EMPIRE CF: A Phase 2 Study to Evaluate the Efficacy, Safety, and Tolerability of CTX-4430 in Adult Cystic Fibrosis (CF) Patients

NCT02443688 | Completed Phase 2 Results DMC

• 1 other identifier: CTX-4430-CF-201
• Study Type: interventional
• Target: 200
• Locations: 7 countries
• Sites: 75

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.

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

• 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.

  Baseline, Week 48

Secondary Outcomes (7)

• Number of Pulmonary Exacerbations Through 48 Weeks

  Week 48

• Hazard Ratio Pulmonary Exacerbation While in the Study

  Week 48

• Subjects Without a Pulmonary Exacerbation While in the Study

  Week 48

• Relative Change (Percent Change) From Baseline in ppFEV1

  Baseline, Week 48

• 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

Other Outcomes (6)

• Number of Pulmonary Exacerbation Per Year for Participants With ppFEV1 >75 at Baseline

  Week 48

• Hazard Ratio Pulmonary Exacerbation for Participants With ppFEV1 >75 at Baseline

  Week 48

• Subjects Without a Pulmonary Exacerbation by Participants With ppFEV1 >75 at Baseline

  Week 48

Study Arms (3)

50 mg CTX-4430

EXPERIMENTAL

Once daily oral capsule for 48 weeks

Drug: CTX-4430

100 mg CTX-4430

EXPERIMENTAL

Once daily oral capsule for 48 weeks

Drug: CTX-4430

Matching Placebo

PLACEBO COMPARATOR

Once daily oral capsule for 48 weeks

Drug: Placebo

Interventions

CTX-4430 DRUG

100 mg CTX-4430; 50 mg CTX-4430

Placebo DRUG

Matching Placebo

Eligibility Criteria

• Age: 18 Years - 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Forced expiratory volume at one second (FEV1) ≥50 percent predicted at Screening
• At least 1 pulmonary exacerbation in the 12 months before Screening

You may not qualify if:

• 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

Sponsors & Collaborators

• Celtaxsys, Inc.: lead

Study Sites (75)

University of Alabama

Birmingham, Alabama, 35233, United States

Location

Providence Health and Services

Anchorage, Alaska, 99508, United States

Location

Banner University of Arizona Medical Center

Tucson, Arizona, 85724, United States

Location

Childrens Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

Pediatric Pulmonary and Cystic Fibrosis Clinic, Stanford University

Palo Alto, California, 94304, United States

Location

University of California Davis Medical Center

Sacramento, California, 95817, United States

Location

National Jewish Health

Denver, Colorado, 80206, United States

Location

Yale University

New Haven, Connecticut, 06520, United States

Location

University of Florida

Gainesville, Florida, 32610, United States

Location

Central Florida Pulmonary Group

Orlando, Florida, 32803, United States

Location

Emory University

Atlanta, Georgia, 30322, United States

Location

University of Chicago

Chicago, Illinois, 60637, United States

Location

St. Francis Medical Center

Peoria, Illinois, 61637, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

Maine Medical Center

Portland, Maine, 04106, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

University of Michigan

Ann Arbor, Michigan, 49286, United States

Location

Harper University Hospital

Detroit, Michigan, 48201, United States

Location

Spectrum Health Butterworth Campus

Grand Rapids, Michigan, 49546, United States

Location

University of Mississippi Medical Center

Jackson, Mississippi, 39216, United States

Location

University of New Mexico

Albuquerque, New Mexico, 87131, United States

Location

Albany Medical College

Albany, New York, 12208, United States

Location

Columbia University Medical Center

New York, New York, 10032, United States

Location

SUNY Upstate Medical University

Syracuse, New York, 13210, United States

Location

Wake Forest Hospital

Winston-Salem, North Carolina, 27157, United States

Location

UC Cincinnati Children's Hospital

Cincinnati, Ohio, 45267, United States

Location

University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, 73104, United States

