Prospective Intervention Study on Vitamin D in Patients With Cystic Fibrosis

Not Applicable

Completed

• Conditions: Cystic Fibrosis
• Interventions: Dietary Supplement: Supplementation with vitamin D2/D3

• Registration Number: NCT01321905
• Lead Sponsor: Karolinska Institutet

Brief Summary

The vast majority of Cystic Fibrosis (CF) patients worldwide are vitamin D insufficient. There is no evidence of benefit of vitamin D supplementation for CF patients yet. However, descriptive cross-sectional studies suggest that vitamin D might be beneficial with respect to bone health, as well as to the newly described "non-classical" functions of vitamin D such as the potential anti-diabetic and immunomodulatory effects. To prove causation, and to determine which serum vitamin D concentration is optimal for CF patients, vitamin D supplementation interventional studies are needed, such as our trial.

Detailed Description

Not available

Study Timeline

• Study Start: 2010-04
• Study First Submitted: 2011-03-23
• Study First Submitted QC: 2011-03-23
• Study First Posted: 2011-03-24
• Primary Completion: 2011-07
• Study Completion: 2011-07
• Status Verified: 2024-10
• Last Update Submitted: 2024-10-11
• Last Update Posted: 2024-10-15

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 16

Inclusion Criteria

• Established diagnosis of cystic fibrosis
• Age 6 years and more
• Serum 25-hydroxy vitamin D concentration at the latest visit < 75 nmol/L

Exclusion Criteria

• Pregnancy or lactation
• Established diagnosis of CF-related diabetes
• CF-related liver disease
• Status post transplantation (lung, liver or other)
• Long-term corticosteroid treatment per os
• Hypercalcaemia or kidney stones
• Use of tanning beds more often than once a month
• At inclusion, plans to travel to a sunny location for more than 1 week during the study period
• Any known disorders of the endocrine system affecting vitamin D metabolism (hyperparathyroidism, malignancy, advanced renal disease)
• Inclusion into another study testing immunomodulatory substances

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Ergocalciferol	Supplementation with vitamin D2/D3	Patients younger than 16 years of age are administered 35,000 IU ergocalciferol per week divided into doses 5000 IU per day as a starting dose that is further adjusted by serum 25-hydroxy vitamin D concentration monitoring. Patients 16 or more years of age are administered 50,000 IU ergocalciferol per week divided into doses 7150 IU per day as a starting dose that is further adjusted by serum 25-hydroxy vitamin D concentration monitoring.
Cholecalciferol	Supplementation with vitamin D2/D3	Patients younger than 16 years of age are administered 35,000 IU cholecalciferol per week divided into doses 5000 IU per day as a starting dose that is further adjusted by serum 25-hydroxy vitamin D concentration monitoring. Patients 16 or more years of age are administered 50,000 IU cholecalciferol per week divided into doses 7150 IU per day as a starting dose that is further adjusted by serum 25-hydroxy vitamin D concentration monitoring.

Primary Outcome Measures

Name	Time	Method
Serum 25-hydroxy vitamin D	3 months	This pilot study is primarily designed for establishing effective vitamin D dosing in our specific patient population, and only secondarily designed (and thus, not powered for) for the secondary outcome measures. The results of this study will make it possible for the first time to power the follow-up long-term study for some of the secondary outcome measures followed in this pilot study, some of which might therefore become primary outcome measures in the follow-up study.

Secondary Outcome Measures

Name	Time	Method
Parathyroid hormone (PTH)	3 months	As a surrogate marker of bone health
Inflammatory parameters	3 months	Cytokine profiles, antimicrobial peptides, peripheral blood mononuclear cell profiles, immunoglobulines, acute phase markers, sedimentation rate and other
Infection parameters	3 months	Number of days on intravenous antibiotic treatment; number of infectious episodes; number of common cold episodes; relative number of sputum samples positive for pathological bacteria; and other
Lung function parameters	3 months	FEV1, FVC, PEF, FEF25, FEF50, FEF75 and other
Glucose tolerance parameters	3 months	Insulin, C-peptide, glucagon, fasting plasma glucose, HbA1c, 3-hour 75-g oral glucose tolerance test
Adherence with vitamin D treatment	3 months	Semi-quantitative assessment by a questionnaire (thus, a patient-reported outcome)
Disease-specific quality of life	3 months	Assessment using "CFQ-R" questionnaire, specifically designed to measure the quality of life in CF patients
Plasma calcium	3 months	Proportion of patients with albumin-corrected serum calcium increasing to a concentration greater than 2,75 mmol/L in patients with no hypercalcaemia before vitamin D supplementation was started.
Relative number of patients reaching high abnormal 25(OH)D concentrations	3 months	Proportion of patients in the intervention arms reaching 25(OH)D \>250 nmol/L
Proportion of patients reaching toxic 25(OH)D concentrations	3 months	Proportion of patients in the intervention arms reaching 25(OH)D \>375 nmol/L
Proportion of patients with suspect hypercalcaemia symptoms	3 months	Proportion of patients with suspect hypercalcaemia symptoms in the intervention arms

Trial Locations

Locations (1)

Stockholm Cystic Fibrosis Center, Karolinska University Hospital Huddinge; 🇸🇪 Stockholm, Sweden