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

NCT02613884 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: 128487
• Study Type: interventional
• Target: 26
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Vitamin D Deficiency

Outcome Measures

Primary Outcomes (1)

• Safety of a Single High-dose of Oral Cholecalciferol to Treat a Vitamin D Deficiency in Children With Cystic Fibrosis

  The safety of a single high-dose of oral cholecalciferol will be assessed using a serum calcium measurement after administration of treatment. Instances of hypercalcemia will be assessed at 1 week and at 3 months post-treatment. The treatment will be considered to be safe if the serum calcium level does not exceed 14 mg/dL.

  1 week, 3 months

Secondary Outcomes (2)

• Efficacy of a Single High-dose of Oral Cholecalciferol in Treating a Vitamin D Insufficiency/Deficiency in Children With Cystic Fibrosis

  3 months, 6 months and 12 months

• Feasibility of Taking a 1-time High-dose of Cholecalciferol as Assessed by a 5-item Questionnaire

  1 week

Study Arms (1)

Treatment

EXPERIMENTAL

All patients with a 25OHD level \<30 ng/dL will be given 250,000 IU D3 (cholecalciferol) orally at one point in time and during CF clinic.

Drug: Treatment

Interventions

Treatment DRUG

Treatment

Eligibility Criteria

• Age: 36 Months - 18 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Johns Hopkins All Children's Hospital: lead

Study Sites (1)

Johns Hopkins All Children's Hospital

St. Petersburg, Florida, 33701, United States

Location

MeSH Terms

Conditions

Vitamin D Deficiency

Interventions

Therapeutics

Condition Hierarchy (Ancestors)

Avitaminosis; Deficiency Diseases; Malnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Vanessa Millovich
• Organization: Kate Farms

vannanole@gmail.com

3522634409

Study Officials

• Deanna Green, MD

  Johns Hopkins All Children's Hospital

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 20, 2015
• First Posted: November 25, 2015
• Study Start: November 1, 2016
• Primary Completion: November 25, 2019
• Study Completion: November 25, 2019
• Last Updated: February 3, 2021
• Results First Posted: February 3, 2021

Record last verified: 2021-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)