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    Summary
    EudraCT Number:2015-004263-36
    Sponsor's Protocol Code Number:RPL554-010-2015
    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:2017-09-06
    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 number2015-004263-36
    A.3Full title of the trial
    A Phase IIa, randomised, double blind, placebo controlled, three way crossover study to assess the pharmacokinetics of RPL554 administered to adult patients with Cystic Fibrosis.
    Eine randomisierte, doppelblinde, placebokontrollierte Phase IIa-Studie im dreifach Cross-Over Design zur Untersuchung der Pharmakokinetik von RPL554 bei erwachsenen Patienten mit zystischer Fibrose.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to assess the effects of inhaled RPL554 in adults with cystic fibrosis.
    Studie zur Untersuchung der Wirkung von inhalativem RPL554 bei erwachsenen Patienten mit zystischer Fibrose.
    A.3.2Name or abbreviated title of the trial where available
    Study to assess the effects of inhaled RPL554 in patients with cystic fibrosis.
    Studie zur Untersuchung der Wirkung von inhalativem RPL554 bei Patienten mit zystischer Fibrose.
    A.4.1Sponsor's protocol code numberRPL554-010-2015
    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 SponsorVerona Pharma plc
    B.1.3.4CountryUnited Kingdom
    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 supportVerona Pharma Plc
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationVerona Pharma plc
    B.5.2Functional name of contact pointShiva Memari
    B.5.3 Address:
    B.5.3.1Street Address3 More London Riverside
    B.5.3.2Town/ cityLondon
    B.5.3.3Post codeSE1 2RE
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+442032834200
    B.5.6E-mailShiva.Memari@veronapharma.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameRPL554
    D.3.2Product code RPL554
    D.3.4Pharmaceutical form Nebuliser suspension
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPInhalation use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNRPL554
    D.3.9.1CAS number 298680-25-8
    D.3.9.2Current sponsor codeRPL554
    D.3.9.3Other descriptive nameRPL554
    D.3.9.4EV Substance CodeAS1
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number0.6 to 2.4
    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 placeboNebuliser solution
    D.8.4Route of administration of the placeboInhalation use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Cystic Fibrosis (CF)
    Zystische Fibrose
    E.1.1.1Medical condition in easily understood language
    Cystic Fibrosis (Genetic disorder that affects mostly the lungs but also the pancreas, liver, kidneys, and intestine.)
    Zystische Fibrose (Erbliche Erkrankung, die hauptsächlich die Lunge betrifft, aber auch die Bauchspeicheldrüse, Leber, Nieren und den Darm.)
    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
    To investigate pharmacokinetics of single nebulised doses of RPL554 in patients with Cystic Fibrosis.
    E.2.2Secondary objectives of the trial
    - To investigate the bronchodilator effect on peak forced expiratory volume in 1 second after single nebulised doses of RPL554 as compared to placebo.
    - To investigate the bronchodilator effect on area under the curve forced expiratory volume in 1 second over 4, 6 and 8 hours of single nebulised doses of RPL554, as compared to placebo.
    - To assess the tolerability and safety of single nebulised doses of RPL554 in patients with Cystic Fibrosis.
    Exploratory Objective:
    - To examine the anti-inflammatory effects of single nebulised doses of RPL554 in patients with Cystic Fibrosis.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Sign an informed consent document indicating they understand the purpose of and procedures required for the study and are willing to participate in the study.
    2. Male or female aged ≥18 years at the time of informed consent. Females of childbearing potential must have been using a consistent and reliable form of contraception (see protocol appendix 1) from the last menses before the first study treatment administration, and must commit to continue to do so during the study and for 3 months after the last dose of study treatment.
    3. Have a 12-lead ECG recording at screening (Visit 1) and Visit 2 pre-dose showing the following:
    - Heart rate between 45 and 100 beats per minute
    - QT interval corrected for heart rate using Fridericia’s formula (QTcF) interval ≤450 msec
    - QRS interval ≤120 msec
    - PR Interval ≤220 msec
    - No clinically significant abnormality including morphology (e.g. left bundle branch block, atrioventricular nodal dysfunction, ST segment abnormalities)
    4. Capable of complying with all study restrictions and procedures including ability to use the study nebuliser correctly.
    5. Body mass index (BMI) between 18 and 30 kg/m2 (inclusive) with a minimum weight of 40 kg.
    6. Patients with a genetic diagnosis of CF.
    7. Spirometry at screening demonstrating an FEV1 ≥40% and ≤80% of predicted normal.
    8. Capable of withdrawing from long acting bronchodilators for 48 hours prior to study visits, and short acting bronchodilators for 8 hours prior to study visits.
