Vitamin D for Sickle-cell Respiratory Complications
Condition: Sickle Cell Disease; Vitamin D Deficiency; Acute Chest Syndrome; Asthma; Respiratory Infections
MeSH terms: Respiratory Tract Infections; Acute Chest Syndrome; Anemia, Sickle Cell; Vitamin D Deficiency
Study type: Interventional
Phase: Phase 2

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.
    

Intervention name(s): Experimental: Vitamin D3 100,000 IU; Active Comparator: Vitamin D3 12,000 IU
Intervention type(s): Drug; Drug
Intervention description(s): Oral vitamin D3, 100,000 IU [2.5 mg] given once a month; Standard dose oral vitamin D3 12,000 IU [0.3 mg] given once a month
Other intervention name(s): Cholecalciferol; Cholecalciferol
Intervention MeSH terms: Vitamin D; Ergocalciferols; Cholecalciferol; Vitamins
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: sickle cell disease; acute chest syndrome; respiratory complications; vitamin D