Phase 2
N=58
Daily Vitamin D for Sickle-cell Respiratory Complications
Sickle Cell Disease · Anemia, Sickle Cell · Anemia, Hemolytic, Congenital · Respiratory Tract Diseases · Respiration Disorders
Bottom Line
View on ClinicalTrials.gov: NCT04170348 ↗Enrolled (actual)
58
Serious AEs
19.0%
Results posted
Sep 2025
Primary outcome: Primary: Annual Rate of Respiratory Events — 3.3; 3.3; 3.4; 3.2 Respiratory events per year
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Daily oral vitamin D3, 3,333 IU (Drug); Monthly oral vitamin D3, 100,000 IU (Drug); Placebo oral tablet (Drug)
- Age
- Pediatric, Adult · 3+ yrs
- Sex
- All
- Sponsor
- Columbia University
- Primary completion
- Jun 2024
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Annual Rate of Respiratory Events |
3.3; 3.3; 3.4; 3.2 | — |
| SECONDARY Mean Forced Vital Capacity (FVC % Predicted) |
86.3; 84.3; 83.2; 88.7 | — |
| SECONDARY Forced Expiratory Volume in 1 Second (FEV1) |
84.6; 84.4; 85.5; 90.8 | — |
| SECONDARY Forced Expiratory Volume in 1 Second (FEV1)/Forced Vital Capacity Ratio |
97.9; 100.1; 97.5; 97.7 | — |
| SECONDARY Forced Expiratory Flow at 25%-75% Vital Capacity (FEF25-75, % Predicted) |
80.9; 86.6; 81.1; 83.7 | — |
| SECONDARY Ratio of Residual Lung Volume (RV) to Total Lung Capacity (TLC) |
121.0; 101.2; 106.9; 109.1 | — |
| SECONDARY Diffusing Capacity of the Lungs for Carbon Monoxide (DLCO) |
87.3; 85.3; 86.5; 90.0 | — |
| SECONDARY Neutrophil Count |
45.8; 43.9; 47.7; 43.0; 45.5; 46.8 | — |
| SECONDARY Platelet Count |
355.1; 376.2; 390.0; 387.2; 398.9; 359.9 | — |
| SECONDARY Serum C-reactive Protein (CRP) |
2.1; 2.9; 5.9; 3.6; 3.0; 4.6 | — |
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. 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).
Eligibility Criteria
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
Data sourced from ClinicalTrials.gov (NCT04170348). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.