PIPE: Prednisone in Cystic Fibrosis Pulmonary Exacerbations

Sponsor
The Hospital for Sick Children (Other)
Overall Status
Recruiting
CT.gov ID
NCT03070522
Collaborator
Canadian Cystic Fibrosis Foundation (Other)
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72
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Study Details

Study Description

Brief Summary

This will be a 5 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 and other study sub-sites across 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
Phase 3

Study Design

Study Type:
Interventional
Anticipated Enrollment :
84 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Intervention Model Description:
Randomized placebo controlled trialRandomized 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
Anticipated Primary Completion Date :
May 1, 2023
Anticipated Study Completion Date :
May 1, 2023

Arms and Interventions

Arm Intervention/Treatment
Active Comparator: Placebo

Placebo

Drug: Placebos
Placebo

Active Comparator: Treatment

Prednisone

Drug: Prednisone
oral prednisone for 7 days during pulmonary exacerbation

Outcome Measures

Primary Outcome Measures

  1. Lung function recovery [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 [1 month follow up]

    The proportion of subjects who achieve >90% of their baseline FEV1 % predicted

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

    change in pulmonary function testing

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

    quality of life

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

    quality of life

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

    length of hospitalization

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

    time to subsequent pulmonary exacerbation

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

    number of adverse events

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

    change in sputum inflammatory markers

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

    change in serum inflammatory markers

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

    Duration of antibiotic treatment

Eligibility Criteria

Criteria

Ages Eligible for Study:
N/A and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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

Contacts and Locations

Locations

Site City State Country Postal Code
1 The Governers of The University of Calgary - Alberta Health Services Calgary Alberta Canada T2N 4N1
2 British Columbia Children's Hospital Vancouver British Columbia Canada V6H 3N1
3 St. Paul's Hospital Vancouver British Columbia Canada V6Z 1Y6
4 London Health Sciences Centre - Lawson Health Research Institute London Ontario Canada N6C 2R5
5 The Ottawa Hospital Ottawa Ontario Canada K1H 8L6
6 Unity Health Toronto - St. Michael's Hospital Toronto Ontario Canada M5B 1W8
7 SickKids Toronto Ontario Canada M5G1X8
8 The Centre hospitalier de l'Université de Montréal (CHUM) Montréal Quebec Canada H2X A09
9 Centre hospitalier universitaire Sainte-Justine Montréal Quebec Canada H3T 1C5
10 CHU de Quebec-Universite Laval Quebec City Quebec Canada G1R 2J6
11 Institut universitaire de cardiologie et de pneumologie de Québec - Université Laval Quebec City Quebec Canada G1V 4G5
12 University of Saskatchewan - Saskatchewan Health Authority Saskatoon Saskatchewan Canada S7N 0W8

Sponsors and Collaborators

  • The Hospital for Sick Children
  • Canadian Cystic Fibrosis Foundation

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Valerie Waters, Staff physician, The Hospital for Sick Children
ClinicalTrials.gov Identifier:
NCT03070522
Other Study ID Numbers:
  • REB1000053825
First Posted:
Mar 3, 2017
Last Update Posted:
Jun 9, 2021
Last Verified:
Jun 1, 2021
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:

Study Results

No Results Posted as of Jun 9, 2021