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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

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.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Pulmonary Exacerbation Drug: Prednisone Drug: Placebos Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 84 participants
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
Official Title: Randomized Controlled Trial of Prednisone in Cystic Fibrosis Pulmonary Exacerbations
Actual Study Start Date : May 1, 2017
Estimated Primary Completion Date : May 2020
Estimated Study Completion Date : May 2021

Resource links provided by the National Library of Medicine

Drug Information available for: Prednisone

Arm Intervention/treatment
Active Comparator: Placebo
Placebo
Drug: Placebos
Placebo

Active Comparator: Treatment
Prednisone
Drug: Prednisone
oral prednisone for 7 days during pulmonary exacerbation




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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

  2. change in pulmonary function testing [ Time Frame: at day 7, 14 and 1 month follow up ]
    change in pulmonary function testing

  3. quality of life as measured by CFQ-R questionnaire [ Time Frame: at day 7, 14 and 1 month follow up ]
    quality of life

  4. quality of life as measured by CF Respiratory Symptom Diary [ Time Frame: at day 7, 14 and 1 month follow up ]
    quality of life

  5. length of hospitalization [ Time Frame: Through study completion, up to 100 weeks ]
    length of hospitalization

  6. time to subsequent pulmonary exacerbation [ Time Frame: 1 year follow up time ]
    time to subsequent pulmonary exacerbation

  7. number of adverse events [ Time Frame: At day day 14 of antibiotic therapy and 1 month follow up ]
    number of adverse events

  8. change in sputum inflammatory markers [ Time Frame: at day 7, 14 and 1 month follow up ]
    change in sputum inflammatory markers

  9. change in serum inflammatory markers [ Time Frame: at day 7, 14 and 1 month follow up ]
    change in serum inflammatory markers

  10. Duration of antibiotic treatment [ Time Frame: Through study completion, up to 100 weeks ]
    Duration of antibiotic treatment



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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

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): NCT03070522


Contacts
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Contact: Valerie Waters, MD 416-813-7654 ext 204541 valerie.waters@sickkids.ca
Contact: Nancy McDonald 416-813-7654 nancy.mcdonald@sickkids.ca

Locations
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Canada, Ontario
SickKids Recruiting
Toronto, Ontario, Canada, M5G1X8
Contact: Valerie Waters    416-813-7654    valerie.waters@sickkids.ca   
Contact: Nancy McDonald       nancy.mcdonald@sickkids.ca   
Sponsors and Collaborators
The Hospital for Sick Children
Canadian Cystic Fibrosis Foundation

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Responsible Party: Valerie Waters, Staff physician, The Hospital for Sick Children
ClinicalTrials.gov Identifier: NCT03070522     History of Changes
Other Study ID Numbers: REB1000053825
First Posted: March 3, 2017    Key Record Dates
Last Update Posted: June 26, 2019
Last Verified: June 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Cystic Fibrosis
Pulmonary Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Prednisone
Anti-Inflammatory Agents
Glucocorticoids
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Antineoplastic Agents, Hormonal
Antineoplastic Agents