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    Summary
    EudraCT Number:2017-003319-21
    Sponsor's Protocol Code Number:PTI-808-01
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2018-07-03
    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 ConcernedUK - MHRA
    A.2EudraCT number2017-003319-21
    A.3Full title of the trial
    A Phase 1 / 2 Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of PTI-808 in Healthy Adult Subjects and in Adults with Cystic Fibrosis
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study Assessing the Safety, Tolerability and Pharmacokinetics of PTI-808 in Healthy Adult Subjects and in Adults with Cystic Fibrosis
    A.4.1Sponsor's protocol code numberPTI-808-01
    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 SponsorProteostasis Therapeutics, 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 supportProteostasis Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSFL Regulatory Services GmbH
    B.5.2Functional name of contact pointWalter Fuerst
    B.5.3 Address:
    B.5.3.1Street AddressEuropaplatz 2/1/2
    B.5.3.2Town/ cityWien
    B.5.3.3Post code1150
    B.5.3.4CountryAustria
    B.5.4Telephone number+431253915587
    B.5.6E-mailw.fuerst@sfl-services.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 nameN-(5-hydroxy-2,4-bis(trimethylsilyl)phenyl)-4-oxo-1,4-dihydroquinoline-3-carboxamide
    D.3.2Product code PTI-808
    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 INNNone
    D.3.9.1CAS number Not assigned
    D.3.9.2Current sponsor codePTI-808
    D.3.9.3Other descriptive namePTI-808
    D.3.9.4EV Substance CodeSUB189687
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number10 to 150
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameSodium (R)-8-methyl-2-(3-methylbenzofuran-2-yl)-5-(1-(tetrahydro-2H-pyran-4-yl)ethoxy)quinoline-4-ca
    D.3.2Product code PTI-801
    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 INNNone
    D.3.9.1CAS number Not assigned
    D.3.9.2Current sponsor codePTI-801
    D.3.9.3Other descriptive namePTI-801
    D.3.9.4EV Substance CodeSUB189686
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number50 to 100
    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: 3
    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 nameN-(trans-3-(5-((R)-1-hydroxyethyl)-1,3,4-oxadiazol-2-yl)cyclobutyl)-3-phenylisoxazole-5-carboxamide
    D.3.2Product code PTI-428
    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 INNNone
    D.3.9.1CAS number Not assigned
    D.3.9.2Current sponsor codePTI-428
    D.3.9.3Other descriptive namePTI-428
    D.3.9.4EV Substance CodeSUB189650
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number5 to 100
    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
    D.8 Placebo: 2
    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
    D.8 Placebo: 3
    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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    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
    Part 1 of the study is only conducted in US. Primary objectives are:
    - Evaluate the safety and tolerability of a single oral dose and multiple oral doses of PTI-808 in healthy adults.
    - Evaluate the PK profile of a single oral dose and multiple oral doses of PTI-808 in healthy adults.
    - Evaluate the effect of food on the PK profile of PTI-808 in healthy adults.

    Part 2 of the study is only conducted in US. Primary objective is: Evaluate the safety and tolerability of PTI-808 co-administered with PTI-801 and PTI-428 in healthy adults.

    Part 3 of the study will be conducted in EU, US and Canada. Primary objective is: Evaluate the safety and tolerability of PTI-808 co-administered with PTI-801 and PTI-428 in subjects with cystic fibrosis.

    Part 4 of the study will be conducted in EU, US and Canada. Primary objective is: Evaluate the safety and tolerability of PTI-808 co-administered with PTI-801 and PTI-428 in subjects with cystic fibrosis.
    E.2.2Secondary objectives of the trial
    Part 1:
    Sec. obj.: safety and tolerability of a single oral dose of PTI-808 in healthy adults
    PD obj.: effect of PTI-808 on the QT interval

    Part 2:
    Sec. obj.: PK profile of PTI-808, PTI-801, and PTI-428 when PTI-808 is co-administered with PTI-801 and PTI-428 in healthy adults
    Expl. obj.: effect on genes expressed in nasal epithelial cells

    Part 3:
    Sec. obj.: Please see section 5.1 of the updated protocol
    Expl. obj.: sweat chloride concentrations, weight, BMI, blood glucose over time and treatment effect on genes expressed in nasal epithelial cells

    Part 4:
    Sec. obj.: PK profiles of PTI-808, PTI-801, and PTI-428 when PTI-808 is co-administered with PTI-801 with or without PTI-428 in subjects with cystic fibrosis, pulmonary function (FEV1) and sweat chloride concentration over time
    Expl. obj.: weight, BMI, blood glucose, disease-specific health-related quality of life over time and the treatment effect on genes expressed in nasal epithelial cells
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Study part 1 (in US only): A list of inclusion criteria for part 1 of the Study is available in the clinical study protocol number PTI-808-01 version 8.2; dated 22 October 2019 (section 3.4.1).

