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Completed

NCT Number: NCT04170348

Daily Vitamin D for Sickle-cell Respiratory Complications

This study aims to answer the question whether daily oral vitamin D supplementation can reduce the risk of respiratory or lung complications in children and adolescents with sickle cell disease. Respiratory problems are the leading causes of sickness and of death in sickle cell disease. The investigators hypothesize that daily oral vitamin D3, compared to monthly oral vitamin D, will rapidly increase circulating vitamin D3, and reduce the rate of respiratory complications by 50% or more within the first year of supplementation in children and adolescents with sickle cell disease.

This study is funded by the FDA Office of Orphan Products Development (OOPD).

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Key information

Conditions

Sickle Cell Disease Acute Chest Syndrome Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Asthma Avitaminosis Bronchial Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Deficiency Diseases Deficiency Diseases Vitamin Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Hypersensitivity Hypersensitivity, Immediate Immune System Diseases Infections Lung Diseases Lung Diseases, Obstructive Malnutrition Nutrition Disorders Nutritional and Metabolic Diseases Respiration Disorders Respiratory Hypersensitivity Respiratory Tract Diseases Respiratory Tract Infections Vitamin D Deficiency

Age range

3 year–20 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Columbia University Medical Center

New York, 10032, United States

About this study

This is a 2-year controlled, double-blind, randomized Phase 2 clinical trial comparing the efficacy in reducing the rate of respiratory events in sickle-cell disease of daily oral vitamin D3 (3,333 IU/d) with monthly bolus oral vitamin D3, (100,000 IU/mo) as a control. The scientific premise of the clinical trial is that circulating concentrations of vitamin D3, the parent compound, are the principal determinant of the anti-infective and immunomodulatory effects of supplementation.

Eligible participants will be initially screened to determine their blood vitamin D levels. Those with 25-hydroxyvitamin D levels between 5 and 60 ng/mL will be assigned by chance to one of the two arms for 24 months. Participants will be checked every month and will have periodic blood and urine tests to monitor for any side effects of the study treatments. Children above 5 y/o who can cooperate and understand the procedure will have lung function test at baseline and at 24 months. Showing that a monthly dose of vitamin D reduces lung infections, asthma and the acute chest syndrome could help establish this simple, low-cost treatment as a way to decrease sickness and deaths in children and adolescents with sickle-cell disease.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Diagnosis of sickle cell disease (Hb SS, Hb SC, Hb S-Beta-thalassemia)
  • Age 3-20 years old

Exclusion criteria

  • Patient unwilling or unable to provide written informed consent (and assent, if applicable)
  • Patient unable or unwilling to comply with requirements of the clinical trial
  • Participation in another clinical trial
  • Current diagnosis of rickets
  • History of hypercalcemia or diagnosis of any medical condition associated with hypercalcemia, including primary hyperparathyroidism, malignancy, sarcoidosis, tuberculosis, granulomatous disease, familial hypocalciuric hypercalcemia
  • Current use of corticosteroids, excluding inhaled steroids
  • Current use of anticonvulsants (phenytoin, phenobarbital, carbamazepine)
  • Therapy with thiazide diuretics or lithium carbonate
  • Known liver or renal disease
  • Patients taking medications for pulmonary complications of sickle cell disease not on a stable dose of medications, as defined by a change in medications or doses within the three months prior to study entry
  • Patients on chronic red blood cell transfusion therapy

Treatment and study plan

Daily oral vitamin D3, 3,333 IU

Drug

Oral vitamin D3, 3,333 IU, will be administered daily.

Other names: Cholecalciferol for oral administration

Monthly oral vitamin D3, 100,000 IU

Drug

Oral vitamin D3, 100,000 IU, will be administered monthly.

Other names: Cholecalciferol for oral administration

Placebo oral tablet

Drug

Participants randomized to receive once monthly oral bolus of vitamin D3, will receive placebo on all other days of the month.

Primary outcomes

  1. Annual Rate of Respiratory Events

    Time frame: Month 12, Month 24

    Respiratory events will be calculated as the sum of respiratory infection, asthma exacerbation, and acute chest syndrome, as ascertained by use of a validated questionnaire.

Secondary outcomes

  1. Mean Forced Vital Capacity (FVC % Predicted)

    Time frame: Baseline, Month 24

    This is to measure the forced vital capacity (FVC; % predicted) at baseline and at month 24.

    Forced Vital Capacity (FVC) is a key measure of lung function that indicates the total volume of air a person can forcefully exhale after taking a deep breath. It is calculated using spirometry, which assesses lung capacity and helps diagnose respiratory conditions. Predicted FVC: The FVC is compared to predicted values based on age, height, and sex to determine if it is within the normal range (80% or more of predicted). Interpretation: A low FVC may indicate obstruction (e.g., asthma or COPD), while a high FVC may suggest restriction in lung function. Baseline Measurement: It is essential to establish a baseline FVC to monitor changes over time and assess the effectiveness of treatments. For accurate interpretation, it is crucial to compare FVC results with other measurements, such as FEV1, to identify the presence of obstructive or restrictive lung disease.

  2. Forced Expiratory Volume in 1 Second (FEV1)

    Time frame: Baseline, Month 24

    Forced Expiratory Volume in 1 second (FEV1; % predicted) at baseline and at month 24.

  3. Forced Expiratory Volume in 1 Second (FEV1)/Forced Vital Capacity Ratio

    Time frame: Baseline, Month 24

    Forced Expiratory Volume in 1 second (FEV1; % predicted)/Forced Vital Capacity (FVC) [FEV1/FVC] % predicted at baseline and month 24

  4. Forced Expiratory Flow at 25%-75% Vital Capacity (FEF25-75, % Predicted)

    Time frame: Baseline, Month 24

    Forced Expiratory Flow at 25%-75% vital capacity (FEF25-75) % predicted at baseline and month 24 .

  5. Ratio of Residual Lung Volume (RV) to Total Lung Capacity (TLC)

    Time frame: Baseline, Month 24

    Ratio of Residual Lung Volume (RV) to Total Lung Capacity (RV/TLC) at baseline and month 24.

  6. Diffusing Capacity of the Lungs for Carbon Monoxide (DLCO)

    Time frame: Baseline, Month 24

    Diffusing Capacity of the Lungs for Carbon Monoxide (DLCO; % predicted) at baseline and month 24

  7. Neutrophil Count

    Time frame: Baseline, Month 12, Month 24

    Blood Neutrophil Count in percentage at baseline, month 12 and month 24

  8. Platelet Count

    Time frame: Baseline, Month 12, Month 24

    Blood Platelet Count (Platelets*10^3/ per μL) at baseline, month 12 and month 24

  9. Serum C-reactive Protein (CRP)

    Time frame: Baseline, Month 12, Month 24

    Serum C-reactive protein (CRP; mg/L) at baseline, month 12 and month 24

Sponsors and collaborators

Lead sponsor

Columbia University

Other

Collaborators

  • FDA Office of Orphan Products Development

Registry information

Acronym: ViDAS-2

Important dates

Study start
2020
Primary completion
2024
Study completion
2024
First posted
Nov 20, 2019
Registry last updated
Sep 24, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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