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    The EU Clinical Trials Register currently displays   42869   clinical trials with a EudraCT protocol, of which   7063   are clinical trials conducted with subjects less than 18 years old.
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    EudraCT Number:2020-005169-15
    Sponsor's Protocol Code Number:GB002-2102
    National Competent Authority:Czechia - SUKL
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-04-07
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedCzechia - SUKL
    A.2EudraCT number2020-005169-15
    A.3Full title of the trial
    An Open-label Extension Study Evaluating the Long-term Safety and Efficacy of Oral Inhalation of GB002 for the Treatment of WHO Group 1 Pulmonary Arterial Hypertension (PAH)
    Otevřená, rozšířená studie k posouzení dlouhodobé bezpečnosti a účinnosti perorální inhalace přípravku GB002 při léčbě plicní arteriální hypertenze (PAH) skupiny 1 dle WHO
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Clinical Study of Inhaled GB002 for the Treatment of WHO Group 1 Pulmonary Arterial Hypertension (PAH)
    A.4.1Sponsor's protocol code numberGB002-2102
    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 SponsorGB002, 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 supportGB002, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGB002, Inc.
    B.5.2Functional name of contact pointClinical Trials Information Website
    B.5.3 Address:
    B.5.3.1Street Address3013 Science Park Road, Suite 200
    B.5.3.2Town/ citySan Diego, CA
    B.5.3.3Post code92121
    B.5.3.4CountryUnited States
    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/3/19/2158
    D.3 Description of the IMP
    D.3.1Product nameGB002 Capsules
    D.3.2Product code GB002
    D.3.4Pharmaceutical form Inhalation powder, hard capsule
    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 INNSeralutinib
    D.3.9.1CAS number 1619931-27-9
    D.3.9.2Current sponsor codeGB002
    D.3.9.3Other descriptive name(S)-3-((3-(1-((6-(3,4-DIMETHOXYPHENYL)PRYAZIN-2- YL)AMINO)ETHYL)PHENYL)CARBAMOYL)-5-METHYLPRIDIN-1-IUM
    D.3.9.4EV Substance CodeSUB195876
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number15
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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 Yes
    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
    Pulmonary Arterial Hypertension (PAH)
    E.1.1.1Medical condition in easily understood language
    WHO Group 1 pulmonary arterial hypertension is associated with raised blood pressure in the pulmonary artery due to abnormalities in the small branches of the pulmonary artery, the arterioles.
    E.1.1.2Therapeutic area Diseases [C] - Cardiovascular Diseases [C14]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10077739
    E.1.2Term Pulmonary arterial hypertension WHO functional class I
    E.1.2System Organ Class 100000004855
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Evaluate the long-term safety and tolerability of orally inhaled GB002 in subjects with World Health Organization (WHO) Group 1 PAH.
    E.2.2Secondary objectives of the trial
    • Evaluate the long-term effect of orally inhaled GB002 on exercise capacity.

    • Evaluate the pharmacodynamics (PD) of orally inhaled GB002;
    • Evaluate additional long-term effects of orally inhaled GB002;
    • Determine the effect of GB002 on Risk Score Category;
    • Evaluate additional long-term safety of orally inhaled GB002.
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Functional Respiratory Imaging (Sub-study)
    Functional respiratory imaging (FRI) by high resolution computed tomography (CT) scan will be conducted in subjects with prior CT imaging in the parent study at designated timepoints in the SoA. The study will use specialized software to determine change from baseline in pulmonary vasculature blood volume, pulmonary blood volume as a percent of total lung volume, fibrosis score, and image-based ventilation to perfusion score.
    E.3Principal inclusion criteria
    Subjects must meet ALL the following inclusion criteria to be enrolled into the extension:

    Age and Sex
    1. Adult female subjects aged 18 to 80 years, inclusive, or adult male subjects aged 50 to 80 years, inclusive, at the time of signing the informed consent form (ICF).

    Type of Subject and Disease Characteristics
    2. Subjects must have completed a prior GB002 PAH study and, in the opinion of the Investigator and Sponsor, have been compliant with study procedures and have completed treatment with IP through parent study EOT visit.
    3. Treatment with standard of care PAH disease-specific background therapies (stable dose).

