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Completed

NCT Number: NCT02443688

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

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.

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Key information

Conditions

Cystic Fibrosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Lung Diseases Pancreatic Diseases Respiratory Tract Diseases

Age range

18 year–30 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Hôpital Erasme, Brussels, Belgium

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About this study

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).

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

CTX-4430

Drug

Placebo

Drug

Primary outcomes

  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 outcomes

  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 outcomes

  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.

Sponsors and collaborators

Lead sponsor

Celtaxsys, Inc.

Other

Registry information

Official study 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

Important dates

Study start
2015
Primary completion
2018
Study completion
2018
First posted
May 14, 2015
Registry last updated
Sep 4, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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