Cystic Fibrosis
Conditions
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.
Interventions
DIETARY_SUPPLEMENTSupplementation with vitamin D2/D3
Patients younger than 16 years of age are administered 35,000 IU ergo-/chole-calciferol 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. The intervention time is 3 months, followed by 2-months wash-out period when patients do not take any more extra vitamin D but they are still monitored. Patients 16 or more years of age are administered 50,000 IU ergo-/chole-calciferol 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.
Sponsors
Region Stockholm
Karolinska Institutet
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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) |
| Parathyroid hormone (PTH) | 3 months | As a surrogate marker of bone health |
| 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 |
| Disease-specific quality of life | 3 months | Assessment using CFQ-R questionnaire, specifically designed to measure the quality of life in CF patients |
Countries
Sweden
Outcome results
None listed