Back to resultsEMPIRE CF: A Phase 2 Study to Evaluate the Efficacy, Safety, and Tolerability of CTX-4430 in Adult Cystic Fibrosis (CF) Patients
Primary Purpose
Cystic Fibrosis
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
CTX-4430
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Cystic Fibrosis
Eligibility Criteria
Inclusion Criteria:
- Forced expiratory volume at one second (FEV1) ≥50 percent predicted at Screening
- At least 1 pulmonary exacerbation in the 12 months before Screening
Exclusion Criteria:
- Pregnant or nursing women
- Medical condition that is unstable, could be adversely impacted by participation in the study, or could impact assessment of the study results
- History of organ transplantation
- History of alcoholism or drug abuse within 2 years before Screening
- Regular use of a high-dose NSAID within 60 days before Screening
Sites / Locations
- University of Alabama
- Providence Health and Services
- Banner University of Arizona Medical Center
- Childrens Hospital Los Angeles
- Pediatric Pulmonary and Cystic Fibrosis Clinic, Stanford University
- University of California Davis Medical Center
- National Jewish Health
- Yale University
- University of Florida
- Central Florida Pulmonary Group
- Emory University
- University of Chicago
- St. Francis Medical Center
- University of Kansas Medical Center
- Maine Medical Center
- Massachusetts General Hospital
- Boston Children's Hospital
- University of Michigan
- Harper University Hospital
- Spectrum Health Butterworth Campus
- University of Mississippi Medical Center
- University of New Mexico
- Albany Medical College
- Columbia University Medical Center
- SUNY Upstate Medical University
- Wake Forest Hospital
- UC Cincinnati Children's Hospital
- University of Oklahoma Health Sciences Center
- Oregon Health & Sciences University
- Hershey Medical Center
- Drexel University
- The Children's Hospital of Philadelphia
- Children's Hospital of Pittsburgh of UPMC
- Sanford Clinical Research
- Universiy of Tennessee Medical Center UHS
- Dell Children's Medical Center
- Cook Children's Hospital
- Virginia Commonwealth University
- Medical College of Wisconsin
- Hôpital Erasme
- UZ Leuven
- University of Calgary
- Ottawa Hospital
- St. Michael's Hospital
- Centre hospitalier de Dunkerque
- Hôpital Albert Michallon
- Hopital Arnaud de Villeneuve
- Hôpital Cochin
- CH Lyon Sud
- Charite' University
- Krankenhaus Donaustauf
- Carl-Gustav-Klinikum Dresden, Mukoviszidose Centrum "Christiane Herzog"
- Ruhrlandklinik Essen
- Institut für klinische Forschung Pneumologie
- Klinikum der Johann Wolfgang Goethe-Universität
- Medizinische Hochschule Hannover
- Universitätsklinikum Jena CF Centre
- Lungenärztliche Praxis München-Pasing
- Klinikum Stuttgart CF Ambulanz
- Ospedali Riuniti di Ancona
- Azienda Ospedaliero Universitaria
- Azienda Ospedaliera A Meyer
- IRCCS Ospedale Pediatrico Bambino
- Ospedale Civile Maggiore
- Belfast City Hospital
- Bristol Royal Infirmary
- Llandough Hospital
- St James's University Hospital
- Liverpool Heart and Chest Hospital
- King's College Hospital
- Royal Brompton Hospital
- University Hospital of South Manchester
- Royal Victoria Infirmary
- University Hospital Southampton
- Royal Stoke University Hospital
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Experimental
Experimental
Placebo Comparator
Arm Label
50 mg CTX-4430
100 mg CTX-4430
Matching Placebo
Arm Description
Once daily oral capsule for 48 weeks
Once daily oral capsule for 48 weeks
Once daily oral capsule for 48 weeks
Outcomes
Primary Outcome Measures
Difference From Placebo in Absolute Change From Baseline in Forced Expiratory Volume in 1 Second Percent Predicted (ppFEV1)
Difference from Placebo in absolute change from Baseline at Week 48 was assessed for FEV1 percent predicted.
Secondary Outcome Measures
Number of Pulmonary Exacerbations Through 48 Weeks
Rate of protocol-defined pulmonary exacerbations reported through the Week 48/Early Termination visit will be annualized where a year is defined by 52 weeks and will be analyzed using a negative binomial regression.
Hazard Ratio Pulmonary Exacerbation While in the Study
Hazard Ratio of pulmonary exacerbation versus placebo for all subjects. Pulmonary exacerbations are defined as treatment with oral, inhaled, or intravenous antibiotic(s) for ≥4 of symptoms/signs per the modified Fuchs criteria.
Subjects Without a Pulmonary Exacerbation While in the Study
Subjects who did not experience a protocol-defined pulmonary exacerbation during the study
Relative Change (Percent Change) From Baseline in ppFEV1
Percent change from Baseline for ppFEV1 at 48 weeks was assessed.