    Study part 2 (in US only): A list of inclusion criteria for part 2 of the Study is available in the clinical study protocol number PTI-808-01 version 8.2; dated 22 October 2019 (section 4.4.1).

    Study part 3 (in EU, US and Canada):
    Inclusion criteria are: A list of inclusion criteria for part 3 of the Study is available in the clinical study protocol number PTI-808-01 version 8.2; dated 22 October 2019 (section 5.4.1).

    Study part 4 (in EU, US and Canada):
    Inclusion criteria are:
    1. Understands the full nature and purpose of the study, including possible risks and side
    effects, is willing and able to comply with all compulsory study procedures, and provides
    written informed consent/permission prior to any study procedures being performed
    2. Adult male or female, ≥18 years of age, at the time of informed consent
    3. Confirmed diagnosis of CF as follows:
    a. For homozygous subjects: Confirmed diagnosis of CF with the F508del CFTR homozygous genotype on record, along with clinical findings consistent with CF, such as, chronic sinopulmonary disease or gastrointestinal/nutritional abnormalities. The medical monitor’s approval is not required for homozygous subjects with a sweat chloride value ≥60 mmol/L at screening or documented in the form of a laboratory report in the subject’s medical record. For subjects with a sweat chloride value <60 mmol/L at screening and no documented historical value of ≥60 mmol/L, medical monitor approval is required based on documented evidence of chronic sinopulmonary disease manifested by at least 1 of the following:
    - Persistent colonization/infection with typical CF pathogens, including but not limited to, Staphylococcus aureus, Haemophilus influenzae, and/or Pseudomonas aeruginosa
    - Chronic cough and sputum production
    - Persistent chest radiograph abnormalities (eg, bronchiectasis, atelectasis, infiltrates, hyperinflation)
    - Nasal polyps, chronic sinusitis, or radiographic or computed tomographic abnormalities of the paranasal sinuses
    b. For heterozygous subjects: Confirmed diagnosis of CF with only one copy of the F508del CFTR mutation on record, along with clinical findings consistent with CF, such as, chronic sinopulmonary disease or gastrointestinal/nutritional abnormalities;
    AND
    a sweat chloride value of ≥60 mmol/L based on quantitative pilocarpine iontophoresis (as documented in the subject’s medical record or as confirmed at the screening visit)
    4. Subject must be able to produce a valid sweat sample (quantity-sufficient; ie, >15 μL) at the screening visit. If the sweat volume is insufficient then the sweat chloride collection may be repeated once with approval from the medical monitor.
    5. Clinically stable CF disease in the opinion of the investigator with no significant changes in health status within 14 days prior to Day 1
    6. FEV1 40% to 90%, predicted, inclusive (subjects with FEV1 <40% or >90% predicted at Day -1 must be reviewed with the medical monitor to ensure subject meets criteria to continue in the study)
    7. Pulse oximetry >92% at rest
    8. BMI ≥16 and <30 kg/m2
    9. Subjects of childbearing potential must meet contraception requirements (Section 8.24.2)
    10. Non-smoker and non-tobacco user for a minimum of 30 days prior to screening, and agrees not to smoke or use tobacco for the duration of the study
    E.4Principal exclusion criteria
    Study part 1 (in US only): A list of exclusion criteria for part 1 of the Study is available in the clinical study protocol number PTI-808-01 version 8.2; dated 22 October 2019 (section 3.4.2).

    Study part 2 (in US only): A list of exclusion criteria for part 2 of the Study is available in the clinical study protocol number PTI-808-01 version 8.2; dated 22 October 2019 (section 4.4.2)

    Study part 3 (in EU, US and Canada): A list of exclusion criteria for part 3 of the Study is available in the clinical study protocol number PTI-808-01 version 8.2; dated 22 October 2019 (section 5.4.2).

