Daily Vitamin D for Sickle-cell Respiratory Complications
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 58
- 试验地点
- 1
- 主要终点
- Annual Rate of Respiratory Events
研究概览
简要总结
This study aims to answer the question whether daily oral vitamin D supplementation can reduce the risk of respiratory or lung complications in children and adolescents with sickle cell disease. Respiratory problems are the leading causes of sickness and of death in sickle cell disease. The investigators hypothesize that daily oral vitamin D3, compared to monthly oral vitamin D, will rapidly increase circulating vitamin D3, and reduce the rate of respiratory complications by 50% or more within the first year of supplementation in children and adolescents with sickle cell disease.
This study is funded by the FDA Office of Orphan Products Development (OOPD).
详细描述
This is a 2-year controlled, double-blind, randomized Phase 2 clinical trial comparing the efficacy in reducing the rate of respiratory events in sickle-cell disease of daily oral vitamin D3 (3,333 IU/d) with monthly bolus oral vitamin D3, (100,000 IU/mo) as a control. The scientific premise of the clinical trial is that circulating concentrations of vitamin D3, the parent compound, are the principal determinant of the anti-infective and immunomodulatory effects of supplementation.
Eligible participants will be initially screened to determine their blood vitamin D levels. Those with 25-hydroxyvitamin D levels between 5 and 60 ng/mL will be assigned by chance to one of the two arms for 24 months. Participants will be checked every month and will have periodic blood and urine tests to monitor for any side effects of the study treatments. Children above 5 y/o who can cooperate and understand the procedure will have lung function test at baseline and at 24 months. Showing that a monthly dose of vitamin D reduces lung infections, asthma and the acute chest syndrome could help establish this simple, low-cost treatment as a way to decrease sickness and deaths in children and adolescents with sickle-cell disease.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Prevention
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
Masking will be performed by the Research Pharmacy; all other research staff and participants will be blinded to allocation.
入排标准
- 年龄范围
- 3 Years 至 20 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of sickle cell disease (Hb SS, Hb SC, Hb S-Beta-thalassemia)
- •Age 3-20 years old
排除标准
- •Patient unwilling or unable to provide written informed consent (and assent, if applicable)
- •Patient unable or unwilling to comply with requirements of the clinical trial
- •Participation in another clinical trial
- •Current diagnosis of rickets
- •History of hypercalcemia or diagnosis of any medical condition associated with hypercalcemia, including primary hyperparathyroidism, malignancy, sarcoidosis, tuberculosis, granulomatous disease, familial hypocalciuric hypercalcemia
- •Current use of corticosteroids, excluding inhaled steroids
- •Current use of anticonvulsants (phenytoin, phenobarbital, carbamazepine)
- •Therapy with thiazide diuretics or lithium carbonate
- •Known liver or renal disease
- •Patients taking medications for pulmonary complications of sickle cell disease not on a stable dose of medications, as defined by a change in medications or doses within the three months prior to study entry
- •Patients on chronic red blood cell transfusion therapy
研究组 & 干预措施
Daily oral vitamin D3
Oral vitamin D3, 3,333 IU
干预措施: Daily oral vitamin D3, 3,333 IU (Drug)
Monthly bolus oral vitamin D3
Bolus oral vitamin D3, 100,000 IU
干预措施: Monthly oral vitamin D3, 100,000 IU (Drug)
Monthly bolus oral vitamin D3
Bolus oral vitamin D3, 100,000 IU
干预措施: Placebo oral tablet (Drug)
结局指标
主要结局
Annual Rate of Respiratory Events
时间窗: Month 12, Month 24
Respiratory events will be calculated as the sum of respiratory infection, asthma exacerbation, and acute chest syndrome, as ascertained by use of a validated questionnaire.
次要结局
- Mean Forced Vital Capacity (FVC % Predicted)(Baseline, Month 24)
- Forced Expiratory Volume in 1 Second (FEV1)(Baseline, Month 24)
- Forced Expiratory Volume in 1 Second (FEV1)/Forced Vital Capacity Ratio(Baseline, Month 24)
- Forced Expiratory Flow at 25%-75% Vital Capacity (FEF25-75, % Predicted)(Baseline, Month 24)
- Ratio of Residual Lung Volume (RV) to Total Lung Capacity (TLC)(Baseline, Month 24)
- Diffusing Capacity of the Lungs for Carbon Monoxide (DLCO)(Baseline, Month 24)
- Neutrophil Count(Baseline, Month 12, Month 24)
- Platelet Count(Baseline, Month 12, Month 24)
- Serum C-reactive Protein (CRP)(Baseline, Month 12, Month 24)
研究者
Margaret T. Lee
Professor of Pediatrics
Columbia University