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Vitamin D for Sickle-cell Respiratory Complications

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01443728
First Posted: September 30, 2011
Last Update Posted: June 23, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Gary M Brittenham, MD, Columbia University
  Purpose

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


Condition Intervention Phase
Sickle Cell Disease Vitamin D Deficiency Acute Chest Syndrome Asthma Respiratory Infections Drug: Vitamin D3 Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Vitamin D for Sickle-cell Respiratory Complications

Resource links provided by NLM:


Further study details as provided by Gary M Brittenham, MD, Columbia University:

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

Secondary Outcome Measures:
  • 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 ]

Estimated Enrollment: 130
Study Start Date: December 2011
Estimated Study Completion Date: January 2018
Primary Completion Date: September 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Vitamin D3 100,000 IU
Oral vitamin D3, 100,000 IU [2.5 mg] given once a month
Drug: Vitamin D3
Vitamin D3 100,000 IU orally once a month
Other Name: Cholecalciferol
Active Comparator: Vitamin D3 12,000 IU
Standard dose oral vitamin D3 12,000 IU [0.3 mg] given once a month
Drug: Vitamin D3
Vitamin D3 12,000 IU orally once a month
Other Name: Cholecalciferol

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.

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   3 Years to 20 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosis of sickle cell disease (HbSS, HbSC, HbS 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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01443728


Locations
United States, New York
Columbia University Medical Center
New York, New York, United States, 10032
Sponsors and Collaborators
Gary M Brittenham, MD
Investigators
Principal Investigator: Gary Brittenham, MD Columbia University
Principal Investigator: Margaret T Lee, MD Columbia University
  More Information

Responsible Party: Gary M Brittenham, MD, James A. Wolff Professor of Pediatrics, Columbia University
ClinicalTrials.gov Identifier: NCT01443728     History of Changes
Other Study ID Numbers: AAAE3244
R01FD003894 ( U.S. FDA Grant/Contract )
First Submitted: September 27, 2011
First Posted: September 30, 2011
Last Update Posted: June 23, 2017
Last Verified: October 2016

Keywords provided by Gary M Brittenham, MD, Columbia University:
sickle cell disease
acute chest syndrome
respiratory complications
vitamin D

Additional relevant MeSH terms:
Anemia, Sickle Cell
Vitamin D Deficiency
Respiratory Tract Infections
Acute Chest Syndrome
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Avitaminosis
Deficiency Diseases
Malnutrition
Nutrition Disorders
Infection
Respiratory Tract Diseases
Lung Diseases
Respiration Disorders
Vitamins
Vitamin D
Ergocalciferols
Cholecalciferol
Micronutrients
Growth Substances
Physiological Effects of Drugs
Bone Density Conservation Agents