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    Summary
    EudraCT Number:2018-000966-12
    Sponsor's Protocol Code Number:EL-004
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2019-06-25
    Trial results View 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 ConcernedGermany - BfArM
    A.2EudraCT number2018-000966-12
    A.3Full title of the trial
    A Phase 2 Open Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Dose Levels of Subcutaneously Administered ELX-02 in Patients with Cystic Fibrosis with at Least One G542X Allele
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Dose Levels of Subcutaneously Administered ELX-02 in Patients with Cystic Fibrosis
    A.4.1Sponsor's protocol code numberEL-004
    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 SponsorEloxx Pharmaceuticals 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 supportEloxx Pharmaceuticals Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationEloxx Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointTim Kachmar
    B.5.3 Address:
    B.5.3.1Street Address480 Arsenal Way, Suite 130
    B.5.3.2Town/ cityWatertown, MA
    B.5.3.3Post code02472
    B.5.3.4CountryUnited States
    B.5.4Telephone number+16176459926
    B.5.6E-mailtim.kachmar@eloxxpharma.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/3/18/2072
    D.3 Description of the IMP
    D.3.2Product code ELX-02
    D.3.4Pharmaceutical form Lyophilisate for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNot Assigned
    D.3.9.1CAS number 1375073-94-1
    D.3.9.2Current sponsor codeELX-02
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number160
    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 Yes
    D.2.1.1.1Trade name Kalydeco
    D.2.1.1.2Name of the Marketing Authorisation holderVertex Pharmaceuticals (Ireland) Limited
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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/3/08/556
    D.3 Description of the IMP
    D.3.1Product nameKalydeco
    D.3.4Pharmaceutical form Film-coated tablet
    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 INNIvacaftor
    D.3.9.1CAS number 873054-44-5
    D.3.9.4EV Substance CodeSUB33103
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Cystic fibrosis (CF)
    E.1.1.1Medical condition in easily understood language
    Inherited conditions caused by nonsense mutations
    E.1.1.2Therapeutic area Body processes [G] - Genetic Phenomena [G05]
    MedDRA Classification
    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
    1. To assess the safety and tolerability of different dose levels and exposures of ELX-02 administered subcutaneously (SC), as monotherapy and in combination with ivacaftor in patients with CF carrying at least one G542X CFTR or phenotypically similar nonsense allele.
    2. To study the PK of multiple SC doses of ELX-02 as monotherapy and in combination with ivacaftor, in patients with CF.
    E.2.2Secondary objectives of the trial
    1. To study the PD of multiple doses of ELX-02 as monotherapy and in combination with ivacaftor on sweat chloride concentration (SCC).
    2. To study the PD of multiple doses of ELX-02 as monotherapy and in combination with ivacaftor on pulmonary function (forced expiratory volume [FEV], forced vital capacity [FVC], and forced expiratory flow at 25-75%] FEF).
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Evidence of signed and dated informed consent/assent document(s) indicating that the subject (and/or his parent/legal guardian) has been informed of all pertinent aspects of the trial
    2. Understands, and is willing and able to comply with all scheduled visits, treatment plan, laboratory tests, and other study procedures
    3. Males and females age 18 years and above; in countries where permitted, males and females age 16 and above
    4. A confirmed diagnosis of nmCF with a documented G542X or phenotypically similar nonsense mutation, homozygote, or compound heterozygote with one of the specified mutations. For heterozygotes, one mutation has to be a G542X or phenotypically similar nonsense mutation, and the second mutation should be any Class 1 or Class 2 mutation. Patients with one G542X or phenotypically similar nonsense allele and a second allele that is not in the above list may be potentially allowed but only after discussion on a case by case basis with and written approval from the Sponsor.
    5. Documented SCC ≥ 60 mEq
    6. FEV1 ≥ 40% predicted normal for age, gender and height at Screening (Knudson Equation)
    7. Stable regimen of oral CF medications, inhaled antibiotics, dornase alfa, and hypertonic saline, and physiotherapy for the period 28 days prior to Screening
    8. Non-smokers for at least 180 days (6 months) prior to Screening
    9. Must be willing to abstain from strenuous exercise during the 24 hours prior study visits
    10. Females of childbearing potential and males capable of fathering a child must meet the contraception requirements outlined in Section ‎6.4
    11. Non-lactating females
    12. Females on hormone replacement therapy (estrogen or progesterone) or contraceptive therapy must be stabilized on a product and dose for at least 30 days prior to Screening
    13. Glomerular filtration rate (GFR) of > 60 mL/min/1.73m2 at Screening (GFR to be calculated using the Chronic Kidney Disease Epidemiology Collaboration CKD-EPI formula
    14. Have not received systemic medications with potential to impair renal function on a frequent basis (e.g., non steroidal anti inflammatory drugs [NSAIDs]) or with ototoxic potential (e.g., quinine or salicylates), or any injectable aminoglycosides for a period of at least 14 days prior to dosing
    15. Negative human immunodeficiency virus (HIV), hepatitis B surface antigen (HBsAg), and hepatitis C virus antigen (HCV Ag) serology tests at Screening
    16. No history of alcohol or drug abuse within the 6 months prior to Screening
    17. Body Mass Index (BMI) of 19.0 to 30.0 kg/m2 (inclusive). Patients with a lower BMI may be entered into the study at the discretion of the investigator following consultation with the Sponsor.
