Accelerated Correction of Vitamin D Deficiency in Cholestatic Children: a Comparative Trial of Vitamin D2 Monotherapy vs Combination Therapy With Calcitriol
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 入组人数
- 54
- 试验地点
- 1
- 主要终点
- Change in Serum 25-Hydroxyvitamin D Level From Baseline to Month 3
研究概览
简要总结
The goal of this clinical trial is to learn whether adding calcitriol to vitamin D2 can improve vitamin D deficiency in children with cholestasis and chronic liver disease. Cholestasis is a condition in which bile flow is reduced, which can make it difficult for the body to absorb and process vitamin D. The study will also learn about the safety of using vitamin D2 together with calcitriol.
The main questions it aims to answer are:
- Does vitamin D2 plus calcitriol increase blood 25-hydroxyvitamin D (25-OHD) levels more than vitamin D2 alone after 3 months of treatment?
- Does vitamin D2 plus calcitriol help more children reach an adequate vitamin D level by 3 and 6 months?
- What medical problems, especially high calcium or high phosphorus levels, occur during treatment?
Researchers will compare children who receive vitamin D2 alone with children who receive vitamin D2 plus calcitriol to see which treatment improves vitamin D levels more effectively and safely.
Participants will:
- Take vitamin D2 alone or vitamin D2 plus calcitriol as assigned by randomization
- Visit the clinic for study assessments at the start of the study, at 3 months, and at 6 months
- Have blood tests to measure vitamin D levels, calcium, phosphorus, parathyroid hormone, liver function, and other safety markers
- Have their treatment reviewed at 3 months; participants whose vitamin D level remains low may have their treatment adjusted according to the study plan
- Bring back medication packages so researchers can check how regularly the study medicines were taken
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Double (Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- — 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients younger than 18 years of age.
- •Patients diagnosed with cholestasis, defined as direct/conjugated bilirubin >1 mg/dL for more than 1 month.
- •Patients diagnosed with chronic liver disease.
- •Patients with vitamin D deficiency, defined as serum 25-hydroxyvitamin D (25-OHD) level <20 ng/mL, according to the Endocrine Society Clinical Practice Guideline.
排除标准
- •Pre-existing hypercalciuria, screened by urine calcium testing before enrollment.
- •Patients with benign or malignant tumors.
- •Patients with renal tubular defects, screened by electrolyte testing before enrollment.
- •Patients currently receiving anticonvulsant therapy.
- •Patients who do not attend scheduled follow-up visits.
研究组 & 干预措施
Vitamin D2 Monotherapy
Participants in this arm will receive vitamin D2 40,000 IU orally once weekly for 3 months. At month 3, participants whose 25-hydroxyvitamin D level remains below 20 ng/mL may receive treatment adjustment according to the study protocol.
干预措施: Vitamin D2 (Drug)
Vitamin D2 Plus Calcitriol
Participants in this arm will receive vitamin D2 40,000 IU orally once weekly plus calcitriol 50 ng/kg/day orally for 3 months. At month 3, participants whose 25-hydroxyvitamin D level remains below 20 ng/mL may receive treatment adjustment according to the study protocol.
干预措施: Vitamin D2 (Drug)
Vitamin D2 Plus Calcitriol
Participants in this arm will receive vitamin D2 40,000 IU orally once weekly plus calcitriol 50 ng/kg/day orally for 3 months. At month 3, participants whose 25-hydroxyvitamin D level remains below 20 ng/mL may receive treatment adjustment according to the study protocol.
干预措施: Calcitriol (Drug)
结局指标
主要结局
Change in Serum 25-Hydroxyvitamin D Level From Baseline to Month 3
时间窗: Baseline to Month 3
Change in serum 25-hydroxyvitamin D (25-OHD) level, measured in ng/mL, from baseline to 3 months after treatment initiation. The change will be compared between participants receiving vitamin D2 monotherapy and those receiving vitamin D2 plus calcitriol.
Change From Baseline in Serum 25-Hydroxyvitamin D Concentration at Month 3
时间窗: Baseline and Month 3
Change from baseline in serum 25-hydroxyvitamin D \[25(OH)D\] concentration, measured in ng/mL, at 3 months after treatment initiation. The change will be calculated as the Month 3 value minus the baseline value and compared between treatment groups.
次要结局
- Proportion of Participants With Correction of Vitamin D Deficiency at Month 3(Month 3)
- Proportion of Participants With Correction of Vitamin D Deficiency at Month 6(Month 6)
- Change in Serum 25-Hydroxyvitamin D Level From Baseline to Month 6(Baseline to Month 6)
- Change in Serum Parathyroid Hormone Level(Change in serum parathyroid hormone (PTH) level from baseline to Month 3 and Month 6.)
- Change in Serum 1,25-Dihydroxyvitamin D Level(baseline , Month 3, Month 6)
- Incidence of Hypercalcemia(Baseline to Month 6)
- Change in Pediatric End-Stage Liver Disease Score(Baseline, Month 3, and Month 6)
- Change in Liver Function Tests(Baseline, Month 3, and Month 6)
- Number of Participants With Correction of Vitamin D Deficiency at Month 3(Month 3)
- Number of Participants With Correction of Vitamin D Deficiency at Month 6(Month 6)
- Change From Baseline in Serum Parathyroid Hormone Concentration at Month 3 and Month 6(Baseline, Month 3, and Month 6)
- Change From Baseline in Serum 1,25-Dihydroxyvitamin D Concentration at Month 3 and Month 6(baseline , Month 3, Month 6)
- Number of Participants With Hypercalcemia During the 6-Month Study Period(Baseline to Month 6)
- Change From Baseline in Pediatric End-Stage Liver Disease Score at Month 3 and Month 6(Baseline, Month 3, and Month 6)
- Change From Baseline in Serum Alanine Aminotransferase Concentration at Month 3 and Month 6(Baseline, Month 3, and Month 6)
- Change From Baseline in Serum Aspartate Aminotransferase Concentration at Month 3 and Month 6(Baseline, Month 3, and Month 6)
- Change From Baseline in Serum Alkaline Phosphatase Concentration at Month 3 and Month 6(Baseline, Month 3, and Month 6)
- Change From Baseline in Serum Total Bilirubin Concentration at Month 3 and Month 6(Baseline, Month 3, and Month 6)
- Change From Baseline in Serum Direct Bilirubin Concentration at Month 3 and Month 6(Baseline, Month 3, and Month 6)
- Change From Baseline in International Normalized Ratio at Month 3 and Month 6(Baseline, Month 3, and Month 6)
- Change From Baseline in Serum Albumin Concentration at Month 3 and Month 6(Baseline, Month 3, and Month 6)