Sickle Cell DiseasePhase 2ViDAS-2NCT04170348
Daily Vitamin D for Sickle-cell Respiratory Complications
A Phase 2 study testing Daily oral vitamin D3, 3,333 IU and Monthly oral vitamin D3, 100,000 IU in people with Sickle Cell Disease and Anemia, Sickle Cell, run by Columbia University at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 3 to 20 years
- Conditions the study is for
- Sickle Cell Disease, Anemia, Sickle Cell, Anemia, Hemolytic, Congenital
- What is being tested?
- Daily oral vitamin D3, 3,333 IU and Monthly oral vitamin D3, 100,000 IU
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 58 participants
- Where
- 1 siteUnited States
- Study dates
- 15 Sep 2020to 18 Jun 2024
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 20 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosis of sickle cell disease (Hb SS, Hb SC, Hb S-Beta-thalassemia)
- Age 3-20 years old
Who cannot join
- 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
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- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Columbia University Medical Center
New York, New York, 10032
About this study
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).
Read the detailed description
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.
- Conditions
- Sickle Cell Disease, Anemia, Sickle Cell, Anemia, Hemolytic, Congenital, Respiratory Tract Diseases, Respiration Disorders, Acute Chest Syndrome
- Being tested
- Daily oral vitamin D3, 3,333 IU, Monthly oral vitamin D3, 100,000 IU, Placebo oral tablet
- Keywords
- Sickle Cell Disease, Acute Chest Syndrome, Respiratory Complication, Vitamin D Deficiency
- Medical subject headings
- Anemia, Sickle Cell, Anemia, Hemolytic, Congenital, Respiratory Tract Diseases, Respiration Disorders, Acute Chest Syndrome, Lung Diseases, Asthma, Respiratory Tract Infections
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Daily oral vitamin D3, 3,333 IUDrug
Also called Cholecalciferol for oral administration
Oral vitamin D3, 3,333 IU, will be administered daily.
Monthly oral vitamin D3, 100,000 IUDrug
Also called Cholecalciferol for oral administration
Oral vitamin D3, 100,000 IU, will be administered monthly.
Placebo oral tabletDrug
Participants randomized to receive once monthly oral bolus of vitamin D3, will receive placebo on all other days of the month.
| Study group | Role | What they receive |
|---|---|---|
| Daily oral vitamin D3 | Experimental | Oral vitamin D3, 3,333 IU Receives: Drug: Daily oral vitamin D3, 3,333 IU |
| Monthly bolus oral vitamin D3 | Active comparator | Bolus oral vitamin D3, 100,000 IU Receives: Drug: Monthly oral vitamin D3, 100,000 IU; Drug: Placebo oral tablet |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annual Rate of Respiratory Events
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.
Measured: Month 12, Month 24
Secondary outcomes (9)
Mean Forced Vital Capacity (FVC % Predicted)
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.
Measured: Baseline, Month 24
Forced Expiratory Volume in 1 Second (FEV1)
Forced Expiratory Volume in 1 second (FEV1; % predicted) at baseline and at month 24.
Measured: Baseline, Month 24
Forced Expiratory Volume in 1 Second (FEV1)/Forced Vital Capacity Ratio
Forced Expiratory Volume in 1 second (FEV1; % predicted)/Forced Vital Capacity (FVC) [FEV1/FVC] % predicted at baseline and month 24
Measured: Baseline, Month 24
Forced Expiratory Flow at 25%-75% Vital Capacity (FEF25-75, % Predicted)
Forced Expiratory Flow at 25%-75% vital capacity (FEF25-75) % predicted at baseline and month 24 .
Measured: Baseline, Month 24
Ratio of Residual Lung Volume (RV) to Total Lung Capacity (TLC)
Ratio of Residual Lung Volume (RV) to Total Lung Capacity (RV/TLC) at baseline and month 24.
Measured: Baseline, Month 24
Diffusing Capacity of the Lungs for Carbon Monoxide (DLCO)
Diffusing Capacity of the Lungs for Carbon Monoxide (DLCO; % predicted) at baseline and month 24
Measured: Baseline, Month 24
Neutrophil Count
Blood Neutrophil Count in percentage at baseline, month 12 and month 24
Measured: Baseline, Month 12, Month 24
Platelet Count
Blood Platelet Count (Platelets*10^3/ per μL) at baseline, month 12 and month 24
Measured: Baseline, Month 12, Month 24
Serum C-reactive Protein (CRP)
Serum C-reactive protein (CRP; mg/L) at baseline, month 12 and month 24
Measured: Baseline, Month 12, Month 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Nov 2019
- Study start15 Sep 2020
- Primary completion18 Jun 2024
- Study completion18 Jun 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by Columbia UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Controlled, double-masked, randomized Phase 2 clinical trial
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 58 participants (actual)
Sponsor and investigators
- Lead sponsor
- Columbia University
- Sponsor type
- University, hospital or other
- Collaborators
- FDA Office of Orphan Products Development
- Responsible party
- Principal investigator, Margaret T. Lee, Columbia University
- Investigators
- Gary M Brittenham, MD, Study chair, Columbia UniversityMargaret T Lee, MD, Principal investigator, Columbia University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04170348
- Sponsor's protocol ID
- AAAS0396
- Other IDs
- R01FD006372
- Acronym
- ViDAS-2
- Status last verified
- Sep 2025
- First submitted
- 1 Oct 2019
- Last updated
- 24 Sep 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Lee MT, Kattan M, Fennoy I, Arpadi SM, Miller RL, Cremers S, McMahon DJ, Nieves JW, Brittenham GM. Randomized phase 2 trial of monthly vitamin D to prevent respiratory complications in children with sickle cell disease. Blood Adv. 2018 May 8;2(9):969-978. doi: 10.1182/bloodadvances.2017013979.
PubMed 29712666Williams KM, Lee MT, Licursi M, Brittenham GM, Fennoy I. Response to Long-term Vitamin D Therapy for Bone Disease in Children With Sickle Cell Disease. J Pediatr Hematol Oncol. 2018 Aug;40(6):458-461. doi: 10.1097/MPH.0000000000001155.
PubMed 29668535De A, Anekwe CV, Kattan M, Yao Y, Jin Z, Brittenham GM, Lee MT. Validation of a Questionnaire to Identify Respiratory Tract Infections in Children With Sickle Cell Disease. J Pediatr Hematol Oncol. 2021 Jul 1;43(5):e661-e665. doi: 10.1097/MPH.0000000000002164.
PubMed 33885042