Children, Cholestatic Liver Disease, Chronic Liver Disease (CLD), Vitamin D Deficiency
Conditions
Keywords
cholestasis, pediatrics, vitamin d deficiency, Calcitriol, 25-Hydroxyvitamin D2
Brief summary
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
Interventions
DRUGVitamin D2
Vitamin D2 40,000 IU/week weekly
DRUGCalcitriol
Calcitriol 50 ng/kg/day once daily
Sponsors
Chulalongkorn University
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
DOUBLE (Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
No minimum to 18 Years
Healthy volunteers
No
Inclusion criteria
* 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.
Exclusion criteria
* 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Serum 25-Hydroxyvitamin D Concentration at Month 3 | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Correction of Vitamin D Deficiency at Month 3 | Month 3 | Number and percentage of participants with serum 25-hydroxyvitamin D \[25(OH)D\] concentration greater than or equal to 20 ng/mL at Month 3. |
| Number of Participants With Correction of Vitamin D Deficiency at Month 6 | Month 6 | Number and percentage of participants with serum 25-hydroxyvitamin D \[25(OH)D\] concentration greater than or equal to 20 ng/mL at Month 6. |
| Change in Serum 25-Hydroxyvitamin D Level From Baseline to Month 6 | Baseline to Month 6 | Change in serum 25-hydroxyvitamin D (25-OHD) level, measured in ng/mL, from baseline to 6 months after treatment initiation. |
| 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 parathyroid hormone concentration, measured in pg/mL, at Month 3 and Month 6. The change will be calculated as the follow-up value minus the baseline value. |
| Change From Baseline in Serum 1,25-Dihydroxyvitamin D Concentration at Month 3 and Month 6 | baseline , Month 3, Month 6 | Change from baseline in serum 1,25-dihydroxyvitamin D concentration, measured in pg/mL, at Month 3 and Month 6. The change will be calculated as the follow-up value minus the baseline value. |
| Number of Participants With Hypercalcemia During the 6-Month Study Period | Baseline to Month 6 | Number and percentage of participants who develop hypercalcemia during the 6-month study period. Hypercalcemia is defined as albumin-corrected serum calcium above the age-specific upper limit of normal according to the study laboratory reference range. |
| 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 the Pediatric End-Stage Liver Disease (PELD) score at Month 3 and Month 6. The PELD score is calculated using serum bilirubin, international normalized ratio, serum albumin, age less than 1 year, and growth failure. The PELD score has no fixed absolute minimum or maximum possible value and may range from negative to positive values. Higher scores indicate more severe liver disease and a higher risk of short-term mortality/transplant priority. |
| 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 alanine aminotransferase (ALT) concentration, measured in U/L, at Month 3 and Month 6. The change will be calculated as the follow-up value minus the baseline value. |
| 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 aspartate aminotransferase (AST) concentration, measured in U/L, at Month 3 and Month 6. The change will be calculated as the follow-up value minus the baseline value. |
| 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 alkaline phosphatase concentration, measured in U/L, at Month 3 and Month 6. The change will be calculated as the follow-up value minus the baseline value. |
| 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 total bilirubin concentration, measured in mg/dL, at Month 3 and Month 6. The change will be calculated as the follow-up value minus the baseline value. |
| Change From Baseline in Serum Direct Bilirubin Concentration at Month 3 and Month 6 | Baseline, Month 3, and Month 6 | Change from baseline in serum direct bilirubin concentration, measured in mg/dL, at Month 3 and Month 6. The change will be calculated as the follow-up value minus the baseline value. |
| Change From Baseline in International Normalized Ratio at Month 3 and Month 6 | Baseline, Month 3, and Month 6 | Change from baseline in international normalized ratio (INR) at Month 3 and Month 6. The change will be calculated as the follow-up value minus the baseline value. Higher INR values indicate worse coagulation function. |
| Change From Baseline in Serum Albumin Concentration 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. The change will be calculated as the follow-up value minus the baseline value. Lower serum albumin values indicate worse hepatic synthetic function. |
Countries
Thailand
Contacts
CONTACTTHANAPORN NiLYAEM
Outcome results
None listed