING
Worcester
Michigan
Corewell Health
RECRUITING
Grand Rapids
New York
Columbia University Medical Center
RECRUITING
New York
Texas
Neurology Rare Disease Center
RECRUITING
Denton
Virginia
Child's Hosp King's Daughters
RECRUITING
Norfolk
Other Locations
Italy
IRCCS Istituto G. Gaslini
RECRUITING
Genoa
Fondazione IRCCS Istituto Neurologico ?Carlo Besta?
RECRUITING
Milan
Policlinico Agostino Gemelli
RECRUITING
Rome
Poland
Instytut Centrum Zdrowia Matki Polki
RECRUITING
?ód?
Uniwersyteckie Centrum Kliniczne
RECRUITING
Gda?sk
Uniwersytecki Szpital Kliniczny w Poznaniu
RECRUITING
Późna
Uniwersyteckie Centrum Kliniczne WUM, Centralny Szpital Kliniczny
RECRUITING
Warsaw
Spain
Hospital Universitario Torrecardenas;Servicio de Neurologia
RECRUITING
Almería
Hospital U. Central de Asturias
RECRUITING
Asturias
Hospital Sant Joan De Deu
RECRUITING
Esplugues De Llobregas
Hospital Universitario la Fe
RECRUITING
Valencia
Contact Information
Primary
Reference Study ID Number: BN45398 https://forpatients.roche.com/
global-roche-genentech-trials@gene.com
888-662-6728 (U.S.)
Backup
Global Medical Information:
global-roche-genentech-trials@gene.com
Time Frame
Start Date: 2025-04-04
Estimated Completion Date: 2027-11-15
Participants
Target number of participants: 50
Treatments
Experimental: 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.
Sponsors
Leads: Hoffmann-La Roche

This content was sourced from clinicaltrials.gov