Prednisone in Cystic Fibrosis Pulmonary Exacerbations (PIPE)

June 27, 2024 updated by: Valerie Waters, The Hospital for Sick Children

Randomized Controlled Trial of Prednisone in Cystic Fibrosis Pulmonary Exacerbations

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
76

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Contact Backup

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Active Comparator: Placebo
Drug: Placebos
Placebo
Active Comparator: Treatment
Prednisone
Drug: Prednisone
oral prednisone for 7 days during pulmonary exacerbation

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Lung function recovery
Time Frame: 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.
At 14 days of antibiotic therapy

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
lung function recovery at follow up visit
Time Frame: 1 month follow up
The proportion of subjects who achieve >90% of their baseline FEV1 % predicted
1 month follow up
change in pulmonary function testing
Time Frame: at day 7, 14 and 1 month follow up
change in pulmonary function testing
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
quality of life
at day 7, 14 and 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
at day 7, 14 and 1 month follow up
length of hospitalization
Time Frame: Through study completion, up to 100 weeks
length of hospitalization
Through study completion, up to 100 weeks
time to subsequent pulmonary exacerbation
Time Frame: 1 year follow up time
time to subsequent pulmonary exacerbation
1 year follow up time
number of adverse events
Time Frame: At day day 14 of antibiotic therapy and 1 month follow up
number of adverse events
At day day 14 of antibiotic therapy and 1 month follow up
change in sputum inflammatory markers
Time Frame: at day 7, 14 and 1 month follow up
change in sputum inflammatory markers
at day 7, 14 and 1 month follow up
change in serum inflammatory markers
Time Frame: at day 7, 14 and 1 month follow up
change in serum inflammatory markers
at day 7, 14 and 1 month follow up
Duration of antibiotic treatment
Time Frame: Through study completion, up to 100 weeks
Duration of antibiotic treatment
Through study completion, up to 100 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
The Hospital for Sick Children

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Canadian Cystic Fibrosis Foundation

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
May 1, 2017

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
October 7, 2023

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
October 7, 2023

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
February 8, 2017

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 28, 2017

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
March 3, 2017

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
July 1, 2024

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 27, 2024

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
June 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • REB1000053825

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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