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    Summary
    EudraCT Number:2019-001598-10
    Sponsor's Protocol Code Number:PB1046-PT-CL-0004
    National Competent Authority:Greece - EOF
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2019-12-13
    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 ConcernedGreece - EOF
    A.2EudraCT number2019-001598-10
    A.3Full title of the trial
    A Randomized, Double-Blind, Parallel Group, Phase 2 Study to Assess the Safety, Tolerability, and Efficacy of Once Weekly Subcutaneous Injections of a Sustained-Release VIP Analogue, PB1046, in Adult Subjects with Symptomatic Pulmonary Arterial Hypertension
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Phase 2 Study to Assess Safety, Tolerability and Efficacy of Once Weekly Subcutaneous Injections of PB1046 (study drug) in Subjects With Symptomatic Pulmonary Arterial Hypertension
    A.4.1Sponsor's protocol code numberPB1046-PT-CL-0004
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03556020
    A.5.4Other Identifiers
    Name:US IND NumberNumber:135,417
    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 SponsorPhaseBio 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 supportPhaseBio Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPhaseBio Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointPB1046 Clinical Project Manager
    B.5.3 Address:
    B.5.3.1Street Address1 Great Valley Parkway, Suite 30
    B.5.3.2Town/ cityMalvern
    B.5.3.3Post codePA 19355
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1610981-6514
    B.5.5Fax number+1610981-6520
    B.5.6E-mailPB1046clinicaltrials@phasebio.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 nameVasomera (PB1046) Injection
    D.3.2Product code PB1046
    D.3.4Pharmaceutical form 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 available
    D.3.9.1CAS number N/A
    D.3.9.2Current sponsor codePB1046
    D.3.9.3Other descriptive namePB1046, VIP-ELP1-120, Vasomera
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    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 No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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 Yes
    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
    Symptomatic Pulmonary Arterial Hypertension
    E.1.1.1Medical condition in easily understood language
    PAH is a progressive and life-threatening disease which is characterized by high blood pressure in the lungs caused by abnormal constriction and remodeling of the arteries in the lungs.
    E.1.1.2Therapeutic area Diseases [C] - Cardiovascular Diseases [C14]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10064911
    E.1.2Term Pulmonary arterial hypertension
    E.1.2System Organ Class 10038738 - Respiratory, thoracic and mediastinal disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Assess the safety, tolerability, and effect on pulmonary vascular resistance (PVR) of dose titrated levels of PB1046 administered subcutaneously once-weekly for 16 weeks in subjects with symptomatic pulmonary arterial hypertension (PAH)
    E.2.2Secondary objectives of the trial
    • To investigate the effect of PB1046 on exercise capacity following treatment in PAH subjects
    • To investigate the effect of PB1046 on change in N-terminal fragment of the prohormone brain natriuretic peptide (NT-proBNP)
    • To investigate the effect of PB1046 on cardiopulmonary hemodynamics
    • To evaluate the multi-dose pharmacokinetic profile of individually dose-titrated PB1046 administered once-weekly for 16 weeks
    • To evaluate the immunogenicity profile of PB1046 PAH subjects
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects will be eligible for inclusion in the study if they meet all the following criteria at screening and up to the time of first dose as indicated:
    1. Male and female subjects with PAH, ≥18 and ≤ 79 years of age, who are symptomatic and have reduced exercise capacity due primarily to their PAH diagnosis and have been assessed by a qualified individual (i.e. physician, physician assistant, nurse practitioner) to be in NYHA/WHO functional class II or III
    2. Willing and able to sign a written informed consent prior to all study-related procedures
    3. Subjects with PAH belonging to one of the following subgroups of the NICE Clinical Classification of Pulmonary Hypertension Group 1:
    a. Idiopathic
    b. Heritable
    c. Drug or toxin-induced
    d. Associated with connective tissue disease, HIV infection, portal hypertension, congenital heart disease (pulmonary-to-systemic shunt, e.g., atrial septal defect (ASD) or patent ductus arteriosus (PDA), at least 1 year after surgical repair)
    4. Two 6MWT results > 50 m and < 550 m prior to randomization with results +/- 10% of each other. Note: Up to four tests may be conducted between Screening and Randomization for eligibility purposes (no more than two 6MWT may be performed on the same day, and must be completed at least two hours apart)
