The Vitamin D for Enhancing the Immune System in Cystic Fibrosis (DISC) trial: Rationale and design of a multi-center, double-blind, placebo-controlled trial of high dose bolus administration of vitamin D3 during acute pulmonary exacerbation of cystic fibrosis

• Type of Material: Article
• Authors: Vin Tangpricha, Emory University; Ellen M. Smith, Emory University; Jose N Binongo, Emory University; Suzanne E. Judd, University of Alabama Birmingham; Thomas R Ziegler, Emory University; Seth Walker, Emory University; Rabindra M. Tirouvanziam, Emory University; Susu M Zughaier, Emory University; Moon Jeong Lee, Emory University; Supavit Chesdachai, Emory University; Wendy A. Hermes, Emory University; James F. Chmiel, Case Western Reserve University; Amit Gaggar, University of Alabama Birmingham; Ruth E. Grossmann, University of Iowa; Patricia M. Joseph, University of Cincinnati; Jessica A. Alvarez, Emory University
• Language: English
• Date: 2017-06-01
• Publisher: Elsevier: Creative Commons Attribution Non-Commercial No-Derivatives License
• Publication Version: Final Published Version
• Copyright Statement: © 2017 Published by Elsevier Inc.
• License: Creative Commons Attribution-NonCommercial-NoDerivs 4.0 International
• Final Published Version (URL): http://dx.doi.org/10.1016/j.conctc.2017.02.010
• Title of Journal or Parent Work: Contemporary Clinical Trials Communications
• ISSN: 2451-8654
• Volume: 6
• Start Page: 39
• End Page: 45
• Grant/Funding Information: The study was supported by a grant from the CF Foundation Clinical Research Award Program (TANGPR11A0) and indirectly by the CF Foundation Therapeutic Drug Network and Center Grants.; The National Center for Advancing Translational Sciences of the National Institutes of Health and the Atlanta Clinical and Translational Science Institute (UL1TR000454) provided additional research support at the coordinating site at Emory University.
• Keywords: Life Sciences & Biomedicine; CHOLECALCIFEROL; Pulmonary exacerbation; Vitamin D; ERGOCALCIFEROL; INFLAMMATION; Science & Technology; Diet; D DEFICIENCY; CHILDREN; 25-HYDROXYVITAMIN-D; Cystic fibrosis; Randomized controlled trial; Nutrition; ADULTS; SUPPLEMENTATION; AIRWAY SMOOTH-MUSCLE; DISEASE; Research & Experimental Medicine; Medicine, Research & Experimental
• Research Categories: Health Sciences, Immunology; Biology, Microbiology; Health Sciences, Nutrition

Abstract

Vitamin D deficiency is highly prevalent in children and adults with cystic fibrosis (CF). Recent studies have found an association between vitamin D status and risk of pulmonary exacerbations in children and adults with CF. The ongoing Vitamin D for enhancing the Immune System in Cystic fibrosis (DISC) study, a multi-center, double-blind, randomized, placebo-controlled trial, will test the hypothesis of whether high dose vitamin D given as a single oral bolus of 250,000 IU to adults with CF during a pulmonary exacerbation followed by a maintenance dose of vitamin D will improve time to next pulmonary exacerbation and re-hospitalization, improve survival and lung function compared to placebo and reduce the rates of pulmonary exacerbation. Subjects will be randomized 1:1 at each clinical site to vitamin D or placebo within 72 h of hospital admission for pulmonary exacerbation. Clinical follow-up visits will occur at 1, 2, 3, and 7 days, and 1, 3, 6 and 12 months after randomization. Blood and sputum will be collected and determination of clinical outcomes will be assessed at each visit. The primary endpoint will be the time to next pulmonary exacerbation requiring antibiotics, re-hospitalization or death. The secondary endpoints will include lung function assessed by forced expiratory volume in 1 s (FEV 1 ), blood markers of inflammatory cytokines, anti-microbial peptide expression by peripheral blood mononuclear cells and circulating concentrations in blood. Other exploratory endpoints will examine the phenotype of neutrophils and monocyte/macrophages in sputum. Nutritional status will be assessed by 3 day food records and food frequency questionnaire.