    Contraception use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
    4. Women of childbearing potential must have a negative urine or serum pregnancy test (minimum sensitivity 25 IU/L or equivalent units of human chorionic gonadotropin [hCG]) at extension enrolment visit before first administration of GB002 in this study. If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test is required, and results must be negative.
    5. Women of nonchildbearing potential: Evidence of post-menopausal status. Women will be considered post-menopausal if they have been amenorrheic for 12 months without an alternative medical cause. The following age-specific requirements apply:

    − Women < 50 years of age would be considered post-menopausal if they have been amenorrheic for 12 months or more following cessation of exogenous hormonal treatments and if they have luteinizing hormone and follicle-stimulating hormone levels in the post-menopausal range for the institution or underwent surgical sterilization (bilateral oophorectomy, bilateral salpingectomy, or hysterectomy).
    − Women ≥ 50 years of age would be considered post-menopausal if they have been amenorrheic for 12 months or more following cessation of all exogenous hormonal treatments, had radiation-induced menopause with last menses > 1 year ago, had chemotherapy-induced menopause with last menses > 1 year ago, or underwent surgical sterilization (bilateral oophorectomy, bilateral salpingectomy, or hysterectomy).

    6. Women of childbearing potential who are not abstinent and intend to be sexually active with a non-sterilized male partner must be willing to use a highly effective method of contraception from consent through 30 days following the last administration of GB002; acceptable methods include hormonal contraception (oral contraceptives – as long as on stable dose, patch, implant, or injection), intrauterine devices or other form of highly effective contraception.
    Note: Abstinence is acceptable if this is the usual lifestyle and preferred contraception for the subject. Periodic abstinence, the rhythm method, and the withdrawal method are not acceptable.
    Note: A vasectomized partner is acceptable.
    7. Male subjects: Non-sterilized male subjects who are not abstinent and intend to be sexually active with a female partner of childbearing potential must use a male condom from consent through 90 days after the last dose of GB002. Male subjects should refrain from sperm donation throughout this period (except for male subjects participating in fertility analysis as part of this protocol).
    Note: Abstinence is acceptable if this is the usual lifestyle and preferred contraception for the subject. Periodic abstinence, the rhythm method, and the withdrawal method are not acceptable.

    Informed Consent
    8. Evidence of a personally signed and dated informed consent document indicating that the subject has been informed of all pertinent aspects of the study prior to initiation of any protocol-mandated procedures.
    9. Willingness and ability to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.
    E.4Principal exclusion criteria
    The subject must be excluded from participating in the study if he/she meets any of the following:

    Medical Conditions
    1. Persistent and clinically significant systemic hypertension or hypotension.
    2. Interval history of newly developed left-sided heart disease with onset or severity increased after participation in the parent or lead in-study, and/or clinically significant cardiac disease, unless reviewed and approved as eligible to participate in the study by Sponsor’s Medical Monitor (or designee), including but not limited to any of the following:
    a. Aortic or mitral valve disease (stenosis or regurgitation) defined as greater than mild aortic insufficiency, mild aortic stenosis (AS), mild mitral stenosis (MS), moderate mitral regurgitation (MR);
    b. Mechanical or bioprosthetic cardiac valve;
    c. Pericardial constriction or pericardial effusion with tamponade physiology;
    d. Restrictive or congestive cardiomyopathy;
    e. Left ventricular ejection fraction (LVEF) ≤50%.
    f. Symptomatic coronary disease;
    g. Significant (2+ for regurgitation) valvular disease other than tricuspid or pulmonary regurgitation;
    h. Acutely decompensated left heart failure within 1 month (30 days) of extension enrolment visit;
    i. History of severe and untreated obstructive sleep apnoea.
    3. Potentially life-threatening cardiac arrhythmia with an ongoing risk.
    4. Uncontrolled bacterial, viral, or fungal infections which require systemic therapy.
    5. Other severe acute or chronic medical or laboratory abnormality that may increase the risk associated with study participation or GB002 administration or may interfere with the interpretation of study results and, in the judgment of the Investigator, would make the subject inappropriate for entry into this study; including but not necessarily limited to the following: malignancy within 5 years of extension enrolment visit, with the exception of effectively treated or excised localized non-metastatic basal cell carcinoma of the skin and in situ carcinoma of the cervix; psychiatric disorder that compromises ability to give informed consent, substance abuse, coagulopathy, history of stroke, transient ischemic attack (TIA) requiring concurrent oral coagulation therapy, or intracranial haemorrhage; history of pulmonary embolus or deep vein thrombosis (DVT), history of vasovagal syncope with phlebotomy.
    6. Currently pregnant or breastfeeding or intends to become pregnant during the duration of the study.
    7. History of portopulmonary hypertension or portal hypertension classified as Child-Pugh Class A or higher.
    8. Subjects with a history of severe milk protein allergy. In addition, subjects with known intolerance or hypersensitivity to lactose who, in the opinion of the investigator, may experience severe symptoms following the ingestion of lactose.
    9. Current use of inhaled tobacco and/or inhaled marijuana. Ingestible or topical marijuana is allowed, per local restrictions and regulations.
    10. Documented history of alcohol abuse and/or history of utilizing drugs of abuse (amphetamines, methamphetamines, cocaine, phencyclidine [PCP]).
    11. Have any other condition or reason that, in the opinion of the Investigator and/or the Sponsor’s Medical Monitor (or designee), would prohibit the subject from participating in the study.

