Safety and Efficiency of Denosumab in Pediatric Subjects With Glucocorticoid-induced Osteoporosis
Evaluate the Safety and Efficacy of Denosumab in Pediatric Subjects With, Glucocorticoid-induced Osteoporosis
About this trial
This is an interventional treatment trial for Evaluate the Safety and Efficacy of Denosumab in Pediatric Subjects With focused on measuring Denosumab, Pediatric GIOP, Glucocorticoid-induced, Osteoporosis
Eligibility Criteria
Inclusion Criteria:
- Male or female subjects, age 5 to 17 years, inclusive, at the time of informed consent.
- Clinical diagnosis of GiOP as defined by the following (and consistent with the International Society for Clinical Densitometry definition of osteoporosis in children and adolescents [Bishop et al, 2014])
- A confirmed diagnosis of non-malignant condition(s) requiring treatment with systemic GC (including, but not limited to, chronic rheumatologic, gastrointestinal, neurologic, respiratory, and/or nephrological conditions)
- Subjects who are on systemic GC only as replacement therapy for adrenal insufficiency are not eligible for the study - Treatment with systemic GC (intravenous or oral) of any duration for the underlying non-malignant condition(s) within the 12 months prior to screening
Evidence of at least 1 vertebral compression fracture of Genant Grade 1 or higher, as assessed by the central imaging vendor on lateral spine X-rays performed at screening or within 2 months prior to screening; OR, in the absence of vertebral compression fractures, presence of both clinically significant fracture history (ie, ≥ 2 long-bone fractures by age 10 years or ≥ 3 long-bone fractures at any age up to 17 years) and lumbar spine BMD Z-score ≤ -2.0, as assessed by the central imaging vendor.
- Subject's legally acceptable representative has provided informed consent when the subject is legally too young to provide informed consent and the subject has provided assent based on local regulations and/or guidelines prior to any study-specific activities/procedures being initiated
- Male or female subjects, age 5 to 17 years, inclusive, at the time of informed consent.
- Clinical diagnosis of GiOP as defined by the following (and consistent with the International Society for Clinical Densitometry definition of osteoporosis in children and adolescents [Bishop et al, 2014])
- A confirmed diagnosis of non-malignant condition(s) requiring treatment with systemic GC (including, but not limited to, chronic rheumatologic, gastrointestinal, neurologic, respiratory, and/or nephrological conditions)
- Subjects who are on systemic GC only as replacement therapy for adrenal insufficiency are not eligible for the study
- Treatment with systemic GC (intravenous or oral) of any duration for the underlying non malignant condition(s) within the 12 months prior to screening
- Prepubertal children should be expected to require significant GC use during the study, per investigator opinion
Exclusion criteria will include the following:
- Current hyperthyroidism (unless well controlled on stable antithyroid therapy)
- Current clinical hypothyroidism (unless well controlled on stable thyroid replacement therapy)
- History of hyperparathyroidism
- Current hypoparathyroidism
- Duchenne muscular dystrophy with symptomatic cardiac abnormality
- Current malabsorption
- Active infection or history of infections
History of malignancy
- Current hyperthyroidism (unless well controlled on stable antithyroid therapy)
- Current clinical hypothyroidism (unless well controlled on stable thyroid replacement therapy)
- History of hyperparathyroidism
- Current hypoparathyroidism
- Any causes of primary or secondary osteoporosis (other than GC use), or previous exposure to non-GC medications, which the investigator considers to have been a major factor contributing to the patient's fracture(s)
- Current adrenal insufficiency as the sole indication for GC therapy
- Duchenne muscular dystrophy with symptomatic cardiac abnormality
- Current malabsorption (in children with serum albumin -lower limit of normal [LLN], malabsorption should be clinically ruled out by the investigator to confirm eligibility)
- Known intolerance to calcium or vitamin D supplements
- Active infection or history of infections, defined as follows:
- Any active infection for which systemic anti-infectives were used within 4 weeks prior to screening
- Serious infection, defined as requiring hospitalization or intravenous anti infectives within 8 weeks prior to screening
- Recurrent or chronic infection or other active infection that, in the opinion of the investigator, might compromise the safety of the subject
Sites / Locations
- Childrens Hospital of Los Angeles
- AI Dupont Hospital for Children
- Indiana University Hospital
- University of Minnesota Masonic Childrens Hospital Discovery Clinic
- Metrohealth Medical Center
- Nationwide Childrens Hospital
- Perth Childrens Hospital
- Cliniques Universtaire Saint Luc Universite Catholique de Louvain
- Medical Centre Synexus Sofia EOOD
- Childrens Hospital of Eastern Ontario
- Centre Hospitalier Universitaire Sainte Justine
- Center for Clinical and Basic Research Colombia
- Solano y Terront Servicios Medicos Ltda - Unidad Integral de Endocrinologia Uniendo
- Foscal Internacional-Fundacion Oftalmologica de Santander
- Gandhi Medical College
- Sir Ganga Ram Hospital
- KLES Dr Prabhakar Kore Hospital and Medical Research Centre
- Christian Medical College
- Azienda Ospedaliera Universitaria Meyer
- Istituto Auxologico Italiano Istituto di Ricovero e Cura a Carattere Scientifico
- Azienda Ospedaliera Policlinico Umberto I
- RM Pharma Specialists SA de CV
- Instituto Nacional de Salud del Nino
- Centro Especializado de Enfermedades Neoplasicas
- Hospital Nacional Alberto Sabogal Sologuren
- Clinica Angloamericana
- Centro de Investigacion Ricardo Palma
- FSAI Scientific Center of Childrens Health of MoH of the RF
- SBEI of HPE First Moscow state medical university na I M Sechenov of MoH of Russian Federation
- SBHI of Novosibirsk region City Pediatric Clinical Hospital of Emergency Care
- LLC Medical Technologies
- Ankara Universitesi Tip Fakultesi
- Ataturk Universitesi Tip Fakultesi
- Marmara Universitesi Pendik Egitim Arastirma Hastanesi
- Ege Universitesi Tip Fakultesi
- CI Dnipropetrovsk Regional Children Clinical Hospital of Dnipropetrovsk Regional Council
- Communal Institution of Healthcare Kharkiv City Clinical Children Hospital 16
- National Childrens Specialized Hospital OHMATDYT
Arms of the Study
Arm 1
Arm 2
Other
Experimental
Placebo
Denosumab
SC Q6M placebo
1 mg/kg BW (up to a maximum of 60 mg) SC Q6M