Prednisone in Cystic Fibrosis Pulmonary Exacerbations

NCT03070522 | Completed Phase 3 DMC

• 1 other identifier: REB1000053825
• Study Type: interventional
• Target: 76
• Locations: 1 country
• Sites: 12

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.

Conditions

Cystic Fibrosis Pulmonary Exacerbation

Outcome Measures

Primary Outcomes (1)

• 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.

  At 14 days of antibiotic therapy

Secondary Outcomes (10)

• lung function recovery at follow up visit

  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

  at day 7, 14 and 1 month follow up

• quality of life as measured by CF Respiratory Symptom Diary

  at day 7, 14 and 1 month follow up

• length of hospitalization

  Through study completion, up to 100 weeks

Study Arms (2)

Placebo

ACTIVE COMPARATOR

Placebo

Drug: Placebos

Treatment

ACTIVE COMPARATOR

Prednisone

Drug: Prednisone

Interventions

Prednisone DRUG

oral prednisone for 7 days during pulmonary exacerbation

Treatment

Placebos DRUG

Placebo

Placebo

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• The Hospital for Sick Children: lead
• Canadian Cystic Fibrosis Foundation: collaborator

Study Sites (12)

The Governers of The University of Calgary - Alberta Health Services

Calgary, Alberta, T2N 4N1, Canada

Location

British Columbia Children's Hospital

Vancouver, British Columbia, V6H 3N1, Canada

Location

St. Paul's Hospital

Vancouver, British Columbia, V6Z 1Y6, Canada

Location

London Health Sciences Centre - Lawson Health Research Institute

London, Ontario, N6C 2R5, Canada

Location

The Ottawa Hospital

Ottawa, Ontario, K1H 8L6, Canada

Location

Unity Health Toronto - St. Michael's Hospital

Toronto, Ontario, M5B 1W8, Canada

Location

SickKids

Toronto, Ontario, M5G1X8, Canada

Location

The Centre hospitalier de l'Université de Montréal (CHUM)

Montreal, Quebec, H2X A09, Canada

Location

Centre hospitalier universitaire Sainte-Justine

Montreal, Quebec, H3T 1C5, Canada

Location

CHU de Quebec-Universite Laval

Québec, Quebec, G1R 2J6, Canada

Location

Institut universitaire de cardiologie et de pneumologie de Québec - Université Laval

Québec, Quebec, G1V 4G5, Canada

Location

University of Saskatchewan - Saskatchewan Health Authority

Saskatoon, Saskatchewan, S7N 0W8, Canada

Location

Related Publications (1)

• 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.

  PMID: 38697648 RESULT

MeSH Terms

Interventions

Prednisone

Intervention Hierarchy (Ancestors)

Pregnadienediols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: placebo
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Staff physician

Model Details: Randomized placebo controlled trial

Study Record Dates

• First Submitted: February 8, 2017
• First Posted: March 3, 2017
• Study Start: May 1, 2017
• Primary Completion: October 7, 2023
• Study Completion: October 7, 2023
• Last Updated: July 1, 2024

Record last verified: 2024-06

Data Sharing

• IPD Sharing: Will not share

Locations

CA (12)