Vitamin D for Sickle-cell Respiratory Complications

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Gary M Brittenham, MD, Columbia University
ClinicalTrials.gov Identifier:
NCT01443728
First received: September 27, 2011
Last updated: August 31, 2014
Last verified: August 2014

September 27, 2011
August 31, 2014
December 2011
July 2015   (final data collection date for primary outcome measure)
Respiratory events (defined as respiratory infection, acute asthma exacerbation, and acute chest syndrome) [ Time Frame: Every year for 2 years ] [ Designated as safety issue: No ]
Annual rate of respiratory events [ Time Frame: Up to 2 years ] [ Designated as safety issue: Yes ]
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.
Complete list of historical versions of study NCT01443728 on ClinicalTrials.gov Archive Site
  • Pulmonary function tests [ Time Frame: Every year for 2 years ] [ Designated as safety issue: No ]
  • Immune function [ Time Frame: Every 6 months for 2 years ] [ Designated as safety issue: No ]
    Serum cytokines to measure T-cell effector and regulatory function Measures of systemic inflammation (hs-CRP, WBC, platelets)
  • Bone function and bone turnover markers [ Time Frame: Every 6 months for 2 years ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
Not Provided
Not Provided
Not Provided
 
Vitamin D for Sickle-cell Respiratory Complications
Vitamin D for Sickle-cell Respiratory Complications

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

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.

Interventional
Phase 2
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
  • Sickle Cell Disease
  • Vitamin D Deficiency
  • Acute Chest Syndrome
  • Asthma
  • Respiratory Infections
  • Drug: Vitamin D3
    Vitamin D3 100,000 IU orally once a month
    Other Name: Cholecalciferol
  • Drug: Vitamin D3
    Vitamin D3 12,000 IU orally once a month
    Other Name: Cholecalciferol
  • Experimental: Vitamin D3 100,000 IU
    Oral vitamin D3, 100,000 IU [2.5 mg] given once a month
    Intervention: Drug: Vitamin D3
  • Active Comparator: Vitamin D3 12,000 IU
    Standard dose oral vitamin D3 12,000 IU [0.3 mg] given once a month
    Intervention: Drug: Vitamin D3
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
130
July 2015
July 2015   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • Diagnosis of sickle cell disease (HbSS, SC, S 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
Both
3 Years to 20 Years
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT01443728
AAAE3244, R01FD003894
Yes
Gary M Brittenham, MD, Columbia University
Gary M Brittenham, MD
Not Provided
Principal Investigator: Gary Brittenham, MD Columbia University
Principal Investigator: Margaret T Lee, MD Columbia University
Columbia University
August 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP