Clinical Trial: Vitamin D for Sickle-cell Respiratory Complications

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: Vitamin D for Sickle-cell Respiratory Complications

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 Summary:

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.


Sponsor: Gary M Brittenham, MD

Current Primary Outcome: Respiratory events (defined as respiratory infection, acute asthma exacerbation, and acute chest syndrome) [ Time Frame: Every year for 2 years ]

Original Primary Outcome: Annual rate of respiratory events [ Time Frame: Up to 2 years ]

Various laboratory measurements for safety and monitoring of adverse events. Monthly oral vitamin D3 (100,000 IU [2.5 mg]), given to children and adolescents with sickle cell disease, will reduce the rate of respiratory events, defined as respiratory infections, exacerbations of asthma, and episodes of the acute chest syndrome.


Current Secondary Outcome:

  • Pulmonary function tests [ Time Frame: Every year for 2 years ]
  • Immune function [ Time Frame: Every 6 months for 2 years ]
    Serum cytokines to measure T-cell effector and regulatory function Measures of systemic inflammation [high-sensitivity C-reactive protein (hs-CRP), Whole Blood Count (WBC), platelets)
  • Bone function and bone turnover markers [ Time Frame: Every 6 months for 2 years ]
    Intact parathyroid hormone Serum C-terminal telopeptides of Type I collagen (CTX) Aminoterminal propeptide of Type 1 procollagen (P1NP)
  • Muscle strength (Hand grip) [ Time Frame: Every year for 2 years ]


Original Secondary Outcome:

Information By: Columbia University

Dates:
Date Received: September 27, 2011
Date Started: December 2011
Date Completion: January 2017
Last Updated: October 10, 2016
Last Verified: October 2016