    5. Hemodynamic assessment of PAH by right heart catheterization (RHC) demonstrating elevated mPAP and PVR as indicated below during the Screening Period
    a. mPAP of ≥ 25 mmHg; and
    b. PVR ≥ 400 dyne•sec/cm5; and
    c. PCWP or left ventricular end diastolic pressure (LVEDP) of ≤ 12 mmHg if PVR ≥ 400 and < 500 dynes•sec/cm5; or PCWP/LVEDP ≤ 15 mmHg if PVR ≥ 500 dynes•sec/cm5
    6. Body mass index (BMI) ≥ 18 kg/m2 and ≤ 40 kg/m2 at screening
    7. Meet the following criteria determined by pulmonary function tests completed no more than 24 weeks prior to screening
    a. Forced expiratory volume in one second (FEV1) ≥ 55% of predicted normal
    b. FEV1: FVC (forced vital capacity) ratio ≥ 0.60
    8. Agrees to use a highly effective method of contraception (both male and female patients) throughout the entire study period and continuing for 30 days after their last dose of study drug if the possibility of conception exists. Highly effective methods of contraception include the following: combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, or transdermal); progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, implantable); intrauterine device (IUD); intrauterine hormone-releasing system; bilateral tubal occlusion; vasectomized partner (provided that partner is the sole sexual partner of the trial participant and that the vasectomized partner has received medical assessment of the surgical success); and sexual abstinence (defined as refraining from heterosexual intercourse during the entire period of risk associated with the study treatments). Female subjects < 45 years of age of non-childbearing potential are defined as being surgically sterile by bilateral tubal ligation, bilateral oophorectomy, or hysterectomy. Female subject 45 to 60 years of age, inclusive, who is post-menopausal for at least 1 year and have a follicle-stimulating hormone (FSH) level confirmation indicating post-menopausal status, will be considered to be of non-childbearing potential. Female subjects >60 years of age are considered post-menopausal and of non-childbearing potential
    9. Stable background medical regimen of up to 3 PAH disease-specific therapies for at least 3 months comprised of any combination of oral and/or inhaled or IV therapy and at stable doses for at least 30 days prior to Screening
    10. If a subject has historical diagnosis (prior to screening visit) of being positive for human immunodeficiency virus (HIV), hepatitis B surface antigen (HBsAg), or hepatitis C virus (HCV), must be clinically stable and if on therapy, must be on stable therapy for HIV or HCV for at least 3 months
    11. Willing and able to understand and follow instructions; return to the study unit for specified study visits; and able to participate in the study for the entire period
    E.4Principal exclusion criteria
    Subjects will be excluded from the study if they meet any of the following criteria:
    1. Concomitant medical disorder, condition, or history, that in the opinion of the Investigator would impair the subject’s ability to participate in or complete the requirements of the study
    2. Concomitant medical disorder that is expected to limit the subject’s life-expectancy to ≤ 1 year
    3. Pregnant or lactating female subjects
    4. First positive result from serology testing at Visit 1 (screening labs) for HIV, HBsAg, or HCV prior to randomization
    5. Participation in another investigational drug study within 30 days prior to screening or participating in a non-medication study which, in the opinion of the Investigator, would interfere with the study compliance or outcome assessments
    6. Use of subcutaneous prostanoid/prostacyclin therapy for PAH within 30 days prior to the screening visit
    7. More than mild mitral or aortic valve disease, left ventricular ejection fraction < 50%, or left ventricular regional wall motion abnormality suggestive of active coronary artery disease on documented 2D-echocardiography occurring within 12 months of Screening
    8. Sustained SBP < 95 mmHg and/or diastolic blood pressure (DBP) < 50 mmHg (confirmed by a duplicate seated reading) on at least 3 consecutive readings (self-monitored or office) at screening and prior to dosing, or overt symptomatic hypotension
    9. Sustained resting heart rate >110 beats per minute (BPM) (confirmed by duplicate assessments of office vital signs or consecutive ECG assessments) on at least 3 consecutive readings at screening and prior to dosing
    10. Clinically significant renal dysfunction based on Screening labs as measured by the estimated glomerular filtration rate (eGFR) of < 30 mL/min/1.73m2 as calculated by the IDMS-Traceable MDRD equation: eGFR = (mL/min/1.73 m2) = 175 x (serum creatinine in mg/dL) – 1.154 x (Age) – 0.203 x (0.742 if female) x (1.212 if African American) (conventional units)
    11. Significant liver dysfunction as measured by any one of the following at screening:
    a. alanine aminotransferase (ALT) >3.0 times ULN or
    b. aspartate aminotransferase (AST) >3.0 times ULN or