Location

Oregon Health & Sciences University

Portland, Oregon, 97239, United States

Location

Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

Drexel University

Philadelphia, Pennsylvania, 19104, United States

Location

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, 15224, United States

Location

Sanford Clinical Research

Sioux Falls, South Dakota, 57104, United States

Location

Universiy of Tennessee Medical Center UHS

Knoxville, Tennessee, 37920, United States

Location

Dell Children's Medical Center

Austin, Texas, 78723, United States

Location

Cook Children's Hospital

Fort Worth, Texas, 76104, United States

Location

Virginia Commonwealth University

Richmond, Virginia, 23298, United States

Location

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

Hôpital Erasme

Brussels, Belgium

Location

UZ Leuven

Leuven, Belgium

Location

University of Calgary

Calgary, Alberta, T2N 4N1, Canada

Location

Ottawa Hospital

Ottawa, Ontario, K1H 8L6, Canada

Location

St. Michael's Hospital

Toronto, Ontario, m5B 1W8, Canada

Location

Centre hospitalier de Dunkerque

Dunkirk, France

Location

Hôpital Albert Michallon

Grenoble, France

Location

Hopital Arnaud de Villeneuve

Montpellier, Cedex 5, France

Location

Hôpital Cochin

Paris, France

Location

CH Lyon Sud

Pierre-Bénite, France

Location

Charite' University

Berlin, 10117, Germany

Location

Krankenhaus Donaustauf

Donaustauf, Germany

Location

Carl-Gustav-Klinikum Dresden, Mukoviszidose Centrum "Christiane Herzog"

Dresden, Germany

Location

Ruhrlandklinik Essen

Essen, Germany

Location

Institut für klinische Forschung Pneumologie

Frankfurt, Germany

Location

Klinikum der Johann Wolfgang Goethe-Universität

Frankfurt, Germany

Location

Medizinische Hochschule Hannover

Hanover, Germany

Location

Universitätsklinikum Jena CF Centre

Jena, 07740, Germany

Location

Lungenärztliche Praxis München-Pasing

München-Pasing, Germany

Location

Klinikum Stuttgart CF Ambulanz

Stuttgart, Germany

Location

Ospedali Riuniti di Ancona

Ancona, Italy

Location

Azienda Ospedaliero Universitaria

Catania, Italy

Location

Azienda Ospedaliera A Meyer

Florence, Italy

Location

IRCCS Ospedale Pediatrico Bambino

Rome, Italy

Location

Ospedale Civile Maggiore

Verona, Italy

Location

Belfast City Hospital

Belfast, United Kingdom

Location

Bristol Royal Infirmary

Bristol, BS2 8HW, United Kingdom

Location

Llandough Hospital

Cardiff, CF64 2XX, United Kingdom

Location

St James's University Hospital

Leeds, LS97TF, United Kingdom

Location

Liverpool Heart and Chest Hospital

Liverpool, United Kingdom

Location

King's College Hospital

London, SE5 9RS, United Kingdom

Location

Royal Brompton Hospital

London, United Kingdom

Location

University Hospital of South Manchester

Manchester, United Kingdom

Location

Royal Victoria Infirmary

Newcastle upon Tyne, United Kingdom

Location

University Hospital Southampton

Southampton, United Kingdom

Location

Royal Stoke University Hospital

Stoke-on-Trent, United Kingdom

Location

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

acebilustat

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Results Point of Contact

• Title: Director, Clincal Operations
• Organization: Celtaxsys, Inc.

sbirnbaum@celtaxsys.com

4702060153

Study Officials

• Steven Rowe, MD

  University of Alabama at Birmingham, USA

  PRINCIPAL INVESTIGATOR

• Stuart Elborn, MD

  Royal Brompton Hospital, London UK

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 8, 2015
• First Posted: May 14, 2015
• Study Start: October 30, 2015
• Primary Completion: April 20, 2018
• Study Completion: May 16, 2018
• Last Updated: September 4, 2019
• Results First Posted: July 25, 2019

Record last verified: 2019-08

Locations

GB (11); CA (3); DE (10); US (39); FR (5); IT (5); BE (2)