Phase 2
N=62
Vitamin D for Sickle-cell Respiratory Complications
Sickle Cell Disease · Vitamin D Deficiency · Acute Chest Syndrome · Asthma · Respiratory Infections
Bottom Line
View on ClinicalTrials.gov: NCT01443728 ↗Enrolled (actual)
62
Serious AEs
27.4%
Results posted
Aug 2024
Primary outcome: Primary: Mean Annual Rate of Respiratory Events — 3.91; 4.34; 3.34; 4.28 events per year
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Experimental: Vitamin D3 100,000 IU (Drug); Active Comparator: Vitamin D3 12,000 IU (Drug)
- Age
- Pediatric, Adult · 3+ yrs
- Sex
- All
- Sponsor
- Gary M Brittenham, MD
- Primary completion
- Jun 2013
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Mean Annual Rate of Respiratory Events |
3.91; 4.34; 3.34; 4.28; 1.54; 1.49 | — |
| SECONDARY Mean 25-Hydroxyvitamin D (25-OHD) |
36.1; 19.1 | — |
| SECONDARY Forced Vital Capacity (FVC) |
85.5; 90.2; 85.2; 92.4; 85.0; 92.1 | — |
| SECONDARY Forced Expiratory Volume (FEV) in 1 Second (FEV1) |
80.4; 84.9; 79.1; 85.3; 78.9; 84.5 | — |
| SECONDARY FEV1/FVC Ratio |
84.0; 83.3; 82.6; 81.7; 82.2; 81.6 | — |
| SECONDARY FEF 25-75 |
69.8; 68.8; 62.9; 64.8; 62.8; 65.6 | — |
| SECONDARY RV/TLC Ratio |
26.9; 25.1; 25.4; 22.6; 22.8; 21.8 | — |
| SECONDARY DLCO |
68.0; 72.3; 62.9; 67.8; 68.1; 72.9 | — |
| SECONDARY FeNO |
19.3; 16.9; 20.5; 16.1; 18.1; 16.4 | — |
| SECONDARY MIP |
69.1; 59.9; 78.7; 80.4; 77.4; 74.6 | — |
| SECONDARY MEP |
66.3; 66.3; 60.4; 57.3; 58.7; 56.2 | — |
| SECONDARY Hand-grip, Right |
21.2; 16.9; 20.6; 16.6; 23.8; 17.4 | — |
| SECONDARY Hand-grip, Left |
19.9; 16.0; 19.0; 13.6; 19.6; 15.8 | — |
| SECONDARY Hand-grip, Dominant |
21.2; 17.1; 20.8; 17.3; 23.8; 18.1 | — |
Summary
This study aims to answer the question whether oral vitamin D supplementation can decrease lung complications in children and adolescents with sickle cell disease. Lung complications are the leading causes of morbidity and of death in sickle cell disease. Infections and increased inflammation play important roles in the development of the lung problems in sickle cell disease. Emerging evidence shows that vitamin D helps the immune system to fight infection and to control inflammation and could potentially help prevent respiratory complications in patients with sickle cell disease. The investigators hypothesize that oral vitamin D3, 100,000 IU (2.5 mg), given once a month to a group of children and adolescents with sickle cell disease, will reduce the rate of respiratory events (infection, asthma exacerbation and acute chest syndrome) compared to the rate in a group given standard dose oral vitamin D3, 12,000 IU (0.3 mg) given once a month.
Funding Source - U.S. Food & Drug Administration, Office of Orphan Products Development
Eligibility Criteria
Inclusion Criteria
- Diagnosis of sickle cell disease (HbSS, HbSC, HbS Beta-thalassemia)
- Age 3 to 20 years old
Exclusion Criteria
- Patient (or parent or guardian) 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 other therapeutic 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
- Absence of baseline record of respiratory events (respiratory infections, asthma exacerbations, episodes of acute chest syndrome) for the preceding year
- Pregnancy
Data sourced from ClinicalTrials.gov (NCT01443728). 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.