Change From Baseline at 48 Weeks for Forced Vital Capacity Percent Predicted (FVC) and FEF25-75% (Forced Expiratory Flow During the Middle Half of the Forced Vital Capacity) Percent Predicted
Change From Baseline for Specified Biomarkers
Results were only calculated in subjects who had a verifiable result at the Baseline and Week 48 visits.
Change From Baseline for C-reactive Protein (Hs-CRP)
Results were only calculated in subjects who had a verifiable result at the Baseline and Week 48 visits.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02443688
Brief Title
EMPIRE CF: A Phase 2 Study to Evaluate the Efficacy, Safety, and Tolerability of CTX-4430 in Adult Cystic Fibrosis (CF) Patients
Official Title
A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy, Safety, and Tolerability of CTX-4430 Administered Orally Once-Daily for 48 Weeks in Adult Patients With Cystic Fibrosis
Study Type
Interventional
2. Study Status
Record Verification Date
August 2019
Overall Recruitment Status
Completed
Study Start Date
October 30, 2015 (Actual)
Primary Completion Date
April 20, 2018 (Actual)
Study Completion Date
May 16, 2018 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Celtaxsys, Inc.
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
This study is a Phase 2, double-blind, randomized, placebo-controlled study to evaluate the safety and efficacy of CTX-4430 administered once-daily for 48 weeks for treatment of CF.
Detailed Description
This study is a Phase 2, double-blind, randomized, placebo-controlled study to evaluate the safety and efficacy of CTX-4430 administered once-daily for 48 weeks for treatment of CF. A total of 195 pulmonary CF patients that meet all the inclusion and no exclusion criteria and provide written informed consent will be randomized to receive 50 mg CTX-4430, 100 mg CTX-4430, or placebo in a 1:1:1 ratio. Follow-up visits will be conducted approximately every 4 weeks from Week 4 to Week 52 (4 weeks after completion of treatment).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
200 (Actual)
8. Arms, Groups, and Interventions
Arm Title
50 mg CTX-4430
Arm Type
Experimental
Arm Description
Once daily oral capsule for 48 weeks
Arm Title
100 mg CTX-4430
Arm Type
Experimental
Arm Description
Once daily oral capsule for 48 weeks
Arm Title
Matching Placebo
Arm Type
Placebo Comparator
Arm Description
Once daily oral capsule for 48 weeks
Intervention Type
Drug
Intervention Name(s)
CTX-4430
Intervention Type
Drug
Intervention Name(s)
Placebo
Primary Outcome Measure Information:
Title
Difference From Placebo in Absolute Change From Baseline in Forced Expiratory Volume in 1 Second Percent Predicted (ppFEV1)
Description
Difference from Placebo in absolute change from Baseline at Week 48 was assessed for FEV1 percent predicted.
Time Frame
Baseline, Week 48
Secondary Outcome Measure Information:
Title
Number of Pulmonary Exacerbations Through 48 Weeks
Description
Rate of protocol-defined pulmonary exacerbations reported through the Week 48/Early Termination visit will be annualized where a year is defined by 52 weeks and will be analyzed using a negative binomial regression.
Time Frame
Week 48
Title
Hazard Ratio Pulmonary Exacerbation While in the Study
Description
Hazard Ratio of pulmonary exacerbation versus placebo for all subjects. Pulmonary exacerbations are defined as treatment with oral, inhaled, or intravenous antibiotic(s) for ≥4 of symptoms/signs per the modified Fuchs criteria.
Time Frame
Week 48
Title
Subjects Without a Pulmonary Exacerbation While in the Study
Description
Subjects who did not experience a protocol-defined pulmonary exacerbation during the study
Time Frame
Week 48
Title
Relative Change (Percent Change) From Baseline in ppFEV1
Description
Percent change from Baseline for ppFEV1 at 48 weeks was assessed.
Time Frame
Baseline, Week 48
Title
Change From Baseline at 48 Weeks for Forced Vital Capacity Percent Predicted (FVC) and FEF25-75% (Forced Expiratory Flow During the Middle Half of the Forced Vital Capacity) Percent Predicted
Time Frame
Baseline, Week 48
Title
Change From Baseline for Specified Biomarkers
Description
Results were only calculated in subjects who had a verifiable result at the Baseline and Week 48 visits.
Time Frame
Baseline, Week 48
Title
Change From Baseline for C-reactive Protein (Hs-CRP)
Description
Results were only calculated in subjects who had a verifiable result at the Baseline and Week 48 visits.
Time Frame
Baseline, Week 48
Other Pre-specified Outcome Measures:
Title
Number of Pulmonary Exacerbation Per Year for Participants With ppFEV1 >75 at Baseline
Description
Rate of protocol-defined pulmonary exacerbations reported through the Week 48/Early Termination visit will be annualized where a year is defined by 52 weeks and will be analyzed using a negative binomial regression.