    Study part 4 (in EU, US and Canada):
    Exclusion criteria are:
    1. History or current evidence of any clinically significant cardiac (eg, heart failure, left ventricular hypertrophy, myocardial infarction, and arrhythmia), endocrinologic, hematologic, hepatobiliary (eg, clinically significant cirrhosis with or without portal hypertension), immunologic, metabolic, urologic, pulmonary (besides CF), neurologic (eg, subarachnoid hemorrhage, intracranial hemorrhage, cerebrovascular accident, intracranial trauma, and autonomic neuropathy), dermatologic, psychiatric, renal, or other major disease, that is unstable or could interfere with the subject’s participation in or completion of the study, in the opinion of the investigator (eg, subjects with CFRD can participate in the study if the CFRD is well controlled using a stable medication regimen)
    2. Clinically significant screening results that would exclude subject from the study (eg, medical histories, physical exams, ECGs, vital signs, pulse oximetry, laboratory profiles) or any conditions that, would make the subject unsuitable for enrollment or could interfere with the subject’s participation in or completion of the study, in the opinion of the investigator. The medical monitor must be contacted for review of any subjects with screening results or conditions that may make them unsuitable for enrollment or could interfere with participation in or completion of the study.
    3. Prolonged QTcF >450 msec at screening
    4. Abnormal liver function as defined by AST, ALT, gamma-glutamyl transferase (GGT), or alkaline phosphatase ≥3 times or total bilirubin ≥2 times upper limit of the normal range
    5. Hemoglobin <10 g/dL
    6. Platelet count <150,000 cells/mm3
    7. Abnormal renal function at screening defined as creatinine clearance <60 mL/min using the Modified Diet in Renal Disease (MDRD)
    8. Hospitalization, sinopulmonary infection, CF exacerbation, or other clinically significant infection or illness (in the opinion of the investigator) requiring an increase or addition of medication, such as antibiotics or corticosteroids, within 28 days of Day 1
    9. Lung infection with organisms associated with a more rapid decline in pulmonary status (eg, Burkholderia cenocepacia, Burkholderia dolosa, and Mycobacterium abscessus). For subjects who have had a history of a positive culture in the past, the investigator will apply the following criteria to establish whether the subject is free of infection with such organisms:
    - The subject has had 2 respiratory tract cultures negative for these organisms within the 12 months before the screening visit, with no subsequent positive cultures.
    - These 2 respiratory tract cultures were separated by at least 3 months, and 1 of them was obtained within 6 months before the screening visit.
    10. Subject is currently taking or has taken a CFTR modulator within 14 days prior to the screening visit
    11. Participation in another clinical trial or treatment with an investigational agent within 30 days or 5 half-lives, whichever is longer, prior to screening. The duration of the elapsed time may be longer if required by local regulations.
    12. History of cancer (excluding cervical carcinoma in situ and non-melanoma skin cancer with curative therapy for at least 5 years prior to screening)
    13. History of organ or hematologic transplantation
    14. History or current evidence of alcohol or drug abuse or dependence within 12 months of screening, in the opinion of the investigator
    15. Positive blood screen for HIV, HBsAg, or HCVAb at screening
    16. Initiation of any new chronic therapy (eg, ibuprofen, hypertonic saline, azithromycin, Pulmozyme®, Cayston®, TOBI®) or any change in chronic therapy (excluding pancreatic enzyme replacement therapy) within 28 days prior to Day 1
    17. Known or suspected hypersensitivity or idiosyncratic reaction to the study drugs or any components thereof
    18. Pregnant or nursing women
    19. Special or vulnerable status (eg, institutionalized, or person related to or an employee of the sponsor or investigator)
    E.5 End points
    E.5.1Primary end point(s)
    Part 1 (in US only):
    Primary safety endpoints for Healthy Volunteer SAD and MAD are:
    - Clinical laboratory tests: hematology, chemistry, urinalysis, and coagulation
    - 12-lead electrocardiograms (ECGs)
    - Physical examinations
    - Vital signs
    - Adverse events (AEs)

    Primary PK endpoints for Healthy Volunteer SAD and MAD are:
    - For plasma PTI-808: Parameters include, but not limited to, apparent terminal half-life (t1/2), time to reach maximum plasma concentration (Tmax), maximum plasma concentration (Cmax), AUC(0-24), AUC from time 0 to time of last measurable concentration (AUC0-last), AUC from time 0 to infinity (AUC0-inf), percentage of AUC(0-inf) due to extrapolation from time of last measurable concentration to infinity (AUC%extrap), apparent total clearance (CL/F), apparent volume of distribution (V/F), and terminal elimination rate constant (λz) using non compartmental methods as appropriate

    - For urine PTI-808 (Healthy Volunteer MAD only): Parameters include, but are not limited to, cumulative amount of PTI-808 excreted unchanged in urine (Ae) and renal clearance (CLR) as appropriate

    Primary PK endpoints for Healthy Volunteer Food Effect are:
    - For plasma PT-808 with fasting and after a high fat, high calorie meal: parameters include, but not limited to, t1/2, Tmax, Cmax, AUC(0-24), AUC(0-last), AUC(0-inf), AUC(%extrap), CL/F, V/F, and λz, using non-compartmental methods, as appropriate

    Part 2 (in US only):
    Safety and tolerability endpoints are :
    - Clinical laboratory tests (hematology, serum chemistry, urinalysis, and coagulation), 12-lead ECGs, physical exams, vital signs, and AEs

