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    Summary
    EudraCT Number:2015-005490-20
    Sponsor's Protocol Code Number:2002C011G
    National Competent Authority:Norway - NOMA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2016-04-04
    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 ConcernedNorway - NOMA
    A.2EudraCT number2015-005490-20
    A.3Full title of the trial
    A Phase 2/3, Open-Label, Repeat-Dose Study of the Pharmacokinetics, Efficacy, and Safety of ProMetic Plasminogen Intravenous Infusion in Subjects with Hypoplasminogenemia
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical study, in which patients with Plasminogen deficiency are treated with injections of a Plasminogen solution (the study drug). The amount of the study drug in the blood stream will be measured during the study, and the tolerability and the efficacy of the study drug will be tested.
    A.4.1Sponsor's protocol code number2002C011G
    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 SponsorProMetic BioTherapeutics 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 supportProMetic BioTherapeutics Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationKLIFO A/S
    B.5.2Functional name of contact pointVice President, Clinical Trial Serv
    B.5.3 Address:
    B.5.3.1Street AddressSmedeland 36
    B.5.3.2Town/ cityGlostrup
    B.5.3.3Post codeDK-2600
    B.5.3.4CountryDenmark
    B.5.4Telephone number+4544 222 921
    B.5.5Fax number+4539 209 045
    B.5.6E-mailjoris.wilms@klifo.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/3/15/1511
    D.3 Description of the IMP
    D.3.1Product namePlasminogen (Human) ProMetic
    D.3.4Pharmaceutical form Powder for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNHuman Plasminogen
    D.3.9.3Other descriptive namePLASMINOGEN (HUMAN) INTRAVENOUS LYOPHILIZED
    D.3.9.4EV Substance CodeSUB179214
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5.0
    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 Yes
    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
    Hypoplasminogenemia
    E.1.1.1Medical condition in easily understood language
    Low plasminogen levels
    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 18.1
    E.1.2Level PT
    E.1.2Classification code 10035493
    E.1.2Term Plasminogen decreased
    E.1.2System Organ Class 10022891 - Investigations
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To achieve an increase of individual trough plasminogen activity by at least an absolute 10% (i.e., 10 U/dL) from baseline in adult and pediatric subjects with hypoplasminogenemia during the 12 weeks of plasminogen replacement therapy in Segment 2.
    E.2.2Secondary objectives of the trial
    To evaluate the safety and tolerability of plasminogen replacement therapy during the 12 weeks of dosing in Segment 2.
    To evaluate the efficacy of plasminogen replacement therapy on clinically evident or visible symptoms of hypoplasminogenemia during the 12 weeks of dosing in Segment 2.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject or legal guardian has provided informed consent (as well as assent by subjects with ages dictated by local Investigational Review Board (IRB) guidelines).
    2. Subject is male or female between the ages of 2 and 80 years (inclusive).
    3. Subject has a documented history of lesions and symptoms consistent with a diagnosis of hypoplasminogenemia.
    4. Subject has plasminogen levels =< 45%.
    5. Subject has documented vaccination to hepatitis A virus (HAV) and hepatitis B virus (HBV), or has received the first dose of HAV and HBV vaccine prior to the first dose of IMP and is scheduled to receive the second vaccine dose. If subject has documented vaccination more than 1 year before screening but has a negative antibody titer to HAV and/or HBV at screening, subject is required to begin a re-vaccination series with the first dose of HAV and/or HBV vaccine prior to the first dose of IMP and is scheduled to receive the second vaccine dose. No re-vaccination is required, if the documented vaccination took place within 1 year of screening.
    6. Subject agrees to use contraceptive methods from screening through 14 days after last dose of study treatment (unless documented as biologically or surgically sterile (e.g. postmenopausal, vasectomized), or has not reached reproductive age.
    E.4Principal exclusion criteria
    1. Subject has a history of anaphylactic reactions to blood or blood products that may interfere with participation in the study in the opinion of the investigator.
    2. Subject has uncontrolled hypertension.
    3. Subject has clinical or laboratory evidence of an intercurrent infection as evidenced by symptoms including fever, tachycardia, or other systemic signs and symptoms. (Note: Subjects with an intercurrent clinically significant infection cannot participate; however, once the infection has resolved according to the investigator, they can be re-screened if enrollment is still open.)
    4. Subject is pregnant and/or lactating.
    5. Subject has a malignancy, except for basal or squamous cell skin cancer, within 3 years before screening.
    6. Subject is a previous organ transplant recipient.
    7. Subject is in receipt of exogenous plasminogen (ocular or IV), such as laboratory grade plasminogen, fresh frozen plasma, or ProMetic Plasminogen (Human) within 2 weeks of the first dose of the IMP.
    8. Subject has a psychiatric disorder, other mental disorder, or any other medical disorder that impairs the subject's ability to provide informed consent or to comply with the requirements of the study protocol.
    9. Subject has evidence of renal dysfunction defined as of > 2 x the upper limit of normal (ULN) in serum creatinine.
    10. Subject has evidence of hepatic dysfunction defined as > 3 x ULN in alanine aminotransferase (ALT), aspartate aminotransferase (AST), or alkaline phosphatase (ALP).
    11. Subject has participated in another IRB-approved interventional clinical trial of drug, biologic, or device within 30 days before the first dose of the IMP.
    12. Subject has a chronic or acute clinically significant intercurrent illness (e.g., cardiac, hepatic, renal, endocrine, neurologic, hematologic, neoplastic, immunological, and skeletal) that the investigator determines could interfere with the assessments in this study.


