Phase 2 N=26 Treatment

Safety, Efficacy, and Feasibility of High-dose Cholecalciferol in Pediatric Patients With Cystic Fibrosis

Vitamin D Deficiency

Bottom Line

View on ClinicalTrials.gov: NCT02613884 ↗

Enrolled (actual)

Serious AEs

15.4%

Results posted

Feb 2021

Primary outcome: Primary: Safety of a Single High-dose of Oral Cholecalciferol to Treat a Vitamin D Deficiency in Children With Cystic Fibrosis — 9.57; 9.47 mg/dL

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Treatment (Drug)
Age: Pediatric, Adult · 0+ yrs
Sex: All
Sponsor: Johns Hopkins All Children's Hospital
Primary completion: Nov 2019

Outcome Measures

Outcome	Result	p-value
PRIMARY Safety of a Single High-dose of Oral Cholecalciferol to Treat a Vitamin D Deficiency in Children With Cystic Fibrosis	9.57; 9.47	—
SECONDARY Efficacy of a Single High-dose of Oral Cholecalciferol in Treating a Vitamin D Insufficiency/Deficiency in Children With Cystic Fibrosis	26; 30; 27	—
SECONDARY Feasibility of Taking a 1-time High-dose of Cholecalciferol as Assessed by a 5-item Questionnaire	24; 2; 26; 0; 23; 3	—

Summary

Children with Cystic Fibrosis, ages greater than and equal to 36 months of age and less than or equal to 18 years of age, with a 25OHD level less than 30 ng/dL will be asked to participate in this study. Upon consent, they will be given oral cholecalciferol supplementation of 250,000 IU during their next CF clinic visit. The safety will be assessed by measuring a serum calcium level within 1 week of supplementation. Efficacy will be assessed using repeated 25OHD levels throughout the course of 12 months. Feasibility will be assessed with the previous two measures as well as a brief questionnaire administered via telephone within 1 week of supplementation.

Eligibility Criteria

Inclusion Criteria

Children with Cystic Fibrosis >36 months of age
Serum/blood 25OHD level < 30 ng/dL
Ability to provide valid informed consent to be a part of the study

Exclusion Criteria

Any history of kidney disease, kidney stones or on dialysis
Any history of hypercalcemia
Any history of hypercalciuria
Pregnancy at time of enrollment
Any history of parathyroid disorders
Inability to swallow pills by mouth

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02613884). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.