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.
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-naïve (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).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
30
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).
Arkansas Children's Hospital
Little Rock, Arkansas, United States
University of California Davis Medical Center
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)
BMD of the LS is measured using DEXA.
Time frame: Baseline up to Week 24
All Participants: Change From Baseline to Week 24 in LS BMD Z-score Measured by DEXA
Time frame: Baseline up to Week 24
Group 2: Change From Baseline to Week 24 in Total Body Less Head (TBLH) BMD Z-score Measured by DEXA
Time frame: Baseline up to Week 24
Group 2: Change From Baseline to Week 24 in Total Hip BMD Z-score Measured by DEXA
Time frame: Baseline up to Week 24
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)
An adverse event (AE) is any untoward medical occurrence in a clinical study participant temporally associated with the use of a study treatment, whether or not considered related to the study treatment.
Time frame: Up to 90 weeks
Percentage of Participants With Serious Adverse Events (SAEs)
An SAE is defined as any untoward medical occurrence that, at any dose: results in death; is life threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent disability or incapacity; is a congenital anomaly or birth defect; is medically significant.
Time frame: Up to 90 weeks
Percentage of Participants With Adverse Events of Special Interest (AESIs)
Time frame: Up to 90 weeks
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Sacramento, California, United States
Children's Healthcare of Atlanta Center for Advanced Pediatrics
Atlanta, Georgia, United States
Corewell Health
Grand Rapids, Michigan, 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
Fondazione IRCCS Istituto Neurologico ?Carlo Besta?
Milan, Lombardy, Italy
Uniwersyteckie Centrum Kliniczne
Gdansk, Poland
...and 9 more locations
Observed Serum Concentration of Satralizumab at Specified Trough Timepoints up to Study End
Time frame: Up to 90 weeks
Apparent Clearance (CL) of Satralizumab
CL is a quantitative measure of the rate at which a drug substance is removed from the blood.
Time frame: Up to 90 weeks
Apparent Volume of Distribution (Vd) of Satralizumab
Vd is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of a drug.
Time frame: Up to 90 weeks
Area Under the Concentration-time Curve (AUC) of Satralizumab
AUC from time zero to the last quantifiable concentration of satralizumab in plasma.
Time frame: Up to 90 weeks
Percentage of Participants With Anti-drug Antibodies (ADAs) to Satralizumab at Baseline and During the Study
Time frame: Up to 90 weeks