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Prednisone in Cystic Fibrosis Pulmonary Exacerbations (PIPE)

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ClinicalTrials.gov Identifier: NCT03070522
Recruitment Status : Recruiting
First Posted : March 3, 2017
Last Update Posted : June 26, 2019
Sponsor:
Collaborator:
Canadian Cystic Fibrosis Foundation
Information provided by (Responsible Party):
Valerie Waters, The Hospital for Sick Children

Tracking Information
First Submitted Date  ICMJE February 8, 2017
First Posted Date  ICMJE March 3, 2017
Last Update Posted Date June 26, 2019
Actual Study Start Date  ICMJE May 1, 2017
Estimated Primary Completion Date May 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 28, 2017)
Lung function recovery [ Time Frame: At 14 days of antibiotic therapy ]
The proportion of subjects who achieve >90% of their baseline FEV1 % predicted at day 14 of IV antibiotic treatment for a PEx in each treatment arm.
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03070522 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: February 28, 2017)
  • lung function recovery at follow up visit [ Time Frame: 1 month follow up ]
    The proportion of subjects who achieve >90% of their baseline FEV1 % predicted
  • change in pulmonary function testing [ Time Frame: at day 7, 14 and 1 month follow up ]
    change in pulmonary function testing
  • quality of life as measured by CFQ-R questionnaire [ Time Frame: at day 7, 14 and 1 month follow up ]
    quality of life
  • quality of life as measured by CF Respiratory Symptom Diary [ Time Frame: at day 7, 14 and 1 month follow up ]
    quality of life
  • length of hospitalization [ Time Frame: Through study completion, up to 100 weeks ]
    length of hospitalization
  • time to subsequent pulmonary exacerbation [ Time Frame: 1 year follow up time ]
    time to subsequent pulmonary exacerbation
  • number of adverse events [ Time Frame: At day day 14 of antibiotic therapy and 1 month follow up ]
    number of adverse events
  • change in sputum inflammatory markers [ Time Frame: at day 7, 14 and 1 month follow up ]
    change in sputum inflammatory markers
  • change in serum inflammatory markers [ Time Frame: at day 7, 14 and 1 month follow up ]
    change in serum inflammatory markers
  • Duration of antibiotic treatment [ Time Frame: Through study completion, up to 100 weeks ]
    Duration of antibiotic treatment
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Prednisone in Cystic Fibrosis Pulmonary Exacerbations
Official Title  ICMJE Randomized Controlled Trial of Prednisone in Cystic Fibrosis Pulmonary Exacerbations
Brief Summary This will be a 3 year randomized, double blind, placebo controlled trial of 7 days of oral prednisone in cystic fibrosis (CF) patients receiving intravenous (IV) antibiotic treatment for a pulmonary exacerbation at the Hospital for Sick Children, St Michael's Hospital, St Paul's Hospital, British Columbia Children's Hospital, Hotel Dieu Hospital and Ste Justine Hospital, Canada. The intervention will be oral prednisone 2 mg/kg/day (max 60 mg) divided twice daily for 7 days as an adjunctive therapy for pulmonary exacerbations in CF patients who have not recovered their baseline forced expiratory volume in 1 second (FEV1) after 7 days of IV antibiotic treatment. The primary outcome will be the proportion of subjects who achieve >90% of their baseline FEV1 % predicted at day 14 of IV antibiotic treatment for a pulmonary exacerbation in each treatment arm.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:
Randomized placebo controlled trial
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description:
placebo
Primary Purpose: Treatment
Condition  ICMJE Cystic Fibrosis Pulmonary Exacerbation
Intervention  ICMJE
  • Drug: Prednisone
    oral prednisone for 7 days during pulmonary exacerbation
  • Drug: Placebos
    Placebo
Study Arms  ICMJE
  • Active Comparator: Placebo
    Placebo
    Intervention: Drug: Placebos
  • Active Comparator: Treatment
    Prednisone
    Intervention: Drug: Prednisone
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: February 28, 2017)
84
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE May 2021
Estimated Primary Completion Date May 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Diagnosis of CF by newborn screening or at least one clinical feature of CF, AND either (a) or (b) as follows:
  2. A documented sweat chloride ≥ 60 mEq/L by quantitative pilocarpine iontophoresis
  3. A genotype with two identifiable CF-causing mutations
  4. Age > 6 years old.
  5. Acute pulmonary exacerbation treated with IV antibiotics as previously defined 10% relative drop in FEV1 from baseline at the time of exacerbation
  6. Informed consent by patient or parent/legal guardian
  7. Ability to reproducibly perform pulmonary function testing
  8. Ability to comply with medication use including the ability to take capsules, study visits and study procedures as judged by the site investigator

Exclusion Criteria:

  1. A respiratory tract culture positive for Burkholderia cenocepacia in the 12 months prior to enrollment
  2. A respiratory tract culture positive for Mycobacterium abscessus in the 12 months prior to enrollment
  3. Treatment with IV or oral corticosteroids within 2 weeks of enrollment or from Day 0-Day 7 of the pulmonary exacerbation
  4. Active allergic bronchopulmonary aspergillosis (ABPA) at the time of enrollment as determined by treating physician
  5. Asthma related exacerbation at enrollment as defined by the treating physician based on clinically compatible symptoms (eg. wheeze)
  6. History of avascular necrosis or pathologic bone fracture
  7. Uncontrolled hypertension with end organ damage
  8. Active gastrointestinal bleeding
  9. Status post lung or other organ transplantation
  10. Pregnancy
  11. Lactose intolerance (contained in placebo)
  12. On Lumacaftor-Ivacaftor (Orkambi) at the time of exacerbation
  13. Investigational drug use within 30 days prior to enrollment visit
  14. Physical findings that would compromise the safety of the subject or the quality of the study data as determined by site investigator
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE Child, Adult, Older Adult
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Valerie Waters, MD 416-813-7654 ext 204541 valerie.waters@sickkids.ca
Contact: Nancy McDonald 416-813-7654 nancy.mcdonald@sickkids.ca
Listed Location Countries  ICMJE Canada
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03070522
Other Study ID Numbers  ICMJE REB1000053825
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Valerie Waters, The Hospital for Sick Children
Study Sponsor  ICMJE The Hospital for Sick Children
Collaborators  ICMJE Canadian Cystic Fibrosis Foundation
Investigators  ICMJE Not Provided
PRS Account The Hospital for Sick Children
Verification Date June 2019

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