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

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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.

Study Description
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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.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: CTX-4430 Drug: Placebo Phase 2

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 Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 200 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
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
Actual Study Start Date : October 30, 2015
Actual Primary Completion Date : April 20, 2018
Actual Study Completion Date : May 16, 2018

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arms and Interventions
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Arm Intervention/treatment
Experimental: 50 mg CTX-4430
Once daily oral capsule for 48 weeks
 Drug: CTX-4430
Experimental: 100 mg CTX-4430
Once daily oral capsule for 48 weeks
 Drug: CTX-4430
Placebo Comparator: Matching Placebo
Once daily oral capsule for 48 weeks
 Drug: Placebo


Outcome Measures
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Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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

  4. Relative Change (Percent Change) From Baseline in ppFEV1 [ Time Frame: Baseline, Week 48 ]
    Percent change from Baseline for ppFEV1 at 48 weeks was assessed.

  5. 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 ]
  6. 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.

  7. 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.


Other Outcome Measures:
  1. 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.

  2. 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

  3. 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.

  4. 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.

  5. 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

  6. 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.


Eligibility Criteria
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Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years to 30 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02443688


Locations
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Sponsors and Collaborators
Celtaxsys, Inc.
Investigators
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Principal Investigator: Steven Rowe, MD University of Alabama at Birmingham, USA
Principal Investigator: Stuart Elborn, MD Royal Brompton Hospital, London UK
  Study Documents (Full-Text)

Documents provided by Celtaxsys, Inc.:
Study Protocol  [PDF] May 4, 2016
Statistical Analysis Plan  [PDF] June 15, 2018

More Information
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Responsible Party: Celtaxsys, Inc.
ClinicalTrials.gov Identifier: NCT02443688    
Other Study ID Numbers: CTX-4430-CF-201
First Posted: May 14, 2015    Key Record Dates
Results First Posted: July 25, 2019
Last Update Posted: September 4, 2019
Last Verified: August 2019
Additional relevant MeSH terms:
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Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
 Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases