Prednisone in Cystic Fibrosis Pulmonary Exacerbations

• Register: ClinicalTrials.gov
• Last refreshed on: 8 July 2024
• Main ID: NCT03070522
• Date of registration: 08/02/2017
• Prospective Registration: Yes
• Primary sponsor: The Hospital for Sick Children
• Public title: Prednisone in Cystic Fibrosis Pulmonary Exacerbations PIPE
• Scientific title: Randomized Controlled Trial of Prednisone in Cystic Fibrosis Pulmonary Exacerbations
• Date of first enrolment: May 1, 2017
• Target sample size: 76
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT03070522
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

Canada

Key inclusion & exclusion 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


Age minimum: N/A
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis Pulmonary Exacerbation

Intervention(s)

Drug: Prednisone

Drug: Placebos

Primary Outcome(s)

Lung function recovery [Time Frame: At 14 days of antibiotic therapy]

Secondary Outcome(s)

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

lung function recovery at follow up visit [Time Frame: 1 month follow up]

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

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

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

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

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

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

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

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

Secondary ID(s)

REB1000053825

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Canadian Cystic Fibrosis Foundation