Cystic Fibrosis Pulmonary ExacerbationPhase 3PIPENCT03070522
Prednisone in Cystic Fibrosis Pulmonary Exacerbations
A Phase 3 study testing Prednisone in people with Cystic Fibrosis Pulmonary Exacerbation, run by The Hospital for Sick Children at 12 sites in Canada.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Cystic Fibrosis Pulmonary Exacerbation
- What is being tested?
- Prednisone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 76 participants
- Where
- 12 sitesCanada
- Study dates
- 1 May 2017to 7 Oct 2023
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 14Show fewer
- 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
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
Canada12 sites
The Governers of The University of Calgary - Alberta Health Services
Calgary, Alberta, T2N 4N1
London Health Sciences Centre - Lawson Health Research Institute
London, Ontario, N6C 2R5
The Centre hospitalier de l'Université de Montréal (CHUM)
Montreal, Quebec, H2X A09
Centre hospitalier universitaire Sainte-Justine
Montreal, Quebec, H3T 1C5
The Ottawa Hospital
Ottawa, Ontario, K1H 8L6
CHU de Quebec-Universite Laval
Québec, Quebec, G1R 2J6
Institut universitaire de cardiologie et de pneumologie de Québec - Université Laval
Québec, Quebec, G1V 4G5
University of Saskatchewan - Saskatchewan Health Authority
Saskatoon, Saskatchewan, S7N 0W8
Unity Health Toronto - St. Michael's Hospital
Toronto, Ontario, M5B 1W8
SickKids
Toronto, Ontario, M5G1X8
British Columbia Children's Hospital
Vancouver, British Columbia, V6H 3N1
St. Paul's Hospital
Vancouver, British Columbia, V6Z 1Y6
About this study
Official title: 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.
- Conditions
- Cystic Fibrosis Pulmonary Exacerbation
- Being tested
- Prednisone, Placebos
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PrednisoneDrug
oral prednisone for 7 days during pulmonary exacerbation
PlacebosDrug
Placebo
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Active comparator | Placebo Receives: Drug: Placebos |
| Treatment | Active comparator | Prednisone Receives: Drug: Prednisone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Lung function recovery
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.
Measured: At 14 days of antibiotic therapy
Secondary outcomes (10)
lung function recovery at follow up visit
The proportion of subjects who achieve >90% of their baseline FEV1 % predicted
Measured: 1 month follow up
change in pulmonary function testing
change in pulmonary function testing
Measured: at day 7, 14 and 1 month follow up
quality of life as measured by CFQ-R questionnaire
quality of life
Measured: at day 7, 14 and 1 month follow up
quality of life as measured by CF Respiratory Symptom Diary
quality of life
Measured: at day 7, 14 and 1 month follow up
length of hospitalization
length of hospitalization
Measured: Through study completion, up to 100 weeks
time to subsequent pulmonary exacerbation
time to subsequent pulmonary exacerbation
Measured: 1 year follow up time
number of adverse events
number of adverse events
Measured: At day day 14 of antibiotic therapy and 1 month follow up
change in sputum inflammatory markers
change in sputum inflammatory markers
Measured: at day 7, 14 and 1 month follow up
change in serum inflammatory markers
change in serum inflammatory markers
Measured: at day 7, 14 and 1 month follow up
Duration of antibiotic treatment
Duration of antibiotic treatment
Measured: Through study completion, up to 100 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Mar 2017
- Study start1 May 2017
- Primary completion7 Oct 2023
- Study completion7 Oct 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Cystic Fibrosis Pulmonary ExacerbationStudies of the same condition, open and under way.
- More trials run by The Hospital for Sick ChildrenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Randomized placebo controlled trial
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 76 participants (actual)
Sponsor and investigators
- Lead sponsor
- The Hospital for Sick Children
- Sponsor type
- University, hospital or other
- Collaborators
- Canadian Cystic Fibrosis Foundation
- Responsible party
- Principal investigator, Valerie Waters, The Hospital for Sick Children
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03070522
- Sponsor's protocol ID
- REB1000053825
- Acronym
- PIPE
- Status last verified
- Jun 2024
- First submitted
- 8 Feb 2017
- Last updated
- 1 Jul 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Waters V, Shaw M, Perrem L, Quon BS, Tullis E, Solomon M, Rayment JH, Lavoie A, Tse SM, Daigneault P, Bilodeau L, Price A, Nicholson M, Chin M, Parkins M, McKinney ML, Tam JS, Stanojevic S, Grasemann H, Ratjen F; (PIPE Study Investigators). A randomised trial of oral prednisone for cystic fibrosis pulmonary exacerbation treatment. Eur Respir J. 2024 Jun 6;63(6):2302278. doi: 10.1183/13993003.02278-2023. Print 2024 Jun.
PubMed 38697648
Study at a glance
From the registry record, last updated by the sponsor on 1 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized Controlled Trial of Prednisone in Cystic Fibrosis Pulmonary Exacerbations
- ClinicalTrials.gov ID
- NCT03070522
- Acronym / sponsor ID
- PIPE / REB1000053825
- Conditions
- Cystic Fibrosis Pulmonary Exacerbation
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 76 participants (actual)
- Lead sponsor
- The Hospital for Sick ChildrenUniversity, hospital or other
- Collaborators
- Canadian Cystic Fibrosis Foundation
- Study start
- 1 May 2017 (actual)
- Primary completion
- 7 Oct 2023 (actual)
- Study completion
- 7 Oct 2023 (actual)
- First posted
- 3 Mar 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Randomized placebo controlled trial
| Arm | Type | Receives |
|---|---|---|
| Placebo | Active comparator | Drug: PlacebosPlacebo |
| Treatment | Active comparator | Drug: PrednisonePrednisone |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- 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
Key exclusion criteria (14 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Prednisoneoral prednisone for 7 days during pulmonary exacerbation | Drug | Treatment |
| PlacebosPlacebo | Drug | Placebo |
Primary outcome
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.
10 secondary outcomes
lung function recovery at follow up visit
Time frame: 1 month follow up
change in pulmonary function testing
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
quality of life as measured by CF Respiratory Symptom Diary
Time frame: at day 7, 14 and 1 month follow up
length of hospitalization
Time frame: Through study completion, up to 100 weeks
time to subsequent pulmonary exacerbation
Time frame: 1 year follow up time
number of adverse events
Time frame: 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 serum inflammatory markers
Time frame: at day 7, 14 and 1 month follow up
Duration of antibiotic treatment
Time frame: Through study completion, up to 100 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Jul 2024.
- Results publication: PubMed 38697648Waters V, Shaw M, Perrem L, Quon BS, Tullis E, Solomon M, Rayment JH, Lavoie A, Tse SM, Daigneault P, Bilodeau L, Price A, Nicholson M, Chin M, Parkins M, McKinney ML, Tam JS, Stanojevic S, Grasemann H, Ratjen F; (PIPE Study Investigators). A randomised trial of oral prednisone for cystic fibrosis pulmonary exacerbation treatment. Eur Respir J. 2024 Jun 6;63(6):2302278. doi: 10.1183/13993003.02278-2023. Print 2024 Jun.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Prednisone in people with Cystic Fibrosis Pulmonary Exacerbation, run by The Hospital for Sick Children at 12 sites in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Lung function recovery (time frame: At 14 days of antibiotic therapy). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03070522, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Jul 2024.