    Diagnostic Assessments
    The most recent laboratory assessment from the parent study may be used to evaluate laboratory-associated exclusion criterion, if performed within 6 weeks (± 2 days) of the extension enrolment visit.
    12. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 2 × upper limit of normal (ULN) or total bilirubin ≥ 2 × ULN.
    13. Chronic renal insufficiency as defined by an estimated glomerular filtration rate (eGFR) < 45 mL/min/1.73m2 via CKD-EPI at extension enrolment visit or requires dialytic therapy or hemofiltration.
    14. Haemoglobin (Hgb) concentration <8.5 g/dL.
    15. Absolute neutrophil count (ANC) < 1 x 109/L.
    16. Platelet count <50 x 109/L.
    17. Body weight ≤40 kg at extension enrolment visit.
    Prior Therapy
    18. Use of inhaled prostanoids.
    19. Chronic use of oral anticoagulants (i.e., coumadin or novel oral anticoagulant [NOAC]); if on coumadin or a NOAC it is clinically acceptable to be withdrawn 1 month prior to start of GB002.
    20. Chronic use of any prohibited medication.
    E.5 End points
    E.5.1Primary end point(s)
    • Incidence and frequency of treatment-emergent adverse events (TEAEs).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline, 2, 4, 8, 12, 16, 24, 32, 40, 48, 60 and 72 weeks and 4 weeks post last GB002 dose.
    E.5.2Secondary end point(s)
    Secondary Endpoint
    • Change in the distance achieved on six-minute walk test (6MWT), (Δ6MWD).

    Exploratory Endpoints
    • Changes in N-terminal pro b-type natriuretic peptide (NT-proBNP).
    • Changes in various biomarkers measured in blood samples and/or circulating cells from baseline to end of treatment.

    Long-term Effects
    • Changes in:
    − WHO FC;
    − European QOL - 5 Dimensions - 5 Levels (EQ-5D-5L);
    − Right ventricle (RV) function by imaging (echocardiography);
    − Sub-study: Functional respiratory imaging (FRI) by computed tomography (CT) scan (in subjects with prior CT imaging).

    • Time from first dose of GB002 to first event of protocol-defined clinical worsening event, assessed by measuring the first occurrence of any one of the following events:

    − Death (all causes).

    − Hospital admission for worsening PAH, as a result of any of the following:
    o Non-elective hospitalization caused by clinical conditions directly related to PAH and/or right heart failure;
    o Need for intravenous (IV) diuretics (more than a single dose in 24 hours);
    o Lung or heart/lung transplantation;
    o Atrial septostomy;
    o Initiation of parenteral (IV infusion or subcutaneous injection) therapy with a prostacyclin (if not previously utilizing parenteral prostacyclin therapy).

    − Disease progression, defined as:
    o Worsening symptoms of right heart failure requiring initiation of a new PAH disease-specific medication or an increase in dose, or change in disease-specific background PAH medications or initiation of chronic oxygen therapy (i.e., requires oxygen for >24 hours with the intent of long- term use); or
    o A decrease in distance on 6MWT (6MWD) of at least 15% from extension enrolment visit, directly related to PAH progression, confirmed by 2 assessments of 6MWD performed at 2 consecutive visits and worsening in WHO FC for subjects with WHO FC I/II/III; or
    o Worsening Risk Score Category.

    Risk Score category
    • Change from Baseline in Risk Score Category.

    Additional Long-Term Safety
    • Incidence and frequency of serious TEAEs (SAEs) and AESIs;
    • Changes in clinical laboratory parameters, ECG parameters, and vital signs.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Secondary Endpoint: Baseline, 4, 8, 12, 16, 24, 32, 40, 48, 60, 72 and 4 weeks post last GB002 dose.

    Exploratory Endpoints: Baseline to Week 76 as specified in protocol.
    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 No
    E.6.7Pharmacodynamic Yes
    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) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E. 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.8.3 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 EEA22
    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
    United Kingdom
    United States
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months10
    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: 67
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 13
    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 state1
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 35
    F.4.2.2In the whole clinical trial 80
    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)
    The Investigator should resume standard of care treatment, including treatment with appropriate medications, as deemed necessary.
    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 Decision2021-06-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-06-17
    P. End of Trial
    P.End of Trial StatusOngoing
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