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

Cystic Fibrosis Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02443688
• Other study ID #: CTX-4430-CF-201
• Status: Completed
• Phase: Phase 2
• Start date: October 30, 2015
• Est. completion date: May 16, 2018

Study information

• Verified date: August 2019
• Source: Celtaxsys, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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 information / eligibility

• Status: Completed
• Enrollment: 200
• Est. completion date: May 16, 2018
• Est. primary completion date: April 20, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 30 Years

Eligibility

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

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• CTX-4430

• Placebo

Locations

Country	Name	City	State
Belgium	Hôpital Erasme	Brussels
Belgium	UZ Leuven	Leuven
Canada	University of Calgary	Calgary	Alberta
Canada	Ottawa Hospital	Ottawa	Ontario
Canada	St. Michael's Hospital	Toronto	Ontario
France	Centre hospitalier de Dunkerque	Dunkerque
France	Hôpital Albert Michallon	Grenoble
France	Hopital Arnaud de Villeneuve	Montpellier
France	Hôpital Cochin	Paris
France	CH Lyon Sud	Pierre Benite
Germany	Charite' University	Berlin
Germany	Krankenhaus Donaustauf	Donaustauf
Germany	Carl-Gustav-Klinikum Dresden, Mukoviszidose Centrum "Christiane Herzog"	Dresden
Germany	Ruhrlandklinik Essen	Essen
Germany	Institut für klinische Forschung Pneumologie	Frankfurt
Germany	Klinikum der Johann Wolfgang Goethe-Universität	Frankfurt
Germany	Medizinische Hochschule Hannover	Hannover
Germany	Universitätsklinikum Jena CF Centre	Jena
Germany	Lungenärztliche Praxis München-Pasing	München-Pasing
Germany	Klinikum Stuttgart CF Ambulanz	Stuttgart
Italy	Ospedali Riuniti di Ancona	Ancona
Italy	Azienda Ospedaliero Universitaria	Catania
Italy	Azienda Ospedaliera A Meyer	Florence
Italy	IRCCS Ospedale Pediatrico Bambino	Rome
Italy	Ospedale Civile Maggiore	Verona
United Kingdom	Belfast City Hospital	Belfast
United Kingdom	Bristol Royal Infirmary	Bristol
United Kingdom	Llandough Hospital	Cardiff
United Kingdom	St James's University Hospital	Leeds
United Kingdom	Liverpool Heart and Chest Hospital	Liverpool
United Kingdom	King's College Hospital	London
United Kingdom	Royal Brompton Hospital	London
United Kingdom	University Hospital of South Manchester	Manchester
United Kingdom	Royal Victoria Infirmary	Newcastle Upon Tyne
United Kingdom	University Hospital Southampton	Southampton
United Kingdom	Royal Stoke University Hospital	Stoke on Trent
United States	Albany Medical College	Albany	New York
United States	University of New Mexico	Albuquerque	New Mexico
United States	Providence Health and Services	Anchorage	Alaska
United States	University of Michigan	Ann Arbor	Michigan
United States	Emory University	Atlanta	Georgia
United States	Dell Children's Medical Center	Austin	Texas
United States	University of Alabama	Birmingham	Alabama
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Massachusetts General Hospital	Boston	Massachusetts
United States	University of Chicago	Chicago	Illinois
United States	UC Cincinnati Children's Hospital	Cincinnati	Ohio
United States	National Jewish Health	Denver	Colorado
United States	Harper University Hospital	Detroit	Michigan
United States	Cook Children's Hospital	Fort Worth	Texas
United States	University of Florida	Gainesville	Florida
United States	Spectrum Health Butterworth Campus	Grand Rapids	Michigan
United States	Hershey Medical Center	Hershey	Pennsylvania
United States	University of Mississippi Medical Center	Jackson	Mississippi
United States	University of Kansas Medical Center	Kansas City	Kansas
United States	Universiy of Tennessee Medical Center UHS	Knoxville	Tennessee
United States	Childrens Hospital Los Angeles	Los Angeles	California
United States	Medical College of Wisconsin	Milwaukee	Wisconsin
United States	Yale University	New Haven	Connecticut
United States	Columbia University Medical Center	New York	New York
United States	University of Oklahoma Health Sciences Center	Oklahoma City	Oklahoma
United States	Central Florida Pulmonary Group	Orlando	Florida
United States	Pediatric Pulmonary and Cystic Fibrosis Clinic, Stanford University	Palo Alto	California
United States	St. Francis Medical Center	Peoria	Illinois
United States	Drexel University	Philadelphia	Pennsylvania
United States	The Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Children's Hospital of Pittsburgh of UPMC	Pittsburgh	Pennsylvania
United States	Maine Medical Center	Portland	Maine
United States	Oregon Health & Sciences University	Portland	Oregon
United States	Virginia Commonwealth University	Richmond	Virginia
United States	University of California Davis Medical Center	Sacramento	California
United States	Sanford Clinical Research	Sioux Falls	South Dakota
United States	SUNY Upstate Medical University	Syracuse	New York
United States	Banner University of Arizona Medical Center	Tucson	Arizona
United States	Wake Forest Hospital	Winston-Salem	North Carolina

Sponsors (1)

Lead Sponsor	Collaborator
Celtaxsys, Inc.

Countries where clinical trial is conducted

United States,  Belgium,  Canada,  France,  Germany,  Italy,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Number of Pulmonary Exacerbation Per Year for Participants With ppFEV1 >75 at Baseline	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.	Week 48
Other	Hazard Ratio Pulmonary Exacerbation for Participants With ppFEV1 >75 at Baseline	Hazard ratio of pulmonary exacerbation versus placebo for all subjects	Week 48
Other	Subjects Without a Pulmonary Exacerbation by Participants With ppFEV1 >75 at Baseline	Subjects who did not experience a protocol-defined pulmonary exacerbation during the study.	Week 48
Other	Number of Pulmonary Exacerbation by Subjects if Taking CFTR-Modulator Therapy at Baseline	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.	Week 48
Other	Hazard Ratio Pulmonary Exacerbation by Subjects if Taking CFTR-Modulator Therapy at Baseline	Hazard Ratio pulmonary exacerbation versus placebo for all subjects taking CFTR-modulating therapy at Baseline	Week 48
Other	Subjects Without a Pulmonary Exacerbation by Subjects if Taking CFTR-Modulator Therapy at Baseline	Subjects who did not experience a protocol-defined pulmonary exacerbation during the study.	Week 48
Primary	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	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.	Week 48
Secondary	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.	Week 48
Secondary	Subjects Without a Pulmonary Exacerbation While in the Study	Subjects who did not experience a protocol-defined pulmonary exacerbation during the study	Week 48
Secondary	Relative Change (Percent Change) From Baseline in ppFEV1	Percent change from Baseline for ppFEV1 at 48 weeks was assessed.	Baseline, Week 48
Secondary	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
Secondary	Change From Baseline for Specified Biomarkers	Results were only calculated in subjects who had a verifiable result at the Baseline and Week 48 visits.	Baseline, Week 48
Secondary	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.	Baseline, Week 48