Efficacy of Intensive Cholecalciferol Monitoring and Supplementation on Serum vitD Levels in Pediatric Patients With CF

A Randomized, Double-blind Study of the Efficacy of Intensive Cholecalciferol Monitoring and Supplementation on Serum Vitamin D Levels in Pediatric Patients With Cystic Fibrosis

Cystic fibrosis (CF) is an autosomal recessive disease caused by alterations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene, characterized by multisystemic alterations, mainly in the lung, intestine, sweat, and bile ducts. In addition to pulmonary involvement, the presence of exocrine pancreatic insufficiency also increases the risk of survival, as it is associated with malnutrition and deficiency of fat-soluble vitamins, such as vitamin D.

Vitamin D, in addition to its role in bone health, in the case of CF patients with chronic inflammation, it has been suggested that many of the cytokines that regulate the inflammatory response contain elements that respond to vitamin D, so vitamin D could play an essential role in the regulation of the inflammatory response in CF, which could favor lung function.

However, more than 50% of CF patients present vitamin D insufficiency or deficiency, despite the different schemes suggested for supplementation in different age groups, which suggests that new strategies are needed to normalize vitamin D levels, which will allow us to see its clinical effect on the inflammatory response, by decreasing the number of exacerbations and thus perpetuating or improving lung function, as well as on bone mineral health.

Study Overview

• Status: Completed
• Conditions: Vitamin D Deficiency; Cystic Fibrosis; Cystic Fibrosis Pulmonary Exacerbation; Cystic Fibrosis in Children; Bone Density, Low; Inflammation Chronic
• Intervention / Treatment: Drug: Cholecalciferol Pill

Detailed Description

Randomized double-blind clinical trial in patients aged 5 to 18 years attending the CF Comprehensive Care clinic in Mexico City. The intervention group will receive an intensification of vitamin D supplementation, with increments of 4000 IU depending on serum levels, the control group will receive supplementation according to the treatment guidelines of the Cystic Fibrosis Foundation, and the dose will be doubled depending on serum levels. In both groups vitamin D levels will be measured at 3, 6, 9, and 12 months, spirometry and sputum culture will also be performed to evaluate the pulmonary function, densitometry to evaluate the bone mineral density and body composition, together with anthropometric evaluation and quantitative bone ultrasound at each visit, in the biochemical evaluation, calcium/creatinine ratio and alkaline phosphatase will be taken at each visit.

• Study Type: Interventional
• Enrollment (Actual): 40
• Phase: Phase 4

Contacts and Locations

Study Locations

• Mexico
  • Mexico City, Mexico, 06700
    • Hospital Infantil de Mexico Federico Gomez

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years to 18 years (Child, Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Cystic Fibrosis

Exclusion Criteria:

• Another chronic disease (HIV, cancer, renal failure)
• 25-OH-VitD levels < 10 ng/ml or > 30 ng/ml

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Regular supplementation; Baseline dose of 2000 IU of vitamin D3, based on Cystic Fibrosis Foundation (CFF) treatment guidelines. According to serum vitamin D levels, 2000 IU increments will be performed whenever 25-OH-VitD (25-hydroxy vitamin D) values < 30 ng/ml are found.	Drug: Cholecalciferol Pill; Dose increments according to serum levels determined every 2 months
Experimental: Enhanced Supplementation; Basal dose of 4000 IU of vitamin D3. According to serum vitamin D levels, increments of 4000 IU will be made each time 25-OH-VitD values < 30 ng/ml are found.	Drug: Cholecalciferol Pill; Dose increments according to serum levels determined every 2 months

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in VitD Levels	Serum vitamin D levels up to 30 ng/ml	Baseline, every 2 months through study completition, an avarage of 1 year.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes in the number of Pulmonary exacerbations	Number of exacerbations present during 1 year prior and 1 year after the baseline	12 months
Changes in the Bone mineral density	changes in bone mineral content at baseline and at the end of the study	12 months

Collaborators and Investigators

• Sponsor: Hospital Infantil de Mexico Federico Gomez
• Investigators: Principal Investigator: Magali R Reyes Apodaca, MSc, Hospital Infantil de Mexico Federico Gomez; Study Director: Mara Medeiros, MD, Msc, PhD, Hospital Infantil de Mexico Federico Gomez

Study record dates

Study Major Dates

• Study Start (Actual): June 22, 2022
• Primary Completion (Actual): August 30, 2024
• Study Completion (Actual): January 30, 2025

Study Registration Dates

• First Submitted: February 21, 2022
• First Submitted That Met QC Criteria: March 2, 2022
• First Posted (Actual): March 14, 2022

Study Record Updates

• Last Update Posted (Actual): April 8, 2025
• Last Update Submitted That Met QC Criteria: April 4, 2025
• Last Verified: April 1, 2025

More Information

Terms related to this study

• Keywords: cystic fibrosis; vitamin D; pulmonary exacerbation; Vitamin D Deficiency or insufficiency
• Additional Relevant MeSH Terms: Bone Diseases; Musculoskeletal Diseases; Pathologic Processes; Nutrition Disorders; Genetic Diseases, Inborn; Metabolic Diseases; Respiratory Tract Diseases; Digestive System Diseases; Lung Diseases; Infant, Newborn, Diseases; Pancreatic Diseases; Lung Diseases, Interstitial; Avitaminosis; Deficiency Diseases; Malnutrition; Inflammation; Pulmonary Fibrosis; Fibrosis; Vitamin D Deficiency; Cystic Fibrosis; Bone Diseases, Metabolic; Calcium-Regulating Hormones and Agents; Physiological Effects of Drugs; Bone Density Conservation Agents; Micronutrients; Vitamins; Vitamin D; Cholecalciferol
• Other Study ID Numbers: HIM-2021-087

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No