    E.4Principal exclusion criteria
    1. Patients who are investigational site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the Investigator, or patients who are Eloxx Pharmaceuticals employees directly involved in the conduct of the study
    2. Participation in clinical study including administration of any investigational drug or device in the last 30 days or 5 half-lives (whichever is longer) prior to investigational product dosing in the current study
    3. Use of prohibited medications as defined in Section ‎6.1 within the specified windows
    4. History of any comorbidity which in the opinion of the investigator might confound the study or pose an additional risk in administering the study drug to the patient
    5. History of any organ transplantation
    6. Liver disease characterized by cirrhosis or portal hypertension. Patients with alanine aminotransferase (ALT), aspartate aminotransferase (AST), and/or a total bilirubin 3.0 times the upper limit of normal (ULN) will be excluded
    7. History of clinically significant adrenal disease needing hormone replacement therapy
    8. History of congestive heart failure diagnosed clinically or with documented left ventricular ejection fraction (LVEF) ≤ 40%
    9. Evidence or history of clinically relevant psychiatric condition
    10. A positive urine drug screen (amphetamines, benzodiazepines, cocaine and opiates) at Screening
    11. Screening supine 12-lead electrocardiogram (ECG) demonstrating any clinically significant findings as judged by the Investigator
    12. Patients with any abnormalities in clinical laboratory tests at Screening, considered by the study physician as clinically relevant
    13. Major surgery within 180 days (6 months) of Screening
    14. Patients without documented prior aminoglycoside exposure who have a mitochondrial mutation that has been shown to increase sensitivity to aminoglycosides
    15. Known allergy to any aminoglycoside
    16. Patients with any acute medical situation not related to CF (e.g., gastroenteritis) unresolved within 14 days of first dose that is considered of significance by the Investigator
    17. Patients with any abnormality at ENT screening, that indicates the presence of a vestibular toxicity associated with prior exposure to aminoglycosides.
    18. Dizziness Handicap Inventory (DHI)-H score at screening >16.
    19. Patients receiving CFTR modulators or potentiators as defined in Section ‎6.1 within 2 months of study enrollment
    20. Patients unable to avoid foods or drinks that contain grapefruit or Seville oranges, as these may affect the amount of ivacaftor in the body. Patients who meet this exclusion criterion and have already started the study prior to Protocol Amendment 5.0 will not be allowed to proceed to Treatment Period 5
    21. Patients with rare hereditary problems of galactose intolerance, total lactase deficiency or glucose-galactose malabsorption. Patients who meet this exclusion criterion and have already started the study prior to Protocol Amendment 5.0 will not be allowed to proceed to Treatment Period 5

    E.5 End points
    E.5.1Primary end point(s)
    Primary Endpoints:
    1. The incidence and characteristics of adverse events (AEs). Other safety assessments will include injection site reactions (DAIDS Grading Scale) and changes from baseline in vital signs, physical examination, electrocardiograms, blood and urine safety laboratory tests, audiometric testing (including high frequency audiometry) and Tinnitus Handicap Inventory (THI) and Dizziness Handicap Inventory (DHI) questionnaires.
    2. Plasma and urine PK parameters of ELX-02 following the first dose and at steady state, at each dose level (Treatment Periods 1-5).
    3. Plasma PK parameters of ivacaftor following the first dose and at steady state (Treatment Period 5)
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. From screening until End-of-Study (EOS) visit;

    2.
    Blood PK parameters: D1, D2, D7 of treatment periods 1-3, Day 14 of treatment period 4, End-of-Study (EOS) visit.
    Urine PK parameters: D1, D2 of all treatment periods
    E.5.2Secondary end point(s)
    Secondary Endpoints:
    1. PD as assessed by changes from baseline in SCC following ELX-02 treatment
    2. PD as assessed by changes from baseline in pulmonary function including FEV1, FVC, and FEF25-75, absolute values and percent predicted.
    3. Dose and PK relationship.
    E.5.2.1Timepoint(s) of evaluation of this end point
    During treatment
    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 No
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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 No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    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 No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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 concerned3
    E.8.5The trial involves multiple Member States No
    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
    Australia
    Israel
    Germany
    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 years0
    E.8.9.1In the Member State concerned months25
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months25
    E.8.9.2In all countries concerned by the trial days0
    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: 16
    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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 6
    F.4.2.2In the whole clinical trial 16
    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)
    see protocol
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2019-10-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-11-18
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2022-04-05
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