Cystic FibrosisNCT01222273
Open-label Vitamin D Trial for Patients With Cystic Fibrosis and Allergic Bronchopulmonary Aspergillosis
A study testing cholecalciferol (Vitamin D3) in people with Cystic Fibrosis and Allergic Bronchopulmonary Aspergillosis, run by University of Pittsburgh at 2 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Cystic Fibrosis, Allergic Bronchopulmonary Aspergillosis
- What is being tested?
- cholecalciferol (Vitamin D3)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 2 sitesUnited States
- Study dates
- Sep 2010to Sep 2013
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male or female ≥ 12 years of age at enrollment
- Confirmed diagnosis of CF based on the following criteria:
- One or more clinical features consistent with the CF phenotype AND (b or c)
- Positive sweat chloride > 60 mEq/liter (by pilocarpine iontophoresis)
- two identifiable mutations consistent with CF
- 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.
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- Clinically stable at enrollment as assessed by the site investigator
- Past or present respiratory culture positive for Aspergillus fumigatus
- IgE ≥ 250 and/or presence of class II or higher aspergillus specific IgE on enrollment
- Ability to comply with medication use, study visits and study procedures as judged by the site investigator -
Who cannot join
- 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
- 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
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Comprehensive Lung Center - Falk Clinic
Pittsburgh, Pennsylvania, 15213
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
About this study
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.
Read the 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.
- Being tested
- cholecalciferol (Vitamin D3)
- Keywords
- cystic fibrosis, Allergic bronchopulmonary aspergillosis, A. fumigatus, Immune response
- Medical subject headings
- Cystic Fibrosis, Aspergillosis, Allergic Bronchopulmonary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
cholecalciferol (Vitamin D3)Dietary supplement
Also called cholecalciferol
4,000 IU of cholecalciferol (Vitamin D3) orally every day for six months
| Study group | Role | What they receive |
|---|---|---|
| Open label | Other | open label Vitamin D Receives: Dietary Supplement: cholecalciferol (Vitamin D3) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Aspergillus Induced IL-13 Responses in CD4+ T-cells
To test the hypothesis that supplementation with Vitamin D in CF patients with ABPA will reduce Aspergillus induced IL-13 responses in peripheral CD4+ T-cells. Response confirmed for each individual patient and recorded as number of participants with response.
Measured: 6 months
Secondary outcomes (2)
Change in Patient Total IgE Levels
To test the hypothesis that supplementation with Vitamin D in CF patients with ABPA will reduce total IgE levels by the end of the 24-week period
Measured: 6 months
Change in Patient Aspergillus Specific IgE Levels
To test the hypothesis that supplementation with Vitamin D in CF patients with ABPA will reduce aspergillus specific IgE levels by the end of the 24-week period
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Oct 2010 (estimated)
- Study startSep 2010
- Primary completionSep 2013
- Study completionSep 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by University of PittsburghThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Pittsburgh
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Principal investigator, Joseph Pilewski, University of Pittsburgh
- Investigators
- Joseph M Pilewski, MD, Principal investigator, University of PittsburghJay K Kolls, MD, Study director, University of Pittsburgh
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01222273
- Sponsor's protocol ID
- PRO09090370
- Other IDs
- 7P50HL084932-04
- Status last verified
- Mar 2018
- First submitted
- 14 Oct 2010
- Last updated
- 29 Mar 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- No
Publications
Nguyen NL, Pilewski JM, Celedon JC, Mandalapu S, Blanchard ML, DeRicco A, Hartigan E, Alcorn JF, Kolls JK. Vitamin D supplementation decreases Aspergillus fumigatus specific Th2 responses in CF patients with aspergillus sensitization: a phase one open-label study. Asthma Res Pract. 2015;1:3. doi: 10.1186/s40733-015-0003-5. Epub 2015 Jun 4.
PubMed 27011794
Study at a glance
From the registry record, last updated by the sponsor on 29 Mar 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open-label Vitamin D Trial for Patients With Cystic Fibrosis and Allergic Bronchopulmonary Aspergillosis
- ClinicalTrials.gov ID
- NCT01222273
- Acronym / sponsor ID
- PRO09090370
- Keywords
- cystic fibrosis, Allergic bronchopulmonary aspergillosis, A. fumigatus, Immune response
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 7 participants (actual)
- Lead sponsor
- University of PittsburghUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- Sep 2010
- Primary completion
- Sep 2013 (actual)
- Study completion
- Sep 2013 (actual)
- First posted
- 18 Oct 2010 (estimated)
- Results
- Posted 29 Mar 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Open label | Other | Dietary Supplement: cholecalciferol (Vitamin D3)open label Vitamin D |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- Male or female ≥ 12 years of age at enrollment
- Confirmed diagnosis of CF based on the following criteria:
- 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.
- Clinically stable at enrollment as assessed by the site investigator
- Past or present respiratory culture positive for Aspergillus fumigatus
Key exclusion criteria (5 listed)
- 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
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| cholecalciferol (Vitamin D3)Also: cholecalciferol4,000 IU of cholecalciferol (Vitamin D3) orally every day for six months | Dietary supplement | Open label |
Primary outcome
Number of Participants With 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 peripheral CD4+ T-cells. Response confirmed for each individual patient and recorded as number of participants with response.
2 secondary outcomes
Change in Patient Total IgE Levels
Time frame: 6 months
Change in Patient Aspergillus Specific IgE Levels
Time frame: 6 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Mar 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 27011794Nguyen NL, Pilewski JM, Celedon JC, Mandalapu S, Blanchard ML, DeRicco A, Hartigan E, Alcorn JF, Kolls JK. Vitamin D supplementation decreases Aspergillus fumigatus specific Th2 responses in CF patients with aspergillus sensitization: a phase one open-label study. Asthma Res Pract. 2015;1:3. doi: 10.1186/s40733-015-0003-5. Epub 2015 Jun 4.
- Vitamin D supplementation decreases Aspergillus fumigatus specific Th2 responses in CF patients with aspergillus sensitization: a phase one open-label studyLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Joseph M Pilewski, MD principal investigator
University of Pittsburgh
Jay K Kolls, MD study director
University of Pittsburgh
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing cholecalciferol (Vitamin D3) in people with Cystic Fibrosis and Allergic Bronchopulmonary Aspergillosis, run by University of Pittsburgh at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 29 Mar 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 29 Mar 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Aspergillus Induced IL-13 Responses in CD4+ T-cells (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01222273, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Mar 2018.