    9. Clinically stable CF in the 2 weeks prior to randomisation (Visit 2).
    E.4Principal exclusion criteria
    1. History of cirrhotic liver disease or portal hypertension.
    2. CF exacerbation requiring hospitalisation in the month prior to screening (Visit 1) or prior to randomisation (Visit 2).
    3. Use of oral or intravenous antibiotics (in additional to usual maintenance therapy) in the 2 weeks prior to screening (Visit 1) or randomisation (Visit 2).
    4. Other non-CF related respiratory disorders: Patients with a current diagnosis of active tuberculosis, lung cancer, sarcoidosis, sleep apnoea, known alpha-1 antitrypsin deficiency or other active pulmonary diseases.
    5. Previous lung resection or lung transplant.
    6. History of, or reason to believe a patient has, drug or alcohol abuse within the past 3 years.
    7. Received an experimental drug within 3 months or five half-lives, whichever is longer.
    8. Patients with a history of chronic uncontrolled disease, unless CF related, including, but not limited to, cardiovascular (including arrhythmias), endocrine, active hyperthyroidism, neurological, hepatic, gastrointestinal, renal, haematological, urological, immunological or ophthalmic diseases that the Investigator believes are clinically significant.
    9. Documented cardiovascular disease: angina, recent or suspected myocardial infarction, congestive heart failure, a history of unstable, or uncontrolled hypertension, or has been diagnosed with hypertension in last 3 months.
    10. Has had major surgery, (requiring general anaesthesia) in the 6 weeks prior to screening (Visit 1) or will not have fully recovered from surgery, or planned surgery through the end of the study.
    11. Infection with nontuberculous mycobacteria, methicillin-resistant Staphylococcus aureus (MRSA), or Burkholderia species.
    12. Use of immune-suppression; long term use of prednisolone >10mg/day.
    13. History of malignancy of any organ system within 5 years with the exception of localised skin cancers (basal or squamous cell).
    14. Clinically significant abnormal values for safety laboratory tests (haematology, biochemistry or urinalysis) at screening (Visit 1), as determined by the Investigator.
    15. A disclosed history or one known to the Investigator, of significant non-compliance in previous investigational studies or with prescribed medications.
    16. Requires oxygen therapy on a regular basis.
    17. Pregnancy or lactation (female subjects only).
    18. Any other reason that the Investigator considers makes the patient unsuitable to participate.
    E.5 End points
    E.5.1Primary end point(s)
    RPL554 pharmacokinetic parameters (AUC, Cmax, tmax, t1/2).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Treatment Visits (Visits 2 - 4): Pre-dose and up to 24 hours post-dose.
    E.5.2Secondary end point(s)
    1. Peak and AUC FEV1 over 4, 6 and 8 hours
    2. Determination of onset of action
    3. Determination of duration of action
    4. Safety and tolerability:
    a) Continuous monitoring of adverse events
    b) Laboratory safety tests [haematology, biochemistry and urinalysis]
    c) 12-lead ECG (including QTcF and heart rate), supine vital signs [blood pressure and pulse rate] over 8 hours
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Treatment Visits (Visits 2 - 4): Pre-dose, and 15, 30 min, 1, 2, 4, 6, 8 and 24 hours post-dose.
    2. Treatment Visits (Visits 2 - 4): Pre-dose, and 15, 30 min, 1, 2, 4, 6, 8 and 24 hours post-dose.
    3. Treatment Visits (Visits 2 - 4): Pre-dose, and 15, 30 min, 1, 2, 4, 6, 8 and 24 hours post-dose.
    4. a) Screening to End of study.
    b) Screening and End of study.
    c) ECG at pre-dose, 1, 2, 4 and 8 hours post- dose. Supine vital signs at pre-dose, 30 min, 1, 2, 4, 6 and 8 hours post-dose.
    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 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 No
    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 No
    E.8.1.6Cross over Yes
    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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA2
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee No
    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 months3
    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 months5
    E.8.9.2In all countries concerned by the trial days30
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1Number of subjects for this age range: 0
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) No
    F.1.1.5.1Number of subjects for this age range: 0
    F.1.1.6Adolescents (12-17 years) No
    F.1.1.6.1Number of subjects for this age range: 0
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 10
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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 10
    F.4.2.2In the whole clinical trial 10
    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)
    A final assessment of all subjects will be made at the follow-up visit. All AEs will be followed-up until satisfactory resolution. Subjects will not be discharged from the study until they have attended their final assessment. After the end of this study, further post-study therapy is not necessary since subjects will continue their regular prescribed medication. They will stay under the surveillance of their attending physician.
    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 N/A
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2017-10-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-11-13
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2017-11-03
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