Prednisone in Cystic Fibrosis Pulmonary Exacerbations

Last updated: June 27, 2024
Sponsor: The Hospital for Sick Children
Overall Status: Completed

Phase

3

Condition

Cystic Fibrosis

Scar Tissue

Lung Injury

Treatment

Placebos

Prednisone

Clinical Study ID

NCT03070522
REB1000053825
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Diagnosis of CF by newborn screening or at least one clinical feature of CF, ANDeither (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, studyvisits and study procedures as judged by the site investigator

Exclusion

Exclusion Criteria:

  1. A respiratory tract culture positive for Burkholderia cenocepacia in the 12 monthsprior to enrollment

  2. A respiratory tract culture positive for Mycobacterium abscessus in the 12 monthsprior 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 asdetermined by treating physician

  5. Asthma related exacerbation at enrollment as defined by the treating physician basedon 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 ofthe study data as determined by site investigator

Study Design

Total Participants: 76
Treatment Group(s): 2
Primary Treatment: Placebos
Phase: 3
Study Start date:
May 01, 2017
Estimated Completion Date:
October 07, 2023

Connect with a study center

  • The Governers of The University of Calgary - Alberta Health Services

    Calgary, Alberta T2N 4N1
    Canada

    Site Not Available

  • British Columbia Children's Hospital

    Vancouver, British Columbia V6H 3N1
    Canada

    Site Not Available

  • St. Paul's Hospital

    Vancouver, British Columbia V6Z 1Y6
    Canada

    Site Not Available

  • London Health Sciences Centre - Lawson Health Research Institute

    London, Ontario N6C 2R5
    Canada

    Site Not Available

  • The Ottawa Hospital

    Ottawa, Ontario K1H 8L6
    Canada

    Site Not Available

  • SickKids

    Toronto, Ontario M5G1X8
    Canada

    Site Not Available

  • Unity Health Toronto - St. Michael's Hospital

    Toronto, Ontario M5B 1W8
    Canada

    Site Not Available

  • Centre hospitalier universitaire Sainte-Justine

    Montréal, Quebec H3T 1C5
    Canada

    Site Not Available

  • The Centre hospitalier de l'Université de Montréal (CHUM)

    Montréal, Quebec H2X A09
    Canada

    Site Not Available

  • CHU de Quebec-Universite Laval

    Quebec City, Quebec G1R 2J6
    Canada

    Site Not Available

  • Institut universitaire de cardiologie et de pneumologie de Québec - Université Laval

    Quebec City, Quebec G1V 4G5
    Canada

    Site Not Available

  • University of Saskatchewan - Saskatchewan Health Authority

    Saskatoon, Saskatchewan S7N 0W8
    Canada

    Site Not Available

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