Time Frame
Week 48
Title
Hazard Ratio Pulmonary Exacerbation for Participants With ppFEV1 >75 at Baseline
Description
Hazard ratio of pulmonary exacerbation versus placebo for all subjects
Time Frame
Week 48
Title
Subjects Without a Pulmonary Exacerbation by Participants With ppFEV1 >75 at Baseline
Description
Subjects who did not experience a protocol-defined pulmonary exacerbation during the study.
Time Frame
Week 48
Title
Number of Pulmonary Exacerbation by Subjects if Taking CFTR-Modulator Therapy at Baseline
Description
Rate of protocol-defined pulmonary exacerbations reported through the Week 48/Early Termination visit will be annualized where a year is defined by 52 weeks and will be analyzed using a negative binomial regression.
Time Frame
Week 48
Title
Hazard Ratio Pulmonary Exacerbation by Subjects if Taking CFTR-Modulator Therapy at Baseline
Description
Hazard Ratio pulmonary exacerbation versus placebo for all subjects taking CFTR-modulating therapy at Baseline
Time Frame
Week 48
Title
Subjects Without a Pulmonary Exacerbation by Subjects if Taking CFTR-Modulator Therapy at Baseline
Description
Subjects who did not experience a protocol-defined pulmonary exacerbation during the study.
Time Frame
Week 48
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
30 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Forced expiratory volume at one second (FEV1) ≥50 percent predicted at Screening
At least 1 pulmonary exacerbation in the 12 months before Screening
Exclusion Criteria:
Pregnant or nursing women
Medical condition that is unstable, could be adversely impacted by participation in the study, or could impact assessment of the study results
History of organ transplantation
History of alcoholism or drug abuse within 2 years before Screening
Regular use of a high-dose NSAID within 60 days before Screening
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Steven Rowe, MD
Organizational Affiliation
University of Alabama at Birmingham, USA
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Stuart Elborn, MD
Organizational Affiliation
Royal Brompton Hospital, London UK
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Alabama
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35233
Country
United States
Facility Name
Providence Health and Services
City
Anchorage
State/Province
Alaska
ZIP/Postal Code
99508
Country
United States
Facility Name
Banner University of Arizona Medical Center
City
Tucson
State/Province
Arizona
ZIP/Postal Code
85724
Country
United States
Facility Name
Childrens Hospital Los Angeles
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Facility Name
Pediatric Pulmonary and Cystic Fibrosis Clinic, Stanford University
City
Palo Alto
State/Province
California
ZIP/Postal Code
94304
Country
United States
Facility Name
University of California Davis Medical Center
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
National Jewish Health
City
Denver
State/Province
Colorado
ZIP/Postal Code
80206
Country
United States
Facility Name
Yale University
City
New Haven
State/Province
Connecticut
ZIP/Postal Code
06520
Country
United States
Facility Name
University of Florida
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
Country
United States
Facility Name
Central Florida Pulmonary Group
City
Orlando
State/Province
Florida
ZIP/Postal Code
32803
Country
United States
Facility Name
Emory University
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
University of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60637
Country
United States
Facility Name
St. Francis Medical Center
City
Peoria
State/Province
Illinois
ZIP/Postal Code
61637
Country
United States
Facility Name
University of Kansas Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
Maine Medical Center
City
Portland
State/Province
Maine
ZIP/Postal Code
04106
Country
United States
Facility Name
Massachusetts General Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
University of Michigan
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
49286
Country
United States
Facility Name
Harper University Hospital
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48201
Country
United States
Facility Name
Spectrum Health Butterworth Campus
City
Grand Rapids
State/Province
Michigan
ZIP/Postal Code
49546
Country
United States
Facility Name
University of Mississippi Medical Center
City
Jackson
State/Province
Mississippi
ZIP/Postal Code
39216
Country
United States
Facility Name
University of New Mexico
City
Albuquerque
State/Province
New Mexico
ZIP/Postal Code
87131
Country
United States
Facility Name
Albany Medical College
City
Albany
State/Province
New York
ZIP/Postal Code
12208
Country
United States
Facility Name
Columbia University Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
SUNY Upstate Medical University
City
Syracuse
State/Province
New York
