Pilot study to assess the efficacy of a therapy with the RANKL-antibody denosumab in children 5-10 years of age with mutation in COL1A1 or COL1A2 leading to Osteogenesis imperfecta. Efficacy will be assessed by DXA measurements at the lumbar spine of the areal bone mineral density (BMD) which is the most frequently used parameter in trials investigating osteoporosis. The hypothesis of the study is: Osteoclastic activity which is increased in OI could be reduced by inhibition of osteoclast maturation. Denosumab inhibits maturation of the osteoclasts by inhibiting RANKL. BMD could be increased during a 36 week treatment course with denosumab measured after 48 weeks.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
10
Denosumab will be given subcutaneously in a dosage of 1mg/kg body weight every 12 weeks. 4 interventions are planned until trial week 36. There is no control group planned.
University Cologne, Childrens Hospital, Cologne, Germany
Cologne, North Rhine-Westphalia, Germany
Changes of bone mineral density (BMD [g/cm2]) in lumbar spine after 36 weeks of treatment with denosumab. Changes will be calculated between baseline and study week 48.
Time frame: 48 weeks
Decrease of osteoclastic activity measured by urinary deoxypyridinoline (DPD).
Time frame: 14 days (DPD)
Parathormone in study week 12, 24, 36 and 48 compared to baseline.
Descriptive statistical analysis
Time frame: 12 weeks
N-Telopeptides in study week 12, 24, 36 and 48 compared to baseline.
descriptive statistical analysis
Time frame: 12 weeks
Osteocalcin in study week 12, 24, 36 and 48.
descriptive statistical analysis
Time frame: 12 weeks
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