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    Summary
    EudraCT Number:2019-004063-49
    Sponsor's Protocol Code Number:NGAM-10
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2019-10-10
    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
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2019-004063-49
    A.3Full title of the trial
    Post-Marketing Study to Evaluate the Efficacy and Safety of PANZYGA in Pediatric Patients with Chronic Immune Thrombocytopenia (ITP)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to evaluate PANZYGA in pediatric patients with Chronic Immune Thrombocytopenia
    A.4.1Sponsor's protocol code numberNGAM-10
    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 SponsorOctapharma
    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 supportOctapharma
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationClinical Research Management Group
    B.5.2Functional name of contact pointMikaela Raymond
    B.5.3 Address:
    B.5.3.1Street Address1408 Main Street
    B.5.3.2Town/ cityAgawam
    B.5.3.3Post code01001
    B.5.3.4CountryUnited States
    B.5.6E-mailctgov@clinicalresearchmgt.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 Yes
    D.2.1.1.1Trade name PANZYGA
    D.2.1.2Country which granted the Marketing AuthorisationUnited States
    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 namePANZYGA
    D.3.4Pharmaceutical form Concentrate 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 INNPANZYGA
    D.3.9.3Other descriptive nameNORMAL IMMUNOGLOBULIN
    D.3.9.4EV Substance CodeSUB127253
    D.3.10 Strength
    D.3.10.1Concentration unit mg/kg milligram(s)/kilogram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1000
    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) Yes
    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
    Chronic Immune Thrombocytopenia
    E.1.1.1Medical condition in easily understood language
    Chronic Immune Thrombocytopenia
    E.1.1.2Therapeutic area Body processes [G] - Immune system processes [G12]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective is to evaluate the efficacy of PANZYGA in increasing the platelet count in pediatric patients with chronic ITP.
    E.2.2Secondary objectives of the trial
    • Assess safety of up to 2 g/kg PANZYGA (1 g/kg PANZYGA on Day 1 and an optional dose of 1 g/kg PANZYGA on Day 3) infused in the pediatric population within the scope of the clinical study
    • Determine the time to reach a platelet count of 50x109/L
    • Determine duration of time during which the platelet count is maintained at the level ≥50х109/L
    • Determine the maximum platelet count during the study
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Females and males aged from ≥1 year to <18 years old
    2. Confirmed diagnosis of Chronic Immune Thrombocytopenia (ITP) according to American Society of Hematology (ASH) 2011 guidelines
    3. Platelets count <30x109/L at the Baseline Visit
    4. Voluntarily given written informed consent (provided by patient’s parent or legal guardian) and assent (provided by patient [if age-appropriate per IRB requirements])
    5. Females of childbearing potential have been using at least 1 acceptable form of birth control for a minimum of 30 days (or a minimum of 3 months for hormonal contraceptives) prior to the Screening visit, and must agree to use at least 1 acceptable method of contraception for 30 days after the last dose of PANZYGA. Acceptable methods of birth control for this study include: intrauterine device (IUD), hormonal contraception, male or female condom, spermicide gel, diaphragm, sponge, or cervical cap. Abstinence is not considered an acceptable method of birth control.
    6. Parent or legal guardian must agree and be willing to assist the participant attend study visits, and to follow all protocol requirements and instructions of the study doctor
    E.4Principal exclusion criteria
    1. Thrombocytopenia secondary to other diseases (such as Acquired Immunodeficiency Syndrome [AIDS] or systemic lupus erythematosus [SLE]), drug-related thrombocytopenia, or congenital thrombocytopenia
    2. Administration of intravenous immunoglobulin (IGIV) or anti-D immunoglobulin within 3 weeks before enrollment
    3. Administration of thrombopoietin receptor agonists when the dose has NOT been stable within 3 weeks before enrollment and a dosage change is planned before Day 32
    4. Administration of oral immunosuppressants when the dose has NOT been stable during the preceding 2 months (2 weeks for long-term corticosteroid therapy) and a dosage change is planned before Day 32 (Note: topical agents and inhaled corticosteroid therapy use is permitted)
    5. Administration of long-term anti-prolific agents or attenuated androgen therapy when the dose has NOT been stable during the preceding 2 months and no dosage change is planned until Day 32
    6. Nonresponsive to previous treatment with IGIV or anti-D immunoglobulin
    7. Evidence of an active major bleeding episode at Screening
    8. Splenectomy in the previous 4 weeks or planned splenectomy throughout the study period
    9. Evans syndrome (experiencing active disease with 2 out of 3 of the following: autoimmune thrombocytopenia, autoimmune hemolytic anemia, and/or autoimmune neutropenia)
    10. Known or suspected human immunodeficiency virus (HIV), hepatitis B virus (HBV), and/or hepatitis C virus (HCV) infections
    11. Emergency surgery in the previous 4 weeks
    12. Severe liver and/or kidney disease (alanine aminotransferase [ALT] >3x upper limit of normal (ULN), aspartate aminotransferase [AST] >3x upper limit of normal (ULN), and/or creatinine >120 µmol/L)
    13. History of severe hypersensitivity to blood or plasma derived products, or any component of the PANZYGA
    14. Known immunoglobulin A (IgA) deficiency and antibodies against IgA
    15. History of, or suspected alcohol or drug abuse in the previous year
    16. Females who are pregnant or nursing
    17. Unable or unwilling to comply with the study protocol
    18. Receipt of any other investigational medicinal product within 3 months before study entry
    19. Risk factors* for thromboembolic events in whom the risks outweigh the potential benefit of PANZYGA treatment.
    20. Any other condition(s), that in the Investigator’s opinion, make it undesirable for the patient to participate in the study or may interfere with protocol compliance.
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy parameter is defined as an increase in platelet count at least once to ≥50x109/L within 7 days after the first infusion, ie, by Day 8 (increase must occur at least once on any day up to and including Day 8). This definition of clinical response will serve as the basis for the primary endpoint, the response rate (ie, the proportion of patients with an elevation of platelet count at least once to 50x109/L within 7 days [ie, by Day 8] after the first infusion).
    E.5.1.1Timepoint(s) of evaluation of this end point
    7 days
    E.5.2Secondary end point(s)
    The primary efficacy parameter is defined as an increase in platelet count at least once to ≥50x109/L within 7 days after the first infusion, ie, by Day 8 (increase must occur at least once on any day up to and including Day 8). This definition of clinical response will serve as the basis for the primary endpoint, the response rate (ie, the proportion of patients with an elevation of platelet count at least once to 50x109/L within 7 days [ie, by Day 8] after the first infusion).
    E.5.2.1Timepoint(s) of evaluation of this end point
    7 days
    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 No
    E.6.7Pharmacodynamic No
    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) No
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) Yes
    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 Will this trial be conducted at a single site globally? No
    E.8.4 Will this trial be conducted at multiple sites globally? Yes
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA Yes
    E.8.6.3Specify the countries outside of the EEA 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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years2
    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: 20
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 5
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 10
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 5
    F.1.2Adults (18-64 years) No
    F.1.2.1Number of subjects for this age range: 0
    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 No
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Parent or legal guardian
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 20
    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
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: United States
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