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)
• Enrollment: 84
• Locations: 12
• Arms: 2
• Anticipated Duration (Months): 72
• Patients Per Site: 7
• Patients Per Site Per Month: 0.1

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
Cystic Fibrosis Pulmonary Exacerbation	Drug: Prednisone; Drug: Placebos	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: Cystic Fibrosis; Pulmonary Fibrosis; Fibrosis; Prednisone

Study Results

No Results Posted as of Jun 9, 2021