Clinical trial NCT02443688: 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
Overall status: COMPLETED
Phase: PHASE2
Conditions: Cystic Fibrosis
Lead sponsor: Celtaxsys, Inc.
Sponsor class: OTHER
First posted: 2015-05-14
Enrollment: 200
Sex: ALL
Minimum age (years): 18
Maximum age (years): 30
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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).

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

Primary outcomes:
measure: Difference From Placebo in Absolute Change From Baseline in Forced Expiratory Volume in 1 Second Percent Predicted (ppFEV1)
timeFrame: Baseline, Week 48
Secondary outcomes:
measure: Number of Pulmonary Exacerbations Through 48 Weeks
timeFrame: Week 48
measure: Hazard Ratio Pulmonary Exacerbation While in the Study
timeFrame: Week 48
measure: Subjects Without a Pulmonary Exacerbation While in the Study
timeFrame: Week 48
measure: Relative Change (Percent Change) From Baseline in ppFEV1
timeFrame: Baseline, Week 48
measure: 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
timeFrame: Baseline, Week 48
measure: Change From Baseline for Specified Biomarkers
timeFrame: Baseline, Week 48
measure: Change From Baseline for C-reactive Protein (Hs-CRP)
timeFrame: Baseline, Week 48
Other outcomes:
measure: Number of Pulmonary Exacerbation Per Year for Participants With ppFEV1 >75 at Baseline
timeFrame: Week 48
measure: Hazard Ratio Pulmonary Exacerbation for Participants With ppFEV1 >75 at Baseline
timeFrame: Week 48
measure: Subjects Without a Pulmonary Exacerbation by Participants With ppFEV1 >75 at Baseline
timeFrame: Week 48
measure: Number of Pulmonary Exacerbation by Subjects if Taking CFTR-Modulator Therapy at Baseline
timeFrame: Week 48
measure: Hazard Ratio Pulmonary Exacerbation by Subjects if Taking CFTR-Modulator Therapy at Baseline
timeFrame: Week 48
measure: Subjects Without a Pulmonary Exacerbation by Subjects if Taking CFTR-Modulator Therapy at Baseline
timeFrame: Week 48
Locations:
city: Birmingham
country: United States
facility: University of Alabama
state: Alabama
city: Anchorage
country: United States
facility: Providence Health and Services
state: Alaska
city: Tucson
country: United States
facility: Banner University of Arizona Medical Center
state: Arizona
city: Los Angeles
country: United States
facility: Childrens Hospital Los Angeles
state: California
city: Palo Alto
country: United States
facility: Pediatric Pulmonary and Cystic Fibrosis Clinic, Stanford University
state: California
city: Sacramento
country: United States
facility: University of California Davis Medical Center
state: California
city: Denver
country: United States
facility: National Jewish Health
state: Colorado
city: New Haven
country: United States
facility: Yale University
state: Connecticut
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Orlando
country: United States
facility: Central Florida Pulmonary Group
state: Florida
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Chicago
country: United States
facility: University of Chicago
state: Illinois
city: Peoria
country: United States
facility: St. Francis Medical Center
state: Illinois
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Portland
country: United States
facility: Maine Medical Center
state: Maine
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Detroit
country: United States
facility: Harper University Hospital
state: Michigan
city: Grand Rapids
country: United States
facility: Spectrum Health Butterworth Campus
state: Michigan
city: Jackson
country: United States
facility: University of Mississippi Medical Center
state: Mississippi
city: Albuquerque
country: United States
facility: University of New Mexico
state: New Mexico
city: Albany
country: United States
facility: Albany Medical College
state: New York
city: New York
country: United States
facility: Columbia University Medical Center
state: New York
city: Syracuse
country: United States
facility: SUNY Upstate Medical University
state: New York
city: Winston-Salem
country: United States
facility: Wake Forest Hospital
state: North Carolina
city: Cincinnati