    c. serum bilirubin ≥ 1.6 mg/dL
    12. Known history of substance abuse within the past 1 year that in the opinion of the Investigator would impair the subject’s ability to participate in or complete the requirements of the study
    13. Any major surgical procedure within 90 days prior to screening or planned surgical procedure during the study period
    14. Any in-patient hospitalization (defined as greater than 23 hours) within 30 days of subject screening
    15. Enrollment within the past 3 months prior to screening or plans to enroll during the study into a cardiopulmonary rehabilitation program
    16. Other medical or psychiatric condition which, in the opinion of the Investigator, would place the subject at increased risk or would preclude obtaining voluntary consent or would confound the objectives of study
    17. Known hypersensitivity to study drug or any of the excipients of the drug formulation;
    18. Three or more of the following:
    a. BMI >35
    b. Current atrial fibrillation
    c. Current Diabetes Mellitus
    d. Current Hypertension
    e. History of clinically significant coronary artery disease in prior 3 years
    E.5 End points
    E.5.1Primary end point(s)
    Primary Efficacy Endpoint:
    Change from Baseline to end of assessment in Pulmonary Vascular Resistance (PVR)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Right Heart Catherization (RHC) performed between Screening and Baseline and between Visits 17 (End of Treatment) and 18, preferably 48 to 72 hours after the last dose.
    E.5.2Secondary end point(s)
    Secondary Efficacy Endpoints:
    1. Change from Baseline to end of assessment in:
    • Six Minute Walk Test (6MWT)
    • NT-proBNP
    • Cardiac index (CI), mean pulmonary artery pressure (mPAP), mean right atrial pressure (mRAP), pulmonary capillary wedge pressure (PCWP), mixed venous oxygen saturation (SvO2), including pulmonary artery compliance

    2. Safety Endpoints
    • Incidence and severity of AEs and their relationship to study drug
    • Vital signs, laboratory parameters, electrocardiograms (ECGs) and their relationship to PB1046
    • Immunogenicity

    3. Pharmacokinetic Endpoints:
    • Plasma PB1046 concentration at each time point and PK parameters
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. From Baseline (V2) to end of assessment (V18)
    2. From Baseline (V2) to the day of enrollment in the open-label extension (OLE) study PB1046-PT-CL-0006 (V18)
    or
    2. For those not opting to continue into the OLE, safety endpoints will continue to be collected for 28 days after the final dose of PB1046 (V18). Moreover, approximately 56 days after the final dose (V18) a blood sample will be also drawn for immunogenicity testing. If anti-drug antibodies are still present at 56 days post last dose, then subjects will be followed further until resolution of the antibody status.
    3. From Baseline to end of assessment
    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 Yes
    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 Yes
    E.6.13.1Other scope of the trial description
    immunogenicity
    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 Yes
    E.8.1.7.1Other trial design description
    dose titration
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    different treatment schedule of the same IMP
    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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA30
    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
    Austria
    Belgium
    Bulgaria
    Czech Republic
    Germany
    Greece
    Hungary
    Italy
    Poland
    Romania
    Serbia
    Spain
    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 - Visit 18 (Week 17)/End of Treatment Visit
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months7
    E.8.9.1In the Member State concerned days15
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days16
    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: 40
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 23
    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 state25
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 45
    F.4.2.2In the whole clinical trial 63
    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)
    Subjects can choose to enroll into an open label extension (OLE) study (protocol: PB1046-PT-CL-0006) after the end of treatment (EoT) visit. Subjects who do not enroll into the OLE, after the EoT will be monitored from safety point of view for 28 days after the final dose of PB1046. Moreover, approximately 56 days after the final dose, subjects will be also tested for immunogenicity. After that, they will be treated according to the available standard of care, as per treating physician decision.
    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 Decision2020-02-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-12-20
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2021-12-22
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