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Open-label Vitamin D Trial for Patients With Cystic Fibrosis and Allergic Bronchopulmonary Aspergillosis

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ClinicalTrials.gov Identifier: NCT01222273
Recruitment Status : Completed
First Posted : October 18, 2010
Last Update Posted : January 12, 2016
National Heart, Lung, and Blood Institute (NHLBI)
Information provided by (Responsible Party):
Joseph Pilewski, University of Pittsburgh

Brief Summary:
The purpose of this study is to see if giving people with CF and ABPA enough vitamin D to make their blood levels of the vitamin higher, will reduce the allergic response in their body and make the symptoms caused by ABPA better.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Allergic Bronchopulmonary Aspergillosis Dietary Supplement: cholecalciferol (Vitamin D3) Phase 1 Phase 2

Detailed Description:

Many patients with cystic fibrosis (CF) cough up mucus or have throat cultures that grow a common fungus called Aspergillus. In patients with CF, aspergillus is not known to cause direct damage to the lungs, but some patients respond with an allergic reaction that causes them to wheeze, cough, or have difficulty breathing. This allergic reaction is called ABPA. Current treatment for ABPA includes high dose steroids and an "anti-fungal" medicine. Treatment with steroids may be problematic for some people due to its side effects on blood sugar levels and the bones. Steroids are medications that decrease inflammation, including prednisone, medrol, dexamethasone and others.

Ongoing research at UPMC on the study "Mechanisms of Immune Tolerance in ABPA" has studied people with CF and ABPA versus those patients with CF that just grow A. fumigatus (Af) in the sputum, but do not have ABPA. You may have participated in this study. This study has shown that people with CF with the fungus, Af, in their sputum but who do not have ABPA have more of a certain type of cell in their blood that helps the body to regulate or suppress allergic reactions than those people with CF and ABPA.

Recent studies have demonstrated that Vitamin D is a critical factor in the development of these cells that suppress allergic reactions. People with CF, due to their pancreatic insufficiency that causes them to have difficulty absorbing fat, also have lower levels of the fat soluble vitamins which include vitamin D. In the study done at UPMC, "Mechanisms of Immune Tolerance in ABPA", people with CF and ABPA had significantly lower vitamin D levels than people with CF who did not have ABPA.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 15 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Open-label Vitamin D Trial for Patients With Cystic Fibrosis and Allergic Bronchopulmonary Aspergillosis
Study Start Date : September 2010
Primary Completion Date : September 2013
Study Completion Date : September 2013

Arm Intervention/treatment
Experimental: cholecalciferol
2000 Units of cholecalciferol once daily
Dietary Supplement: cholecalciferol (Vitamin D3)
4,000 IU of cholecalciferol (Vitamin D3) orally every day for six months
Other Name: cholecalciferol

Primary Outcome Measures :
  1. Aspergillus induced IL-13 responses in CD4+ T-cells [ Time Frame: 6 months ]
    To test the hypothesis that supplementation with Vitamin D in CF patients with ABPA will reduce Aspergillus induced IL-13 responses in CD4+ T-cells.

Secondary Outcome Measures :
  1. Total IgE, Aspergillus specific IgE and IgG levels, vitamin D levels, FEV1, cytokine production by Aspergillus stimulated peripheral blood T cells, urine calcium/creatinine ratio [ Time Frame: 6 months ]
    To test the hypothesis that supplementation with Vitamin D in CF patients with ABPA will reduce total and aspergillus specific IgE levels

Information from the National Library of Medicine

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Ages Eligible for Study:   16 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Male or female ≥ 12 years of age at enrollment
  2. Confirmed diagnosis of CF based on the following criteria:

    1. One or more clinical features consistent with the CF phenotype AND (b or c)
    2. Positive sweat chloride > 60 mEq/liter (by pilocarpine iontophoresis)
    3. two identifiable mutations consistent with CF
  3. Written informed consent (and assent when applicable) obtained from subject or subject's legal representative and ability for subject to comply with the requirements of the study.
  4. Clinically stable at enrollment as assessed by the site investigator
  5. Past or present respiratory culture positive for Aspergillus fumigatus
  6. IgE ≥ 250 and/or presence of class II or higher aspergillus specific IgE on enrollment
  7. Ability to comply with medication use, study visits and study procedures as judged by the site investigator -

Exclusion Criteria:

  • 1. Systemic corticosteroids (1 mg/kg if < 20 kg or > 20 mg of prednisone per day),.

    2. Investigational drug use within 30 days of screening 3. Laboratory abnormalities at screening

    a. Serum Calcium > 11 mg/dl b. 25(OH) D > 50 ng/ml at screening. c. Creatinine ≥ 1.5, or estimated GFR <60 by Cockcroft-Gault or MDRD equation. d. LFT≥ 3xULN

    4. History of transplantation or currently on lung transplant list 5. Positive serum pregnancy test at screening (to be performed on all post-menarche females) 6. Pregnant, breastfeeding, or if post-menarche female, unwilling to practice birth control during participation in the study 7. Presence of a condition or abnormality that in the opinion of the site investigator would compromise the safety of the subject or the quality of the data 8. Diagnosis of HIV and a CD4+ T cell count below 500 cells/ml or active hepatitis B or C infection.

    9. Undergoing therapy for non-tuberculous mycobacterial infection

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): NCT01222273

United States, Pennsylvania
Comprehensive Lung Center - Falk Clinic
Pittsburgh, Pennsylvania, United States, 15213
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States, 15224
Sponsors and Collaborators
University of Pittsburgh
National Heart, Lung, and Blood Institute (NHLBI)
Principal Investigator: Joseph M Pilewski, MD University of Pittsburgh
Study Director: Jay K Kolls, MD University of Pittsburgh

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Joseph Pilewski, Associate Professor of Pediatrics, University of Pittsburgh
ClinicalTrials.gov Identifier: NCT01222273     History of Changes
Other Study ID Numbers: PRO09090370
7P50HL084932-04 ( U.S. NIH Grant/Contract )
First Posted: October 18, 2010    Key Record Dates
Last Update Posted: January 12, 2016
Last Verified: June 2012

Keywords provided by Joseph Pilewski, University of Pittsburgh:
cystic fibrosis
Allergic bronchopulmonary aspergillosis
A. fumigatus
Immune response

Additional relevant MeSH terms:
Cystic Fibrosis
Pulmonary Aspergillosis
Aspergillosis, Allergic Bronchopulmonary
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Lung Diseases, Fungal
Skin Diseases, Infectious
Skin Diseases
Respiratory Hypersensitivity
Respiratory Tract Infections
Hypersensitivity, Immediate
Immune System Diseases
Vitamin D
Growth Substances
Physiological Effects of Drugs