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

NCT ID: NCT02443688

Last Updated: 2019-09-04

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

200 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-10-30

Study Completion Date

2018-05-16

Brief Summary

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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

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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).

Conditions

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Cystic Fibrosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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50 mg CTX-4430

Once daily oral capsule for 48 weeks

Group Type EXPERIMENTAL

CTX-4430

Intervention Type DRUG

100 mg CTX-4430

Once daily oral capsule for 48 weeks

Group Type EXPERIMENTAL

CTX-4430

Intervention Type DRUG

Matching Placebo

Once daily oral capsule for 48 weeks

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Interventions

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CTX-4430

Intervention Type DRUG

Placebo

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Maximum Eligible Age

30 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Celtaxsys, Inc.

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Steven Rowe, MD

Role: PRINCIPAL_INVESTIGATOR

University of Alabama at Birmingham, USA

Stuart Elborn, MD

Role: PRINCIPAL_INVESTIGATOR

Royal Brompton Hospital, London UK

Locations

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University of Alabama

Birmingham, Alabama, United States

Site Status

Providence Health and Services

Anchorage, Alaska, United States

Site Status

Banner University of Arizona Medical Center

Tucson, Arizona, United States

Site Status

Childrens Hospital Los Angeles

Los Angeles, California, United States

Site Status

Pediatric Pulmonary and Cystic Fibrosis Clinic, Stanford University

Palo Alto, California, United States

Site Status

University of California Davis Medical Center

Sacramento, California, United States

Site Status

National Jewish Health

Denver, Colorado, United States

Site Status

Yale University

New Haven, Connecticut, United States

Site Status

University of Florida

Gainesville, Florida, United States

Site Status

Central Florida Pulmonary Group

Orlando, Florida, United States

Site Status

Emory University

Atlanta, Georgia, United States

Site Status

University of Chicago

Chicago, Illinois, United States

Site Status

St. Francis Medical Center

Peoria, Illinois, United States

Site Status

University of Kansas Medical Center

Kansas City, Kansas, United States

Site Status

Maine Medical Center

Portland, Maine, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status

University of Michigan

Ann Arbor, Michigan, United States

Site Status

Harper University Hospital

Detroit, Michigan, United States

Site Status

Spectrum Health Butterworth Campus

Grand Rapids, Michigan, United States

Site Status

University of Mississippi Medical Center

Jackson, Mississippi, United States

Site Status

University of New Mexico

Albuquerque, New Mexico, United States

Site Status

Albany Medical College

Albany, New York, United States

Site Status

Columbia University Medical Center

New York, New York, United States

Site Status

SUNY Upstate Medical University

Syracuse, New York, United States

Site Status

Wake Forest Hospital

Winston-Salem, North Carolina, United States

Site Status

UC Cincinnati Children's Hospital

Cincinnati, Ohio, United States

Site Status

University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, United States

Site Status

Oregon Health & Sciences University

Portland, Oregon, United States

Site Status

Hershey Medical Center

Hershey, Pennsylvania, United States

Site Status

Drexel University

Philadelphia, Pennsylvania, United States

Site Status

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, United States

Site Status

Sanford Clinical Research

Sioux Falls, South Dakota, United States

Site Status

Universiy of Tennessee Medical Center UHS

Knoxville, Tennessee, United States

Site Status

Dell Children's Medical Center

Austin, Texas, United States

Site Status

Cook Children's Hospital

Fort Worth, Texas, United States

Site Status

Virginia Commonwealth University

Richmond, Virginia, United States

Site Status

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Site Status

Hôpital Erasme

Brussels, , Belgium

Site Status

UZ Leuven

Leuven, , Belgium

Site Status

University of Calgary

Calgary, Alberta, Canada

Site Status

Ottawa Hospital

Ottawa, Ontario, Canada

Site Status

St. Michael's Hospital

Toronto, Ontario, Canada

Site Status

Centre hospitalier de Dunkerque

Dunkirk, , France

Site Status

Hôpital Albert Michallon

Grenoble, , France

Site Status

Hopital Arnaud de Villeneuve

Montpellier, , France

Site Status

Hôpital Cochin

Paris, , France

Site Status

CH Lyon Sud

Pierre-Bénite, , France

Site Status

Charite' University

Berlin, , Germany

Site Status

Krankenhaus Donaustauf

Donaustauf, , Germany

Site Status

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

Dresden, , Germany

Site Status

Ruhrlandklinik Essen

Essen, , Germany

Site Status

Institut für klinische Forschung Pneumologie

Frankfurt, , Germany

Site Status

Klinikum der Johann Wolfgang Goethe-Universität

Frankfurt, , Germany

Site Status

Medizinische Hochschule Hannover

Hanover, , Germany

Site Status

Universitätsklinikum Jena CF Centre

Jena, , Germany

Site Status

Lungenärztliche Praxis München-Pasing

München-Pasing, , Germany

Site Status

Klinikum Stuttgart CF Ambulanz

Stuttgart, , Germany

Site Status

Ospedali Riuniti di Ancona

Ancona, , Italy

Site Status

Azienda Ospedaliero Universitaria

Catania, , Italy

Site Status

Azienda Ospedaliera A Meyer

Florence, , Italy

Site Status

IRCCS Ospedale Pediatrico Bambino

Rome, , Italy

Site Status

Ospedale Civile Maggiore

Verona, , Italy

Site Status

Belfast City Hospital

Belfast, , United Kingdom

Site Status

Bristol Royal Infirmary

Bristol, , United Kingdom

Site Status

Llandough Hospital

Cardiff, , United Kingdom

Site Status

St James's University Hospital

Leeds, , United Kingdom

Site Status

Liverpool Heart and Chest Hospital

Liverpool, , United Kingdom

Site Status

King's College Hospital

London, , United Kingdom

Site Status

Royal Brompton Hospital

London, , United Kingdom

Site Status

University Hospital of South Manchester

Manchester, , United Kingdom

Site Status

Royal Victoria Infirmary

Newcastle upon Tyne, , United Kingdom

Site Status

University Hospital Southampton

Southampton, , United Kingdom

Site Status

Royal Stoke University Hospital

Stoke-on-Trent, , United Kingdom

Site Status

Countries

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United States Belgium Canada France Germany Italy United Kingdom

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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CTX-4430-CF-201

Identifier Type: -

Identifier Source: org_study_id

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