    Part 3 (in EU, US and Canada)
    Primary safety and tolerability endpoints are:
    - Clinical laboratory tests (hematology, serum chemistry, urinalysis, and coagulation) 12-lead ECGs, physical exams, vital signs, and AEs

    Part 4 (in EU, US and Canada)
    Primary safety and tolerability endpoints are:
    - Clinical laboratory tests (hematology, serum chemistry, urinalysis, and coagulation), 12 lead ECGs, physical exams, vital signs, and AEs
    E.5.1.1Timepoint(s) of evaluation of this end point
    Part 1 (in US only):
    - HV SAD (see Table 15-1 in the appendices of the protocol)
    - HV MAD (see Table 15-2 in the appendices of the protocol)
    - HV Food Effect (see Table 15-3 in appendices of the protocol)

    Part 2 (in US only):
    - HV Co-Administration (see Table 15-4 in appendices of the protocol)

    Part 3 (in EU, US and Canada):
    - CF MAD Co-Administration (see Table 15-5 in appendices of the protocol).

    Part 4 (in EU, US, Canada):
    - CF Cohorts (see Table 15-6 in appendices of the protocol).
    - The safety evaluation is performed at Day1, Day 2, Day 7 (± 1 day), Day 14 (± 1 day), Day 21 (± 1 day), Day 28 (-1 day), Day 29 and Day 42 (± 4 days).
    E.5.2Secondary end point(s)
    Part 1 (US only):
    The secondary safety endpoints for HV Food Effect are:
    - Clinical laboratory tests: hematology, chemistry, urinalysis, and coagulation
    - 12-lead ECGs
    - Physical exams
    - Vital signs
    - AEs

    The Pharmacodynamic endpoint for HV SAD, HV MAD and HV Food Effect is:
    - Holter monitoring

    Part 2 (in US only):
    Endpoints to evaluate PK of PTI-808, PTI-801, and PTI-428 in plasma include, but are not limited to:
    - T1/2, Tmax, Cmax, AUC(0-last), and AUC(0-inf) using non-compartmental methods, as appropriate

    Exploratory endpoint:
    - Change in nasal epithelial mRNA and protein expression

    Part 3 (in EU, US and Canada)
    Secondary endpoints are:
    - For plasma PTI-808, PTI-801, and PTI-428: PK parameters including, but not limited to Tmax, Cmax, and AUC(0-last), as appropriate
    - Change in FEV1 over time

    Exploratory endpoints are:
    - Change in sweat chloride concentrations over time
    - Change in weight and BMI over time
    - Change in blood glucose over time
    - Change in nasal epithelial mRNA and protein expression

    Part 4 (in EU, US and Canada)
    Secondary endpoints are:
    - For plasma PTI-808, PTI-801, and PTI-428: PK parameters including, but not limited to Tmax, Cmax, and AUC(0-last), as appropriate
    - Change in FEV1 over time
    - Change in sweat chloride concentrations over time

    Exploratory endpoints are:
    Weight, BMI, blood glucose, Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain, and nasal epithelial mRNA and protein expression
    E.5.2.1Timepoint(s) of evaluation of this end point
    Part 1:
    - HV SAD (see Table 15-1 of the protocol)
    - HV MAD (see Table 15-2 of the protocol)
    - HV Food Effect (see Table 15-3 of the protocol)

    Part 2:
    - HV Co-Administration (see Table 15-4 of the protocol)

    Part 3:
    - CF MAD Co-Administration (see Table 15-5 of the protocol).

    Part 4:
    - CF Cohorts (see Table 15-6 of the protocol).
    - Evaluation timepoints are:
    - For plasma PTI-808, PTI-801, and PTI-428: PK parameters: day 1, 2, 7, 14, 21, 28, 29
    - FEV1 and sweat chloride concentrations over time: day 7, 14, 21, 28 and 42
    - Weight and BMI: day 1, 7, 14, 21, 28, 42
    - Blood glucose: day 28 and 42
    - Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain and in nasal epithelial mRNA and protein expression: day 14, 28 and 42
    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 No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    Safety, Tolerability, and PK of PTI-808 in Healthy Adult Subjects and in Adults with Cystic Fibrosis
    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 trial8
    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 concerned7
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA19
    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
    Denmark
    France
    Germany
    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
    The end of the study is defined as the date of the last study visit for the last subject (which will be their follow-up visit).
    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 months24
    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 months24
    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: 240
    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 Yes
    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 36
    F.4.2.2In the whole clinical trial 240
    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 European Cystic Fibrosis Society- Clinical Trial Network (ECFS- CTN)
    G.4.3.4Network Country Belgium
    G.4 Investigator Network to be involved in the Trial: 2
    G.4.1Name of Organisation Clinical Trials Accelerator Platform (CTAP)
    G.4.3.4Network Country United Kingdom
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2018-05-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-06-04
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2019-12-23
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