EMPIRE CF: A Phase 2 Study to Evaluate the Efficacy, Safety, and Tolerability of CTX-4430 in Adult Cystic Fibrosis (CF) Patients
- Registration Number
- NCT02443688
- Lead Sponsor
- Celtaxsys, Inc.
- 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).
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 200
- Forced expiratory volume at one second (FEV1) ≥50 percent predicted at Screening
- At least 1 pulmonary exacerbation in the 12 months before Screening
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description 50 mg CTX-4430 CTX-4430 Once daily oral capsule for 48 weeks Matching Placebo Placebo Once daily oral capsule for 48 weeks 100 mg CTX-4430 CTX-4430 Once daily oral capsule for 48 weeks
- Primary Outcome Measures
Name Time Method Difference From Placebo in Absolute Change From Baseline in Forced Expiratory Volume in 1 Second Percent Predicted (ppFEV1) Baseline, Week 48 Difference from Placebo in absolute change from Baseline at Week 48 was assessed for FEV1 percent predicted.
- Secondary Outcome Measures
Name Time Method 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 Baseline, Week 48 Number of Pulmonary Exacerbations Through 48 Weeks Week 48 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.
Subjects Without a Pulmonary Exacerbation While in the Study Week 48 Subjects who did not experience a protocol-defined pulmonary exacerbation during the study
Change From Baseline for Specified Biomarkers Baseline, Week 48 Results were only calculated in subjects who had a verifiable result at the Baseline and Week 48 visits.
Hazard Ratio Pulmonary Exacerbation While in the Study Week 48 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.
Relative Change (Percent Change) From Baseline in ppFEV1 Baseline, Week 48 Percent change from Baseline for ppFEV1 at 48 weeks was assessed.
Change From Baseline for C-reactive Protein (Hs-CRP) Baseline, Week 48 Results were only calculated in subjects who had a verifiable result at the Baseline and Week 48 visits.
Trial Locations
- Locations (75)
Childrens Hospital Los Angeles
🇺🇸Los Angeles, California, United States
Emory University
🇺🇸Atlanta, Georgia, United States
Drexel University
🇺🇸Philadelphia, Pennsylvania, United States
University of Chicago
🇺🇸Chicago, Illinois, United States
St. Michael's Hospital
🇨🇦Toronto, Ontario, Canada
Providence Health and Services
🇺🇸Anchorage, Alaska, United States
University of Florida
🇺🇸Gainesville, Florida, United States
Central Florida Pulmonary Group
🇺🇸Orlando, Florida, United States
Universiy of Tennessee Medical Center UHS
🇺🇸Knoxville, Tennessee, United States
CH Lyon Sud
🇫🇷Pierre Benite, France
Krankenhaus Donaustauf
🇩🇪Donaustauf, Germany
Ruhrlandklinik Essen
🇩🇪Essen, Germany
Universitätsklinikum Jena CF Centre
🇩🇪Jena, Germany
Lungenärztliche Praxis München-Pasing
🇩🇪München-Pasing, Germany
Klinikum Stuttgart CF Ambulanz
🇩🇪Stuttgart, Germany
Azienda Ospedaliera A Meyer
🇮🇹Florence, Italy
Hôpital Cochin
🇫🇷Paris, France
Charite' University
🇩🇪Berlin, Germany
University of Calgary
🇨🇦Calgary, Alberta, Canada
Carl-Gustav-Klinikum Dresden, Mukoviszidose Centrum "Christiane Herzog"
🇩🇪Dresden, Germany
Klinikum der Johann Wolfgang Goethe-Universität
🇩🇪Frankfurt, Germany
Medizinische Hochschule Hannover
🇩🇪Hannover, Germany
Ospedali Riuniti di Ancona
🇮🇹Ancona, Italy
Azienda Ospedaliero Universitaria
🇮🇹Catania, Italy
IRCCS Ospedale Pediatrico Bambino
🇮🇹Rome, Italy
Ospedale Civile Maggiore
🇮🇹Verona, Italy
University of Alabama
🇺🇸Birmingham, Alabama, United States
University of California Davis Medical Center
🇺🇸Sacramento, California, United States
Belfast City Hospital
🇬🇧Belfast, United Kingdom
St James's University Hospital
🇬🇧Leeds, United Kingdom
Hôpital Albert Michallon
🇫🇷Grenoble, France
Royal Stoke University Hospital
🇬🇧Stoke on Trent, United Kingdom
Hopital Arnaud de Villeneuve
🇫🇷Montpellier, France
Banner University of Arizona Medical Center
🇺🇸Tucson, Arizona, United States
UZ Leuven
🇧🇪Leuven, Belgium
Liverpool Heart and Chest Hospital
🇬🇧Liverpool, United Kingdom
Centre hospitalier de Dunkerque
🇫🇷Dunkerque, France
University Hospital of South Manchester
🇬🇧Manchester, United Kingdom
Hôpital Erasme
🇧🇪Brussels, Belgium
Ottawa Hospital
🇨🇦Ottawa, Ontario, Canada
Llandough Hospital
🇬🇧Cardiff, United Kingdom
Institut für klinische Forschung Pneumologie
🇩🇪Frankfurt, Germany
Bristol Royal Infirmary
🇬🇧Bristol, United Kingdom
Albany Medical College
🇺🇸Albany, New York, United States
University of New Mexico
🇺🇸Albuquerque, New Mexico, United States
Virginia Commonwealth University
🇺🇸Richmond, Virginia, United States
University of Mississippi Medical Center
🇺🇸Jackson, Mississippi, United States
National Jewish Health
🇺🇸Denver, Colorado, United States
Harper University Hospital
🇺🇸Detroit, Michigan, United States
UC Cincinnati Children's Hospital
🇺🇸Cincinnati, Ohio, United States
Oregon Health & Sciences University
🇺🇸Portland, Oregon, United States
Royal Victoria Infirmary
🇬🇧Newcastle Upon Tyne, United Kingdom
University Hospital Southampton
🇬🇧Southampton, United Kingdom
King's College Hospital
🇬🇧London, United Kingdom
Medical College of Wisconsin
🇺🇸Milwaukee, Wisconsin, United States
Pediatric Pulmonary and Cystic Fibrosis Clinic, Stanford University
🇺🇸Palo Alto, California, United States
Yale University
🇺🇸New Haven, Connecticut, United States
St. Francis Medical Center
🇺🇸Peoria, Illinois, United States
Maine Medical Center
🇺🇸Portland, Maine, United States
Wake Forest Hospital
🇺🇸Winston-Salem, North Carolina, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
University of Michigan
🇺🇸Ann Arbor, Michigan, United States
Spectrum Health Butterworth Campus
🇺🇸Grand Rapids, Michigan, United States
Columbia University Medical Center
🇺🇸New York, New York, United States
SUNY Upstate Medical University
🇺🇸Syracuse, New York, United States
University of Oklahoma Health Sciences Center
🇺🇸Oklahoma City, Oklahoma, United States
The Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Children's Hospital of Pittsburgh of UPMC
🇺🇸Pittsburgh, Pennsylvania, United States
Hershey Medical Center
🇺🇸Hershey, Pennsylvania, United States
Sanford Clinical Research
🇺🇸Sioux Falls, South Dakota, United States
Dell Children's Medical Center
🇺🇸Austin, Texas, United States
Cook Children's Hospital
🇺🇸Fort Worth, Texas, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
Royal Brompton Hospital
🇬🇧London, United Kingdom
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States