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

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

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.
Ages Eligible for Study: | 18 Years to 30 Years (Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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

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

Principal Investigator: | Steven Rowe, MD | University of Alabama at Birmingham, USA | |
Principal Investigator: | Stuart Elborn, MD | Royal Brompton Hospital, London UK |
Documents provided by Celtaxsys, Inc.:
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 |
Cystic Fibrosis Fibrosis Pathologic Processes Pancreatic Diseases Digestive System Diseases |
Lung Diseases Respiratory Tract Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases |