TRANSLATIONAL THERAPY IN PATIENTS WITH OSTEOGENESIS IMPERFECTA - A PILOT TRIAL ON TREATMENT WITH THE RANKL-ANTIBODY DENOSUMAB
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- 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.
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 5 Years 至 11 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- •Hypocalcemia (<1.03 mmol/l ionized Calcium)
- •Subjects with reduced renal function (estimated GFR (Schwartz formula) <30ml/min/1.73m2)
- •Any other abnormal finding such as physical examination or laboratory evaluation, in the opinion of the investigator that is indicative of a disease that would compromise the safety of the patient when getting denosumab s.c.
研究组 & 干预措施
Denosumab subcutaneously
干预措施: Denosumab (Drug)
结局指标
主要结局
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.
时间窗: 48 weeks
次要结局
- Decrease of osteoclastic activity measured by urinary deoxypyridinoline (DPD).(14 days (DPD))
- Parathormone in study week 12, 24, 36 and 48 compared to baseline.(12 weeks)
- N-Telopeptides in study week 12, 24, 36 and 48 compared to baseline.(12 weeks)
- Osteocalcin in study week 12, 24, 36 and 48.(12 weeks)
研究者
Dr. med. Joerg Oliver Semler
Head of the outpatient center for sceletal dysplasias
University of Cologne