    E.5 End points
    E.5.1Primary end point(s)
    The number and percentage of subjects who achieve the target plasminogen activity trough levels for at least 3 measurements by the 12 week time-point during Segment 2.
    The target trough level is defined as an increase in plasminogen activity level of at least an absolute 10% (10 U/dL) from the subject's individual baseline level.
    Baseline level is defined as the plasminogen activity level measured before Segment 1 dosing (or Segment 2, Dose #1, if Segment 1 is not required).
    E.5.1.1Timepoint(s) of evaluation of this end point
    After 12 weeks treatment during Segment 2
    E.5.2Secondary end point(s)
    For analysis of safety and tolerability, TEAEs and SAEs will be summarized descriptively. Clinical laboratory tests (hematology, biochemistry, urinalysis, fibrinolysis/coagulation) will be presented in summary and shift tables. The numbers of subjects who had changes from baseline in viral tests and immunogenicity tests will be presented in individual subject listings and summary tables.

    The secondary efficacy endpoints are:
    - Number and size of lesions assessed at each study site visit.
    - Evidence of improvement in affected organ functionality assessed at each study visit, if appropriate e.g. FEV1, FVC, FEV1/FVC ratio for subjects with bronchial involvement.
    - Patient assessments of general overall health status (Quality of Life - 10-point scale) at each study visit.
    - Physician global clinical impression at each study visit (7-point scale).
    Efficacy endpoints are considered achieved if 30% of subjects (i.e. 4 or more) demonstrate at least a 50% improvement in lesion number/size or functionality impact from baseline.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Safety and tolerability:
    At each study visit during dosing in Segment 1 (Day -3) and Segment 2 (during 12 weeks) and at study discontinuation, which is 30 days after the final dose of IMP.

    Efficacy:
    At each study visit during 12 weeks treatment in Segment 2 and at study discontinuation.
    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 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) 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) Yes
    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 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
    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
    30 days after LVLS
    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 months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months6
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 2
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 1
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 1
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 10
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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 state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 3
    F.4.2.2In the whole clinical trial 12
    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)
    At the end of Segment 2, subjects who wish to continue therapy will have the option to participate in Segment 3. They will continue to receive plasminogen replacement therapy, until product licensing or study termination by sponsor. Subjects will continue on the dosing frequency established in Segment 2, unless modification is necessary based on trough levels and/or individual subject's response. Subjects will return to the study sites for assessments every 3 months.
    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 Decision2016-06-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-06-29
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-10-08
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