Clinical trial NCT01443728
Conditions: Sickle Cell Disease Vitamin D Deficiency Acute Chest Syndrome Asthma Respiratory Infections
Keywords: sickle cell disease acute chest syndrome respiratory complications vitamin D
Sponsor: Gary M Brittenham, MD
Start date: 2011-12-13; Completion date: 2015-02-15
Enrollment: 70 participants; Allocation: RANDOMIZED; Intervention model: PARALLEL

Brief 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

Detailed description:
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.

Arms: arm 1: Oral vitamin D3, 100,000 IU \[2.5 mg\] given once a month arm 2: Standard dose oral vitamin D3 12,000 IU \[0.3 mg\] given once a month
Interventions: intervention 1: Experimental: Vitamin D3 100,000 IU intervention 2: Active Comparator: Vitamin D3 12,000 IU
Intervention descriptions: intervention 1: Oral vitamin D3, 100,000 IU \[2.5 mg\] given once a month intervention 2: Standard dose oral vitamin D3 12,000 IU \[0.3 mg\] given once a month

Protocol documents: