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    Summary
    EudraCT Number:2015-002677-38
    Sponsor's Protocol Code Number:CTX-4430-CF-201
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2016-03-03
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2015-002677-38
    A.3Full title of the trial
    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
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An investigation of the effect of CTX-4430 on lung inflammation in patients with cystic fibrosis
    A.3.2Name or abbreviated title of the trial where available
    EMPIRE CF
    A.4.1Sponsor's protocol code numberCTX-4430-CF-201
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02443688
    A.5.4Other Identifiers
    Name:IND numberNumber:116095
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorCeltaxsys Inc.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportCeltaxsys Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationChiltern International Limited
    B.5.2Functional name of contact pointProject Management
    B.5.3 Address:
    B.5.3.1Street Address171 Bath Road
    B.5.3.2Town/ citySlough, Berkshire
    B.5.3.3Post codeSL1 4AA
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+390399906704
    B.5.6E-mailClaudia.Ulbricht@chiltern.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/14/1363
    D.3 Description of the IMP
    D.3.1Product nameCTX-4430
    D.3.2Product code CTX-4430
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNacebilustat
    D.3.9.1CAS number 943764-99-6
    D.3.9.2Current sponsor codeCTX-4430
    D.3.9.3Other descriptive nameCRC3357, ZK-355322, Leukoton
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 2
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/14/1363
    D.3 Description of the IMP
    D.3.1Product nameCTX-4430
    D.3.2Product code CTX-4430
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNacebilustat
    D.3.9.1CAS number 943764-99-6
    D.3.9.2Current sponsor codeCTX-4430
    D.3.9.3Other descriptive nameCRC3357, ZK-355322, Leukoton
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboCapsule, hard
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Cystic Fibrosis
    E.1.1.1Medical condition in easily understood language
    Cystic Fibrosis
    E.1.1.2Therapeutic area Diseases [C] - Respiratory Tract Diseases [C08]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10011763
    E.1.2Term Cystic fibrosis lung
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10011762
    E.1.2Term Cystic fibrosis
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    - To evaluate the safety and tolerability of CTX-4430 administered orally once-daily to cystic fibrosis (CF) patients for 48 weeks
    - To evaluate the efficacy of CTX-4430 administered orally once-daily to CF patients for 48 weeks as determined by the absolute change from Baseline in FEV1 (forced expiratory volume in 1 second) percent predicted
    E.2.2Secondary objectives of the trial
    1. Efficacy evaluation of CTX-4430 as determined by the relative change from Baseline in FEV1 percent predicted
    2. Evaluation of the effect of orally administered CTX-4430 on FVC % predicted and FEF25-75% predicted
    3. Evaluation of the effect of orally administered CTX-4430 on time to first pulmonary exacerbation while in the study
    3.1 Evaluation of the effect of orally administered CTX-4430 on the number of pulmonary exacerbations
    3.2 Evaluation of the effect of orally administered CTX-4430 on specified biomarkers (sputum DNA and elastase and serum hs-CRP)
    Exploratory Objectives:
    - Effect evaluation of orally administered CTX-4430 on sputum bacterial density
    - Population PK of orally administered CTX-4430 after first dose and at steady state
    - Change from Baseline evaluation in subject’s health-related QOL while on CTX-4430 administered orally 1x daily for 48 w
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patients are eligible to participate in the study if they meet all of the following inclusion criteria:
    1. 18 to 30 years of age inclusive at the time of Screening
    2. Documented, confirmed diagnosis of pulmonary CF (defined as follows):
    • CF signs and symptoms AND
    • Either 2 CFTR mutations on genetic testing OR sweat chloride ≥60 mEq/L
    3. Medically stable, in the Investigator’s opinion
    4. At least 1 pulmonary exacerbation, based on the Investigator’s judgment, in the 12 months before Screening
    5. Resolution of any pulmonary exacerbation of CF at least 14 days before Screening, in the Investigator’s opinion
    6. On a stable regimen of CF treatments with no change for at least 14 days before Screening and between Screening and Baseline
    7. If on ivacaftor or ivacaftor-lumacaftor combination, on a stable regimen for at least 8 weeks before Baseline
    8. No clinical or radiological evidence, per the Investigator’s site procedures, of clinically significant lung abnormalities (e.g., major atelectasis, pneumothorax)
    9. FEV1 ≥50 percent predicted at Screening
    10. Resting oxygen saturation >92% on room air
    11. Body mass index (BMI) ≥17.0 kg/m2
    12. No smoking (including electronic cigarettes) for at least 6 months before Screening and agreement not to use such products for the duration of the study
    13. Females of childbearing potential must have a negative pregnancy test at Screening (unless surgically sterile) and must agree to use an effective contraception method from screening throughout the duration of the study
    14. Able to perform spirometry according to European Respiratory Society/American Thoracic Society guidance
    15. Able to swallow investigational product (IP) whole
    16. Able to comply with the study procedures, in the opinion of the Investigator
    17. Has provided informed consent to participate in the study.
    E.4Principal exclusion criteria
    Patients are eligible to participate in this study if they meet none of the following exclusion criteria:
    1. In the opinion of the Investigator, any significant clinical/laboratory/radiological/spirometric sign of unstable or unexpectedly deteriorating respiratory disease within 14 days before Screening or between Screening and Baseline (These clinical/laboratory/radiological/ spirometric signs include, but are not limited to, features suggestive of a pulmonary exacerbation as suggested by the modified Fuchs’ criteria.)
    2. A medical condition that is unstable, could be adversely impacted by participation in the study, or could impact assessment of the study results, in the opinion of the Investigator
    3. History of organ transplantation
    4. History of either alcoholism or drug abuse in the opinion of the investigator
    5. Clinically significant hemoptysis (e.g., > approximately 30 cc per episode, or clinically significant in the Investigator’s opinion) within 180 days before Screening
    6. Colonization with organisms associated with a more rapid decline in respiratory function in CF patients (e.g. all Burkholderia species, Mycobacterium abscessus). Subjects with a history of a positive culture could be considered free of colonization if she/he has had 6 subsequent respiratory tract cultures negative for these bacteria within the past 24 months prior to Screening, with one of these cultures obtained within 6 months prior to Screening
    7. Active allergic bronchopulmonary aspergillosis at Screening or at Baseline
    8. Any clinically significant ECG abnormality, in the Investigator’s opinion
    9. Positive serology for HIV-1 or HIV-2 antibody, hepatitis C virus antibody, or hepatitis B surface antigen at Screening
    10. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) ≥2x the upper limit of normal (ULN) at Screening
    11. Bilirubin >1.25 x ULN at Screening. Subjects with known Gilbert’s syndrome can be included with bilirubin >1.25 x ULN
    12. Any subject with cirrhosis of the liver
    13. Any subject with portal hypertension
    14. Neutrophil count <1.5 x 109/L at Screening
    15. Platelet count <150,000/μl at Screening
    16. Clinically unstable pancreatic function, in the opinion of the Investigator. Evidence of unstable pancreatic function could include:
    • Clinically significant weight loss (≥5% after a previously stable period)
    • Evidence of uncontrolled hyperglycemia or recent hypoglycemia
    • Change in pancreatic enzyme requirements in the 60 days before Screening
    17. Use of systemic corticosteroids, or systemic antimicrobial therapy (other than chronic antimicrobial use, e.g. azithromycin, flucloxacillin, itraconazole) within 14 days before Screening or between Screening and Baseline
    18. Regular use (>3 times per week) of a high-dose NSAID (e.g., >1.6 g ibuprofen/day) within 60 days before Screening or between Screening and Baseline
    19. Participation in a clinical trial for any medical/device product within 30 days before Screening (participation in a noninterventional or observational study is permitted)
    20. Pregnant or nursing women
    E.5 End points
    E.5.1Primary end point(s)
    Efficacy: Analysis of change from Baseline to Week 48 in FEV1 percent predicted
    Safety: AE recording
    E.5.1.1Timepoint(s) of evaluation of this end point
    Safety, AE recording: screening, baseline and at weeks 4, 6, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52.
    Efficacy, spirometry: screening, baseline and at weeks 4, 8, 12, 16, 24, 32, 40, 48, 52.
    E.5.2Secondary end point(s)
    CLINICAL ENDPOINTS
    - spirometry-based secondary endpoints will be analyzed using the same methods as the primary endpoint. For time points prior to 48 weeks, only descriptive statistics will be presented.
    - Pulmonary exacerbations: time to first pulmonary exacerbation and number of pulmonary exacerbations.
    BIOMARKER ENDPOINTS
    - sputum DNA and elastase, serum hs-CRP
    EXPLORATORY ENDPOINTS
    - pharmacokinetics
    - sputum bacterial density (P aeruginosa, Burkholderia cepacia complex, Achromobacter xylosoxidans, Stenotrophomonas maltophilia, and Staphylococcus aureus (including methicillin–resistant S aureus and small colony variants of S aureus [CFUs])
    - change from Baseline in subject’s health-related quality of life (HRQOL): Cystic Fibrosis Questionnaire
    E.5.2.1Timepoint(s) of evaluation of this end point
    PHARMACOKINETICS
    - PK sampling: predose and at 1, 2, 4, and 6 hours postdose, at baseline and steady state (week 4 and/or week 8)
    CLINICAL ENDPOINTS
    - spirometry-based secondary endpoints: screening, baseline and at weeks 4 , 8, 12, 16, 24, 32, 40, 48, 52.
    - Pulmonary exacerbations checklist: baseline and at weeks 4 , 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52
    BIOMARKER ENDPOINTS
    - Sputum (DNA + elastase): baseline and at weeks 8, 24, 48
    - serum hs-CRP: baseline and at weeks 4, 8, 12, 24, 32, 40, 48 and 52
    EXPLORATORY ENDPOINTS
    - sputum bacterial density: baseline and at weeks 8, 24, 48
    - health-related quality of life, Cystic Fibrosis Questionnaire: baseline and at weeks 12, 24, 48
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Tolerability
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial3
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA36
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Belgium
    Canada
    France
    Germany
    Italy
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months28
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial months32
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 195
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 95
    F.4.2.2In the whole clinical trial 195
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    none
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation CF Clinical Trial Network
    G.4.3.4Network Country Germany
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2016-04-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-05-11
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-05-16
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