Osteoporosis in Cystic Fibrosis: Study of Bone Mass and Bone Metabolism, and Prospective Randomized Therapeutic Trial.
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 171
- 试验地点
- 11
- 主要终点
- Bone mineral density increase at lumbar spine.
研究概览
简要总结
Cystic fibrosis (CF) -- an autosomal recessive genetic disease affecting about 60,000 individuals worldwide, including about 3,800 in Italy -- is often associated with low bone mineral mass. The current aggressive therapies have ensured a much longer survival of CF patients but this has led to a higher frequency of osteoporosis and bone fractures, a serious problem which not only affects quality of life, but also hinders further therapeutic measures.
The aim of this study, conducted on a large group of children, adolescents and young adults with CF, has been the evaluation of bone mass changes after 1 year of a simple treatment with RDA-adjusted dietary calcium plus 25-OH vitamin D supplementation, and the feasibility and efficacy of alendronate treatment (for another year) in patients not responding to calcium + 25-OH vitamin D alone.
详细描述
The study included 2 phases.
Phase 1 (1-year open-label observational study): following baseline evaluation, bone mass changes have been studied with a simple therapy of adequate calcium intake and 25-OH vitamin D supplements in all eligible subjects (N=171).
Phase 2 (1-year double-blind, randomized, placebo-controlled, parallel group study): the 128 subjects showing an insufficient response to calcium + 25-OH vitamin D alone (bone mass increase <5%) at the end of Phase 1, were randomized into 2 groups and assigned to alendronate treatment (N=65) or placebo (N=63) (in addition to calcium and 25-OH vitamin D as during Phase 1).
The study has been carried out by the Coordinator's Institution (Istituto Auxologico Italiano)in collaboration with most Regional Reference Centers for CF in Italy.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 5 Years 至 30 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •age 2-30 years
- •clinically stable condition
- •regular menses in females
- •low Bone Mineral Apparent Density for age (defined as BMAD Z-score ≤-2.0 if age ≤18 years or ≤-2.5 if age >18 years).
排除标准
- •two or more episodes of hypercalcemia and/or hypercalciuria
- •contraindications to 25-OH vitamin D or alendronate treatment
- •recent transplantation
- •other diseases or medications (glucocorticoids excepted) associated with bone loss.
研究组 & 干预措施
Alendronate
128 subjects participated in the study's Phase 2 (1-year double-blind, randomized, placebo-controlled, parallel group study).
65 subjects were randomized to this arm. Oral alendronate dose: 5 mg/day, if body weight ≤25 kg; 10 mg/day, if body weight >25 kg.
干预措施: Alendronate (Drug)
Placebo
128 subjects participated in the study's Phase 2 (1-year double-blind, randomized, placebo-controlled, parallel group study).
63 subjects were randomized to this arm. Oral placebo (inactive pills).
干预措施: Placebo (Drug)
结局指标
主要结局
Bone mineral density increase at lumbar spine.
时间窗: up to 24 months
Bone mineral density evaluated by DXA. Bone mineral apparent density calculated to correct for bone size (growing subjects). Z-score calculated. Measurements: Phase 1 (171 subjects): Baseline, 6 months, 12 months. Phase 2 (128 subjects, randomized to 2 arms: placebo or alendronate): 18 months, 24 months.
次要结局
- Changes in bone turnover markers.(baseline and up to 24 months)
- Fracture rate.(at 12th and 24th month)
- Adverse effects of alendronate.(continuously throughout Phase 2 (2nd year of study))
研究者
Maria Luisa Bianchi
MD, First level manager
Istituto Auxologico Italiano