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

NCT05276960 | Completed Phase 4 DMC

• 1 other identifier: HIM-2021-087
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Vitamin D Deficiency; Cystic Fibrosis; Bone Density, Low; Inflammation Chronic; Cystic Fibrosis in Children; Cystic Fibrosis Pulmonary Exacerbation

Keywords

vitamin D; cystic fibrosis; pulmonary exacerbation; Vitamin D Deficiency or insufficiency

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (2)

• Changes in the number of Pulmonary exacerbations

  12 months

• Changes in the Bone mineral density

  12 months

Study Arms (2)

Regular supplementation

ACTIVE COMPARATOR

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

Enhanced Supplementation

EXPERIMENTAL

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

Interventions

Cholecalciferol Pill DRUG

Dose increments according to serum levels determined every 2 months

Enhanced Supplementation; Regular supplementation

Eligibility Criteria

• Age: 5 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Cystic Fibrosis

You may not qualify if:

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

Sponsors & Collaborators

• Hospital Infantil de Mexico Federico Gomez: lead

Study Sites (1)

Hospital Infantil de México Federico Gómez

Mexico City, Mexico City, 06700, Mexico

Location

MeSH Terms

Conditions

Vitamin D Deficiency; Cystic Fibrosis; Bone Diseases, Metabolic

Interventions

Cholecalciferol

Condition Hierarchy (Ancestors)

Avitaminosis; Deficiency Diseases; Malnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases; Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases; Bone Diseases; Musculoskeletal Diseases; Metabolic Diseases

Intervention Hierarchy (Ancestors)

Cholestenes; Cholestanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Sterols; Vitamin D; Secosteroids; Membrane Lipids; Lipids

Study Officials

• Magali R Reyes Apodaca, MSc

  Hospital Infantil de Mexico Federico Gomez

  PRINCIPAL INVESTIGATOR

• Mara Medeiros, MD, Msc, PhD

  Hospital Infantil de Mexico Federico Gomez

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Masking Details: For the blinding of the maneuver, supplementation will be done with cholecalciferol (vitD3) in dispersible tablets of the same size and color, with doses of 2000 IU (0.05 mg) for the control group and 4000 IU (0.1 mg) for the intervention group. The capsules should be dissolved in 30 ml of water. The dispensing of the medication according to the intervention group and vitamin D levels will be performed by the investigator assigned to blinding of the principal investigator, the containers of the tablets will be of the same color and size and only the investigators responsible for the dispensing will know the corresponding dose
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Model Details: Pediatric CF patients will be recruited from the CF Integrated Care Clinic of the Federico Gomez Children's Hospital of Mexico. Once at the clinic they will be invited to participate in the study. Parents or guardians of patients under 8 years of age will be asked to sign the informed consent form before starting the studies, procedures and intervention. For those patients older than 8 years, in addition to parental consent, they will be given information and asked to sign the informed consent form before starting the studies, procedures, and intervention. The restrictive randomization will be performed by blocks of 8,obtained through the web page https://www.randomizer.org/, and will be carried out by one of the investigators, who will also be responsible for its safekeeping. The concealment will be carried out by the same investigator in charge of the randomization, for which the treatment groups will be placed in metallic gray, non-transparent, previously labeled envelopes

Study Record Dates

• First Submitted: February 21, 2022
• First Posted: March 14, 2022
• Study Start: June 22, 2022
• Primary Completion: August 30, 2024
• Study Completion: January 30, 2025
• Last Updated: April 8, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

ME (1)