Vitamin A in Sickle Cell Disease: Improving Sub-optimal Status With Supplementation
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 42
- 试验地点
- 2
- 主要终点
- Serum Vitamin A status
研究概览
简要总结
This study establishes the safety and efficacy of vit A supplementation doses (3000 and 6000 IU/d) over 8 weeks in children with SCD-SS, ages 9 and older and test the impact of vit A supplementation on key functional and clinical outcomes. Additionally, vitamin A status is assessed in healthy children ages 9 and older to compare to subjects with SCD-SS.
详细描述
Suboptimal vitamin A (vit A) status is prevalent in children with type SS sickle cell disease (SCD-SS) and associated with hospitalizations and poor growth and hematological status. Preliminary data in children with SCD-SS show that vit A supplementation at the dose recommended for healthy children failed to improve vit A status, resulting in no change in hospitalizations, growth or dark adaptation. This indicates an increased vit A requirement most likely due to chronic inflammation, low vit A intake and possible stool or urine loss. The dose of vit A needed to optimize vit A status in subjects with SCD-SS is unknown.
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
- 9 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Sickle cell disease, SS genotype (subjects with sickle cell disease only)
- •Usual state of good health (no hospitalizations, emergency room visits, or unscheduled acute illness clinic visits for two weeks prior to screening)
- •Commitment to a 119-day study (subjects with sickle cell disease only), or a 4-day study (healthy volunteers only)
排除标准
- •Hydroxyurea initiated within the previous 6 weeks (subjects with sickle cell disease only)
- •History of stroke (subjects with sickle cell disease only)
- •Other chronic conditions that may affect growth, dietary intake or nutritional status
- •Retinoic acid (topical or oral), weight loss medication and/or lipid lowering medications
- •Subjects with a BMI greater than 98th percentile for age and sex
- •Pregnant or lactating females (subjects who become pregnant during the course of the study will not continue participation)
- •Liver function tests >4 x upper limit of reference range
- •Participation in another study with impact on vitamin A status (subjects with sickle cell disease only)
- •Use of multi-vitamin or commercial nutritional supplements containing vitamin A (those who are willing to discontinue these supplements, with the approval of the medical care team, will be eligible for the study after a 1 month washout period. Subjects taking nutritional products without vitamin A will be eligible)
- •Inability to swallow pills (subjects with sickle cell disease only)
研究组 & 干预措施
Healthy Comparison Arm
Healthy subjects receive no intervention and undergo comparisons to the two vitamin A supplementation arms at baseline.
Lower Dose Vitamin A
Subjects with SCD-SS in the lower dose Vitamin A arm receive 3000IU of retinyl palmitate daily for 8 weeks.
干预措施: retinyl palmitate (Dietary Supplement)
Higher Dose Vitamin A
Subjects with SCD-SS in the higher dose Vitamin A arm receive 6000IU of retinyl palmitate daily for 8 weeks.
干预措施: retinyl palmitate (Dietary Supplement)
结局指标
主要结局
Serum Vitamin A status
时间窗: Change from baseline after supplementation for 8 weeks
Serum vitamin A as measured by retinol
次要结局
- Coefficient of fat absorption(Change from baseline after supplementation for 8 weeks)
- Hemoglobin(Change from baseline after supplementation for 8 weeks)
- Height Z-score(Change from baseline after supplementation for 8 weeks)
- Weight Z-score(Change from baseline after supplementation for 8 weeks)
- Muscle strength(Change from baseline after supplementation for 8 weeks)
- White blood cell count(Change from baseline after supplementation for 8 weeks)
- Upper limb strength(Change from baseline after supplementation for 8 weeks)
- Reticulocyte count(Change from baseline after supplementation for 8 weeks)
- Retinol binding protein, serum(Change from baseline after supplementation for 8 weeks)
- Muscle function(Change from baseline after supplementation for 8 weeks)
- White blood cell differential(Change from baseline after supplementation for 8 weeks)
- Vitamin A toxicity(Change from baseline after supplementation for 8 weeks)
- Upper arm muscle area(Change from baseline after supplementation for 8 weeks)
- Upper arm fat area(Change from baseline after supplementation for 8 weeks)
- Jump strength(Change from baseline after supplementation for 8 weeks)
- Retinol binding protein, urine(Change from baseline after supplementation for 8 weeks)
- Dietary Intake(Change from baseline after supplementation for 8 weeks)
- Hematocrit(Change from baseline after supplementation for 8 weeks)
- Fetal hemoglobin(Change from baseline after supplementation for 8 weeks)
- Fat Mass(Change from baseline after supplementation for 8 weeks)
- Mean corpuscular hemoglobin(Change from baseline after supplementation for 8 weeks)
- BMI Z-score(Change from baseline after supplementation for 8 weeks)
- Mean corpuscular volume(Change from baseline after supplementation for 8 weeks)
- Serum alanine aminotransferase(Change from baseline after supplementation for 8 weeks)
- Serum aspartate aminotransferase(Change from baseline after supplementation for 8 weeks)
- Serum bilirubin(Change from baseline after supplementation for 8 weeks)
- Tumor necrosis factor alpha(Change from baseline after supplementation for 8 weeks)
- Mean corpuscular hemoglobin concentration(Change from baseline after supplementation for 8 weeks)
- Urine creatinine(Change from baseline after supplementation for 8 weeks)
- Serum alkaline phosphatase(Change from baseline after supplementation for 8 weeks)
- High-sensitivity c-reactive protein(Change from baseline after supplementation for 8 weeks)
- Serum creatinine(Change from baseline after supplementation for 8 weeks)
- Serum gamma glutamyltransferase(Change from baseline after supplementation for 8 weeks)
- Lymphocyte subtypes(Change from baseline after supplementation for 8 weeks)
- Fat-free Mass(Change from baseline after supplementation for 8 weeks)