Vitamin D Supplementation in Children With Sickle Cell Disease
Condition: Sickle Cell Disease
MeSH terms: Anemia, Sickle Cell
Study type: Interventional
Phase: Phase 3

Summary: 
      Sickle cell disease (SCD) is a genetic disease characterized by abnormal hemoglobin, the main
      constituent of red blood cells. People with SCD have nutritional deficiencies, and vitamin D
      deficiency is one of the most common. Symptoms of vitamin D deficiency are similar to those
      of SCD and include chronic pain and bone complications. Correcting vitamin D nutrition of
      children with SCD represents a treatment that will improve their health. A single oral
      high-dose of vitamin D3 will be given to SCD children during one of their follow-up visits at
      the SCD clinic of CHU Sainte-Justine, Montreal, Canada. This mode of administration was
      chosen to ensure a better adherence to the treatment. The investigators will determine
      whether this dose is safe and its administration feasible in clinic. The impact of this dose
      on blood vitamin D and calcium, urinary calcium, growth, inflammation, bone health, pain and
      quality of life will also be assessed. This study intends to propose a new intervention to
      improve the nutrition of children with this disease.
    

Detailed description: 
      Vitamin D deficiency is one of the most common nutritional conditions among patients with
      sickle cell disease (SCD). Since vitamin D deficiency and SCD share common manifestations
      including chronic pain, poor bone health and chronic systemic inflammation, it is reasonable
      to postulate that vitamin D deficiency may contribute to these complications. Thus,
      optimizing vitamin D nutrition represents an inexpensive strategy that may improve vitamin D
      status and health outcomes in SCD children. The working hypothesis is that administration of
      a single oral bolus of 300,000 IU of vitamin D3 to SCD children will result in the attainment
      of vitamin D sufficiency (25OHD levels >75 nmol/L) in 80% of participants after 3 months. The
      primary objectives are to assess feasibility, acceptability, and safety of the vitamin D3
      bolus while secondary objectives are related to the mean change in serum 25OHD from baseline
      to 3 months post-bolus and its clinical impact. Seventy-two SCD children (5-17 years, SS and
      SC genotypes) will be randomized to one bolus of 300,000 IU of vitamin D3 or identical
      placebo. Blood will be collected at baseline and 3-month post-bolus to measure serum 25OHD
      and calculate the change from baseline at 3 months (efficacy outcomes). Other outcomes
      include urinary calcium/creatinine ratio and serum calcium (safety), questionnaires
      (acceptability and musculoskeletal pain) and parameters related to growth, haematology,
      inflammation and bone health (exploratory outcomes).
    

Intervention name(s): Vitamin D bolus; Placebo
Intervention type(s): Dietary Supplement; Dietary Supplement
Intervention description(s): One single oral liquid vitamin D3 supplement of 300 000 IU; Placebo identical in taste and appearance to the vitamin D bolus
Other intervention name(s): Cholecalciferol
Intervention MeSH terms: Vitamin D; Cholecalciferol
Allocation: Randomized
Intervention model: Parallel Assignment
Intervention model description: This is a randomised, quadruble-blind, placebo-controlled, parallel-group trial of vitamin D3 bolus supplementation.
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Other
Keywords: Bolus supplementation; Pilot randomised controlled trial; Vitamin D