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Trial record 2 of 2 for:    CTX-4430 | Cystic Fibrosis

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02443688
Recruitment Status : Completed
First Posted : May 14, 2015
Results First Posted : July 25, 2019
Last Update Posted : September 4, 2019
Sponsor:
Information provided by (Responsible Party):
Celtaxsys, Inc.

Tracking Information
First Submitted Date  ICMJE May 8, 2015
First Posted Date  ICMJE May 14, 2015
Results First Submitted Date  ICMJE April 29, 2019
Results First Posted Date  ICMJE July 25, 2019
Last Update Posted Date September 4, 2019
Actual Study Start Date  ICMJE October 30, 2015
Actual Primary Completion Date April 20, 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 28, 2019)
Difference From Placebo in Absolute Change From Baseline in Forced Expiratory Volume in 1 Second Percent Predicted (ppFEV1) [ Time Frame: Baseline, Week 48 ]
Difference from Placebo in absolute change from Baseline at Week 48 was assessed for FEV1 percent predicted.
Original Primary Outcome Measures  ICMJE
 (submitted: May 11, 2015)
  • To evaluate the safety and tolerability of CTX-4430 administered orally once-daily to cystic fibrosis (CF) patients by the incidence of treatment emergent adverse events as compared to placebo. [ Time Frame: 52 weeks ]
  • To evaluate the efficacy of CTX-4430 administered orally once-daily to CF patients as determined by the absolute change from Baseline in FEV1 (forced expiratory volume in 1 second) percent predicted [ Time Frame: 52 Weeks ]
Change History Complete list of historical versions of study NCT02443688 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: June 28, 2019)
  • Number of Pulmonary Exacerbations Through 48 Weeks [ Time Frame: 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.
  • Hazard Ratio Pulmonary Exacerbation While in the Study [ Time Frame: 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.
  • Subjects Without a Pulmonary Exacerbation While in the Study [ Time Frame: Week 48 ]
    Subjects who did not experience a protocol-defined pulmonary exacerbation during the study
  • Relative Change (Percent Change) From Baseline in ppFEV1 [ Time Frame: Baseline, Week 48 ]
    Percent change from Baseline for ppFEV1 at 48 weeks was assessed.
  • 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 [ Time Frame: Baseline, Week 48 ]
  • Change From Baseline for Specified Biomarkers [ Time Frame: Baseline, Week 48 ]
    Results were only calculated in subjects who had a verifiable result at the Baseline and Week 48 visits.
  • Change From Baseline for C-reactive Protein (Hs-CRP) [ Time Frame: Baseline, Week 48 ]
    Results were only calculated in subjects who had a verifiable result at the Baseline and Week 48 visits.
Original Secondary Outcome Measures  ICMJE
 (submitted: May 11, 2015)
  • To evaluate the efficacy of CTX-4430 administered orally once-daily to CF patients as determined by the relative change from Baseline in FEV1 percent predicted [ Time Frame: 52 weeks ]
  • To evaluate the effect of CTX-4430 administered orally once-daily to CF patients on FVC (forced vital capacity) percent predicted and FEF25-75% (forced expiratory flow during the middle half of the forced vital capacity) percent predicted [ Time Frame: 52 weeks ]
  • To evaluate the effect of CTX-4430 administered orally once-daily to CF patients on time to first pulmonary exacerbation while in the study [ Time Frame: 52 weeks ]
  • To evaluate the effect of CTX-4430 administered orally once-daily to CF patients on the number of pulmonary exacerbations [ Time Frame: 52 weeks ]
  • To evaluate the effect of CTX-4430 administered orally once-daily to CF patients on specified biomarkers [ Time Frame: 52 weeks ]
Current Other Pre-specified Outcome Measures
 (submitted: June 28, 2019)
  • Number of Pulmonary Exacerbation Per Year for Participants With ppFEV1 >75 at Baseline [ Time Frame: 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.
  • Hazard Ratio Pulmonary Exacerbation for Participants With ppFEV1 >75 at Baseline [ Time Frame: Week 48 ]
    Hazard ratio of pulmonary exacerbation versus placebo for all subjects
  • Subjects Without a Pulmonary Exacerbation by Participants With ppFEV1 >75 at Baseline [ Time Frame: Week 48 ]
    Subjects who did not experience a protocol-defined pulmonary exacerbation during the study.
  • Number of Pulmonary Exacerbation by Subjects if Taking CFTR-Modulator Therapy at Baseline [ Time Frame: 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.
  • Hazard Ratio Pulmonary Exacerbation by Subjects if Taking CFTR-Modulator Therapy at Baseline [ Time Frame: Week 48 ]
    Hazard Ratio pulmonary exacerbation versus placebo for all subjects taking CFTR-modulating therapy at Baseline
  • Subjects Without a Pulmonary Exacerbation by Subjects if Taking CFTR-Modulator Therapy at Baseline [ Time Frame: Week 48 ]
    Subjects who did not experience a protocol-defined pulmonary exacerbation during the study.
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE EMPIRE CF: A Phase 2 Study to Evaluate the Efficacy, Safety, and Tolerability of CTX-4430 in Adult Cystic Fibrosis (CF) Patients
Official Title  ICMJE 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
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).
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Cystic Fibrosis
Intervention  ICMJE
  • Drug: CTX-4430
  • Drug: Placebo
Study Arms  ICMJE
  • Experimental: 50 mg CTX-4430
    Once daily oral capsule for 48 weeks
    Intervention: Drug: CTX-4430
  • Experimental: 100 mg CTX-4430
    Once daily oral capsule for 48 weeks
    Intervention: Drug: CTX-4430
  • Placebo Comparator: Matching Placebo
    Once daily oral capsule for 48 weeks
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: May 19, 2017)
200
Original Estimated Enrollment  ICMJE
 (submitted: May 11, 2015)
195
Actual Study Completion Date  ICMJE May 16, 2018
Actual Primary Completion Date April 20, 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 30 Years   (Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Belgium,   Canada,   France,   Germany,   Italy,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02443688
Other Study ID Numbers  ICMJE CTX-4430-CF-201
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Celtaxsys, Inc.
Study Sponsor  ICMJE Celtaxsys, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Steven Rowe, MD University of Alabama at Birmingham, USA
Principal Investigator: Stuart Elborn, MD Royal Brompton Hospital, London UK
PRS Account Celtaxsys, Inc.
Verification Date August 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP