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)

Conditions

Duchenne Muscular Dystrophy (DMD)

Brief summary

1. Change from baseline to Week 52 in LS BMD Z-score measured by DEXA

Detailed description

1. Incidence of treatment-emergent adverse events, 2. Incidence of serious adverse events, 3. Incidence of adverse events of special interest, 4. Clinically significant changes in vital signs and physical examination findings, 5. Clinically significant changes in safety laboratory assessments, ECGs, 6. Change from baseline to Weeks 24, 52 and 104 in LS BMD Z-score measured by DEXA, 7. Change from baseline to Weeks 24, and 52 and 104 in TBLH BMD Z-score measured by DEXA, 8. Change from baseline to Week 24, week 52 and week 104 in total hip BMD Z-score measured by DEXA, 9 . Change from baseline to Weeks 12, 24 and 52 in circulating bone metabolism biomarkers, 10 . Number of new low-trauma long-bone or vertebral fractures by Week 52 and week 104, 11 . Proportion of Participants with new low-trauma long-bone or vertebral fracture by Week 52 and week 104, 12. Summary of observed serum concentration of satralizumab at specified trough timepoints up to Week 104, 13. Population and individual estimates of PK parameters (e.g., apparent clearance and apparent volume of distribution) and secondary PK parameters (e.g., area under the concentration-time curve), 14. Prevalence of ADAs at baseline and incidence of ADAs during the study

Related papers

No related papers found yet.

Interventions

DRUGSatralizumab

Eligibility

Sex/Gender

Male

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
1. Change from baseline to Week 52 in LS BMD Z-score measured by DEXA	—

Secondary

Measure	Time frame
1. Incidence of treatment-emergent adverse events, 2. Incidence of serious adverse events, 3. Incidence of adverse events of special interest, 4. Clinically significant changes in vital signs and physical examination findings, 5. Clinically significant changes in safety laboratory assessments, ECGs, 6. Change from baseline to Weeks 24, 52 and 104 in LS BMD Z-score measured by DEXA, 7. Change from baseline to Weeks 24, and 52 and 104 in TBLH BMD Z-score measured by DEXA, 8. Change from baseline to Week 24, week 52 and week 104 in total hip BMD Z-score measured by DEXA, 9 . Change from baseline to Weeks 12, 24 and 52 in circulating bone metabolism biomarkers, 10 . Number of new low-trauma long-bone or vertebral fractures by Week 52 and week 104, 11 . Proportion of Participants with new low-trauma long-bone or vertebral fracture by Week 52 and week 104, 12. Summary of observed serum concentration of satralizumab at specified trough timepoints up to Week 104, 13. Population and individual	—

Measure

Time frame

1. Incidence of treatment-emergent adverse events, 2. Incidence of serious adverse events, 3. Incidence of adverse events of special interest, 4. Clinically significant changes in vital signs and physical examination findings, 5. Clinically significant changes in safety laboratory assessments, ECGs, 6. Change from baseline to Weeks 24, 52 and 104 in LS BMD Z-score measured by DEXA, 7. Change from baseline to Weeks 24, and 52 and 104 in TBLH BMD Z-score measured by DEXA, 8. Change from baseline to Week 24, week 52 and week 104 in total hip BMD Z-score measured by DEXA, 9 . Change from baseline to Weeks 12, 24 and 52 in circulating bone metabolism biomarkers, 10 . Number of new low-trauma long-bone or vertebral fractures by Week 52 and week 104, 11 . Proportion of Participants with new low-trauma long-bone or vertebral fracture by Week 52 and week 104, 12. Summary of observed serum concentration of satralizumab at specified trough timepoints up to Week 104, 13. Population and individual

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Countries

Denmark, Italy, Poland, Spain

Outcome results

None listed