ZIP/Postal Code
13210
Country
United States
Facility Name
Wake Forest Hospital
City
Winston-Salem
State/Province
North Carolina
ZIP/Postal Code
27157
Country
United States
Facility Name
UC Cincinnati Children's Hospital
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45267
Country
United States
Facility Name
University of Oklahoma Health Sciences Center
City
Oklahoma City
State/Province
Oklahoma
ZIP/Postal Code
73104
Country
United States
Facility Name
Oregon Health & Sciences University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Hershey Medical Center
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
Facility Name
Drexel University
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
The Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Children's Hospital of Pittsburgh of UPMC
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
Facility Name
Sanford Clinical Research
City
Sioux Falls
State/Province
South Dakota
ZIP/Postal Code
57104
Country
United States
Facility Name
Universiy of Tennessee Medical Center UHS
City
Knoxville
State/Province
Tennessee
ZIP/Postal Code
37920
Country
United States
Facility Name
Dell Children's Medical Center
City
Austin
State/Province
Texas
ZIP/Postal Code
78723
Country
United States
Facility Name
Cook Children's Hospital
City
Fort Worth
State/Province
Texas
ZIP/Postal Code
76104
Country
United States
Facility Name
Virginia Commonwealth University
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23298
Country
United States
Facility Name
Medical College of Wisconsin
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53226
Country
United States
Facility Name
Hôpital Erasme
City
Brussels
Country
Belgium
Facility Name
UZ Leuven
City
Leuven
Country
Belgium
Facility Name
University of Calgary
City
Calgary
State/Province
Alberta
ZIP/Postal Code
T2N 4N1
Country
Canada
Facility Name
Ottawa Hospital
City
Ottawa
State/Province
Ontario
ZIP/Postal Code
K1H 8L6
Country
Canada
Facility Name
St. Michael's Hospital
City
Toronto
State/Province
Ontario
ZIP/Postal Code
m5B 1W8
Country
Canada
Facility Name
Centre hospitalier de Dunkerque
City
Dunkerque
Country
France
Facility Name
Hôpital Albert Michallon
City
Grenoble
Country
France
Facility Name
Hopital Arnaud de Villeneuve
City
Montpellier
ZIP/Postal Code
Cedex 5
Country
France
Facility Name
Hôpital Cochin
City
Paris
Country
France
Facility Name
CH Lyon Sud
City
Pierre Benite
Country
France
Facility Name
Charite' University
City
Berlin
ZIP/Postal Code
10117
Country
Germany
Facility Name
Krankenhaus Donaustauf
City
Donaustauf
Country
Germany
Facility Name
Carl-Gustav-Klinikum Dresden, Mukoviszidose Centrum "Christiane Herzog"
City
Dresden
Country
Germany
Facility Name
Ruhrlandklinik Essen
City
Essen
Country
Germany
Facility Name
Institut für klinische Forschung Pneumologie
City
Frankfurt
Country
Germany
Facility Name
Klinikum der Johann Wolfgang Goethe-Universität
City
Frankfurt
Country
Germany
Facility Name
Medizinische Hochschule Hannover
City
Hannover
Country
Germany
Facility Name
Universitätsklinikum Jena CF Centre
City
Jena
ZIP/Postal Code
07740
Country
Germany
Facility Name
Lungenärztliche Praxis München-Pasing
City
München-Pasing
Country
Germany
Facility Name
Klinikum Stuttgart CF Ambulanz
City
Stuttgart
Country
Germany
Facility Name
Ospedali Riuniti di Ancona
City
Ancona
Country
Italy
Facility Name
Azienda Ospedaliero Universitaria
City
Catania
Country
Italy
Facility Name
Azienda Ospedaliera A Meyer
City
Florence
Country
Italy
Facility Name
IRCCS Ospedale Pediatrico Bambino
City
Rome
Country
Italy
Facility Name
Ospedale Civile Maggiore
City
Verona
Country
Italy
Facility Name
Belfast City Hospital
City
Belfast
Country
United Kingdom
Facility Name
Bristol Royal Infirmary
City
Bristol
ZIP/Postal Code
BS2 8HW
Country
United Kingdom
Facility Name
Llandough Hospital
City
Cardiff
ZIP/Postal Code
CF64 2XX
Country
United Kingdom
Facility Name
St James's University Hospital
City
Leeds
ZIP/Postal Code
LS97TF
Country
United Kingdom
Facility Name
Liverpool Heart and Chest Hospital
City
Liverpool
Country
United Kingdom
Facility Name
King's College Hospital
City
London
ZIP/Postal Code
SE5 9RS
Country
United Kingdom
Facility Name
Royal Brompton Hospital
City
London
Country
United Kingdom
Facility Name
University Hospital of South Manchester
City
Manchester
Country
United Kingdom
Facility Name
Royal Victoria Infirmary
City
Newcastle Upon Tyne
Country
United Kingdom
Facility Name
University Hospital Southampton
City
Southampton
Country
United Kingdom
Facility Name
Royal Stoke University Hospital
City
Stoke on Trent
Country
United Kingdom
12. IPD Sharing Statement
Learn more about this trial
EMPIRE CF: A Phase 2 Study to Evaluate the Efficacy, Safety, and Tolerability of CTX-4430 in Adult Cystic Fibrosis (CF) Patients
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