Prednisone in Cystic Fibrosis Pulmonary Exacerbations
- Conditions
- Cystic Fibrosis Pulmonary Exacerbation
- Interventions
- Drug: Placebos
- Registration Number
- NCT03070522
- Lead Sponsor
- The Hospital for Sick Children
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 76
- Diagnosis of CF by newborn screening or at least one clinical feature of CF, AND either (a) or (b) as follows:
- A documented sweat chloride ≥ 60 mEq/L by quantitative pilocarpine iontophoresis
- A genotype with two identifiable CF-causing mutations
- Age > 6 years old.
- Acute pulmonary exacerbation treated with IV antibiotics as previously defined 10% relative drop in FEV1 from baseline at the time of exacerbation
- Informed consent by patient or parent/legal guardian
- Ability to reproducibly perform pulmonary function testing
- Ability to comply with medication use including the ability to take capsules, study visits and study procedures as judged by the site investigator
- A respiratory tract culture positive for Burkholderia cenocepacia in the 12 months prior to enrollment
- A respiratory tract culture positive for Mycobacterium abscessus in the 12 months prior to enrollment
- Treatment with IV or oral corticosteroids within 2 weeks of enrollment or from Day 0-Day 7 of the pulmonary exacerbation
- Active allergic bronchopulmonary aspergillosis (ABPA) at the time of enrollment as determined by treating physician
- Asthma related exacerbation at enrollment as defined by the treating physician based on clinically compatible symptoms (eg. wheeze)
- History of avascular necrosis or pathologic bone fracture
- Uncontrolled hypertension with end organ damage
- Active gastrointestinal bleeding
- Status post lung or other organ transplantation
- Pregnancy
- Lactose intolerance (contained in placebo)
- On Lumacaftor-Ivacaftor (Orkambi) at the time of exacerbation
- Investigational drug use within 30 days prior to enrollment visit
- Physical findings that would compromise the safety of the subject or the quality of the study data as determined by site investigator
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo Placebos Placebo Treatment Prednisone Prednisone
- Primary Outcome Measures
Name Time Method 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
Name Time Method lung function recovery at follow up visit 1 month follow up The proportion of subjects who achieve \>90% of their baseline FEV1 % predicted
change in pulmonary function testing at day 7, 14 and 1 month follow up change in pulmonary function testing
quality of life as measured by CFQ-R questionnaire at day 7, 14 and 1 month follow up quality of life
quality of life as measured by CF Respiratory Symptom Diary at day 7, 14 and 1 month follow up quality of life
change in sputum inflammatory markers at day 7, 14 and 1 month follow up change in sputum inflammatory markers
time to subsequent pulmonary exacerbation 1 year follow up time time to subsequent pulmonary exacerbation
change in serum inflammatory markers at day 7, 14 and 1 month follow up change in serum inflammatory markers
number of adverse events At day day 14 of antibiotic therapy and 1 month follow up number of adverse events
length of hospitalization Through study completion, up to 100 weeks length of hospitalization
Duration of antibiotic treatment Through study completion, up to 100 weeks Duration of antibiotic treatment
Trial Locations
- Locations (12)
The Governers of The University of Calgary - Alberta Health Services
🇨🇦Calgary, Alberta, Canada
British Columbia Children's Hospital
🇨🇦Vancouver, British Columbia, Canada
St. Paul's Hospital
🇨🇦Vancouver, British Columbia, Canada
London Health Sciences Centre - Lawson Health Research Institute
🇨🇦London, Ontario, Canada
The Ottawa Hospital
🇨🇦Ottawa, Ontario, Canada
Unity Health Toronto - St. Michael's Hospital
🇨🇦Toronto, Ontario, Canada
SickKids
🇨🇦Toronto, Ontario, Canada
The Centre hospitalier de l'Université de Montréal (CHUM)
🇨🇦Montréal, Quebec, Canada
Centre hospitalier universitaire Sainte-Justine
🇨🇦Montréal, Quebec, Canada
CHU de Quebec-Universite Laval
🇨🇦Quebec City, Quebec, Canada
Scroll for more (2 remaining)The Governers of The University of Calgary - Alberta Health Services🇨🇦Calgary, Alberta, Canada