country: United States
facility: UC Cincinnati Children's Hospital
state: Ohio
city: Oklahoma City
country: United States
facility: University of Oklahoma Health Sciences Center
state: Oklahoma
city: Portland
country: United States
facility: Oregon Health & Sciences University
state: Oregon
city: Hershey
country: United States
facility: Hershey Medical Center
state: Pennsylvania
city: Philadelphia
country: United States
facility: Drexel University
state: Pennsylvania
city: Philadelphia
country: United States
facility: The Children's Hospital of Philadelphia
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Children's Hospital of Pittsburgh of UPMC
state: Pennsylvania
city: Sioux Falls
country: United States
facility: Sanford Clinical Research
state: South Dakota
city: Knoxville
country: United States
facility: Universiy of Tennessee Medical Center UHS
state: Tennessee
city: Austin
country: United States
facility: Dell Children's Medical Center
state: Texas
city: Fort Worth
country: United States
facility: Cook Children's Hospital
state: Texas
city: Richmond
country: United States
facility: Virginia Commonwealth University
state: Virginia
city: Milwaukee
country: United States
facility: Medical College of Wisconsin
state: Wisconsin
city: Brussels
country: Belgium
facility: Hôpital Erasme
city: Leuven
country: Belgium
facility: UZ Leuven
city: Calgary
country: Canada
facility: University of Calgary
state: Alberta
city: Ottawa
country: Canada
facility: Ottawa Hospital
state: Ontario
city: Toronto
country: Canada
facility: St. Michael's Hospital
state: Ontario
city: Dunkerque
country: France
facility: Centre hospitalier de Dunkerque
city: Grenoble
country: France
facility: Hôpital Albert Michallon
city: Montpellier
country: France
facility: Hopital Arnaud de Villeneuve
city: Paris
country: France
facility: Hôpital Cochin
city: Pierre Benite
country: France
facility: CH Lyon Sud
city: Berlin
country: Germany
facility: Charite' University
city: Donaustauf
country: Germany
facility: Krankenhaus Donaustauf
city: Dresden
country: Germany
facility: Carl-Gustav-Klinikum Dresden, Mukoviszidose Centrum "Christiane Herzog"
city: Essen
country: Germany
facility: Ruhrlandklinik Essen
city: Frankfurt
country: Germany
facility: Institut für klinische Forschung Pneumologie
city: Frankfurt
country: Germany
facility: Klinikum der Johann Wolfgang Goethe-Universität
city: Hannover
country: Germany
facility: Medizinische Hochschule Hannover
city: Jena
country: Germany
facility: Universitätsklinikum Jena CF Centre
city: München-Pasing
country: Germany
facility: Lungenärztliche Praxis München-Pasing
city: Stuttgart
country: Germany
facility: Klinikum Stuttgart CF Ambulanz
city: Ancona
country: Italy
facility: Ospedali Riuniti di Ancona
city: Catania
country: Italy
facility: Azienda Ospedaliero Universitaria
city: Florence
country: Italy
facility: Azienda Ospedaliera A Meyer
city: Rome
country: Italy
facility: IRCCS Ospedale Pediatrico Bambino
city: Verona
country: Italy
facility: Ospedale Civile Maggiore
city: Belfast
country: United Kingdom
facility: Belfast City Hospital
city: Bristol
country: United Kingdom
facility: Bristol Royal Infirmary
city: Cardiff
country: United Kingdom
facility: Llandough Hospital
city: Leeds
country: United Kingdom
facility: St James's University Hospital
city: Liverpool
country: United Kingdom
facility: Liverpool Heart and Chest Hospital
city: London
country: United Kingdom
facility: King's College Hospital
city: London
country: United Kingdom
facility: Royal Brompton Hospital
city: Manchester
country: United Kingdom
facility: University Hospital of South Manchester
city: Newcastle Upon Tyne
country: United Kingdom
facility: Royal Victoria Infirmary
city: Southampton
country: United Kingdom
facility: University Hospital Southampton
city: Stoke on Trent
country: United Kingdom
facility: Royal Stoke University Hospital
Study officials:
affiliation: University of Alabama at Birmingham, USA
name: Steven Rowe, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Royal Brompton Hospital, London UK
name: Stuart Elborn, MD
role: PRINCIPAL_INVESTIGATOR
References: