Clinical trial NCT05276960: Efficacy of Intensive Cholecalciferol Monitoring and Supplementation on Serum vitD Levels in Pediatric Patients With CF
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
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
Lead sponsor: Hospital Infantil de Mexico Federico Gomez
Sponsor class: OTHER
First posted: 2022-03-14
Enrollment: 40
Sex: ALL
Minimum age (years): 5
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: 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
Primary purpose: TREATMENT

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.

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.

Eligibility criteria: Inclusion Criteria:

* Cystic Fibrosis

Exclusion Criteria:

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

Primary outcomes:
measure: Change in VitD Levels
timeFrame: Baseline, every 2 months through study completition, an avarage of 1 year.
Secondary outcomes:
measure: Changes in the number of Pulmonary exacerbations
timeFrame: 12 months
measure: Changes in the Bone mineral density
timeFrame: 12 months
Other outcomes:
Locations:
city: Mexico City
country: Mexico
facility: Hospital Infantil de México Federico Gómez
Study officials:
affiliation: Hospital Infantil de Mexico Federico Gomez
name: Magali R Reyes Apodaca, MSc
role: PRINCIPAL_INVESTIGATOR
affiliation: Hospital Infantil de Mexico Federico Gomez
name: Mara Medeiros, MD, Msc, PhD
role: STUDY_DIRECTOR
References: