An Open-label, Randomized, Active-Controlled, Phase 3 Study of Setrusumab Compared With Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta Types I, III or IV

Conditions

osteogenesis imperfecta

Brief summary

Annualized rate of all radiographically-confirmed fractures, including morphometric vetrebral fractures at the primary analysis

Detailed description

Annualized rate of radiographically-confirmed fractures, excluding morphometric vertebral fractures,but including fractures of the fingers, toes, face, and skull at the primary analysis, Change from baseline in Pediatric Orthopedic Society of North America Pediatric Outcomes Data Collection Instrument (POSNA-PODCI) Sports/Physical Functioning and Pain/Comfort subscale scores at the primary analysis, Serum setrusumab concentration at scheduled time points, Frequency, severity, and relationship to treatment of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and adverse events of special interest (AESIs), Incidence of binding and neutralizing anti-setrusumab antibodies at scheduled time points, Percent change from baseline in DXA BMD at the lumbar spine at the primary analysis, Annualized rate of all radiographically-confirmed fractures, excluding morphometric vertebral fractures and fractures of the fingers, toes, face, and skull, at the primary analysis, Change from baseline in dual-energy X-ray absorptiometry (DXA) bone mineral density (BMD) zscore at the lumbar spine at the primary analysis, Proportion of subjects experiencing new radiographically-confirmed fractures, including morphometric vertebral fractures, at the primary analysis

Related papers

No related papers found yet.

Interventions

DRUGPAMIFOS-30

DRUG30 mg

DRUGproszek i rozpuszczalnik do sporządzania roztworu do infuzji

DRUGPAMIFOS-60

DRUG60 mg

DRUGPamidronate Disodium 3 mg/ml Sterile Concentrate

DRUGPamidronaatdinatrium Hospira 9 mg/ml concentraat voor oplossing voor infusie

DRUGAclasta 5 mg solution for infusion

DRUGPAMIDRONATE DE SODIUM HOSPIRA 9 mg/ml

DRUGsolution à diluer pour perfusion

DRUGNERIXIA 25 mg soluzione iniettabile

DRUGPamidronaatdinatrium Hospira 6 mg/ml concentraat voor oplossing voor infusie

DRUGNERIXIA 100 mg concentrato per soluzione per infusione

DRUGPamidronate disodique Hospira 3 mg / ml solution à diluer pour perfusion

DRUGSetrusumab

DRUGZoledronate EG 5 mg/100 ml solution pour perfusion

DRUGPAMIDRONATE DE SODIUM HOSPIRA 6 mg/ml

DRUGZoledronate EG 4 mg/100 ml solution pour perfusion

DRUGPamidronate disodique Hospira 9 mg / ml solution à diluer pour perfusion

DRUGPAMIFOS-90

DRUG90 mg

DRUGZoledronate EG 5mg/100ml Infusionslösung

DRUGPamidronate disodique Hospira 6 mg / ml solution à diluer pour perfusion

Eligibility

Sex/Gender

All

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
Annualized rate of all radiographically-confirmed fractures, including morphometric vetrebral fractures at the primary analysis	—

Secondary

Measure	Time frame
Annualized rate of radiographically-confirmed fractures, excluding morphometric vertebral fractures,but including fractures of the fingers, toes, face, and skull at the primary analysis, Change from baseline in Pediatric Orthopedic Society of North America Pediatric Outcomes Data Collection Instrument (POSNA-PODCI) Sports/Physical Functioning and Pain/Comfort subscale scores at the primary analysis, Serum setrusumab concentration at scheduled time points, Frequency, severity, and relationship to treatment of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and adverse events of special interest (AESIs), Incidence of binding and neutralizing anti-setrusumab antibodies at scheduled time points, Percent change from baseline in DXA BMD at the lumbar spine at the primary analysis, Annualized rate of all radiographically-confirmed fractures, excluding morphometric vertebral fractures and fractures of the fingers, toes, face, and skull, at the primary analysis, Change	—

Measure

Time frame

Annualized rate of radiographically-confirmed fractures, excluding morphometric vertebral fractures,but including fractures of the fingers, toes, face, and skull at the primary analysis, Change from baseline in Pediatric Orthopedic Society of North America Pediatric Outcomes Data Collection Instrument (POSNA-PODCI) Sports/Physical Functioning and Pain/Comfort subscale scores at the primary analysis, Serum setrusumab concentration at scheduled time points, Frequency, severity, and relationship to treatment of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and adverse events of special interest (AESIs), Incidence of binding and neutralizing anti-setrusumab antibodies at scheduled time points, Percent change from baseline in DXA BMD at the lumbar spine at the primary analysis, Annualized rate of all radiographically-confirmed fractures, excluding morphometric vertebral fractures and fractures of the fingers, toes, face, and skull, at the primary analysis, Change

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Countries

France, Germany, Italy, Netherlands, Poland

Outcome results

None listed