Vitamin D for Sickle-cell Respiratory Complications
| Status: | Active, not recruiting |
|---|---|
| Conditions: | Asthma, Other Indications, Anemia, Gastrointestinal, Pulmonary |
| Therapuetic Areas: | Gastroenterology, Hematology, Pulmonary / Respiratory Diseases, Other |
| Healthy: | No |
| Age Range: | 3 - 20 |
| Updated: | 4/3/2019 |
| Start Date: | December 2011 |
| End Date: | December 2019 |
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
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
This study will be a Phase 2 double-blind randomized clinical trial in 80 patients with
sickle cell disease, ages 3 to 20 years-old, comparing a 2-year monthly oral dose of vitamin
D3, 100,000 IU (equivalent to 3,300 IU/day) to a standard monthly dose, 12,000 IU (400
IU/day) in reducing the rate of respiratory events (defined as respiratory infections, acute
asthma exacerbation, and the acute chest syndrome) in children and adolescents with sickle
cell disease in comparison with the rates of respiratory events over a baseline period of one
year.
Eligible participants (130 patients) will initially be screened to determine their blood
vitamin D levels (serum 25-hydroxyvitamin D). Those with 25-hydroxyvitamin D levels between 5
and 60 ng/mL will be eligible for randomization. At study entry, blood and urine samples will
be collected for routine and special blood tests including tests on immune function,
inflammation, and bone function. Children above 5 years old will also have lung function and
muscle strength tests. Participants will be followed once a month to administer the study
medication (oral vitamin D3) and to monitor any side effects from the study medication by
history, examination and blood and urine tests. After 12 and 24 months of therapy, the same
study procedures at study entry will be repeated.
This study could help establish oral vitamin D3 as a simple, low cost treatment to reduce
respiratory complications in children and adolescents with sickle cell disease.
sickle cell disease, ages 3 to 20 years-old, comparing a 2-year monthly oral dose of vitamin
D3, 100,000 IU (equivalent to 3,300 IU/day) to a standard monthly dose, 12,000 IU (400
IU/day) in reducing the rate of respiratory events (defined as respiratory infections, acute
asthma exacerbation, and the acute chest syndrome) in children and adolescents with sickle
cell disease in comparison with the rates of respiratory events over a baseline period of one
year.
Eligible participants (130 patients) will initially be screened to determine their blood
vitamin D levels (serum 25-hydroxyvitamin D). Those with 25-hydroxyvitamin D levels between 5
and 60 ng/mL will be eligible for randomization. At study entry, blood and urine samples will
be collected for routine and special blood tests including tests on immune function,
inflammation, and bone function. Children above 5 years old will also have lung function and
muscle strength tests. Participants will be followed once a month to administer the study
medication (oral vitamin D3) and to monitor any side effects from the study medication by
history, examination and blood and urine tests. After 12 and 24 months of therapy, the same
study procedures at study entry will be repeated.
This study could help establish oral vitamin D3 as a simple, low cost treatment to reduce
respiratory complications in children and adolescents with sickle cell disease.
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
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Columbia University Medical Center Situated on a 20-acre campus in Northern Manhattan and accounting for...
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