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    Summary
    EudraCT Number:2011-005679-18
    Sponsor's Protocol Code Number:GMX04
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2011-12-07
    Trial results Removed from public view
    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 number2011-005679-18
    A.3Full title of the trial
    A Phase IV, Multicenter, Open-Label Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases (PID) in Children and Adolescents
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A research study to determine if Gammaplex®, is safe, tolerable, and effective when given to children and adolescents who have Primary Immunodeficiency Diseases (PID)
    A.3.2Name or abbreviated title of the trial where available
    n/a
    A.4.1Sponsor's protocol code numberGMX04
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT00504075
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/121/2011
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorBio Products Laboratory Limited
    B.1.3.4CountryUnited Kingdom
    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 supportBio Products Laboratory Limited
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBio Products Laboratory Limited
    B.5.2Functional name of contact pointRegulatory Affairs
    B.5.3 Address:
    B.5.3.1Street AddressDagger Lane
    B.5.3.2Town/ cityElstree
    B.5.3.3Post codeWD6 3BX
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+4402089572213
    B.5.5Fax number+4402089572611
    B.5.6E-mailreg.affairs@bpl.co.uk
    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 Gammaplex
    D.2.1.1.2Name of the Marketing Authorisation holderBio Products Laboratory Ltd.
    D.2.1.2Country which granted the Marketing AuthorisationUnited Kingdom
    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 nameGammaplex
    D.3.2Product code n/a
    D.3.4Pharmaceutical form Solution for injection
    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 NORMAL IMMUNOGLOBULIN
    D.3.9.1CAS number n/a
    D.3.9.2Current sponsor coden/a
    D.3.9.3Other descriptive nameHUMAN NORMAL IMMUNOGLOBULIN
    D.3.9.4EV Substance CodeSUB14196MIG
    D.3.10 Strength
    D.3.10.1Concentration unit % (W/V) percent weight/volume
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    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
    Primary immunodeficiency diseases
    E.1.1.1Medical condition in easily understood language
    Primary immunodeficiency diseases are a mixed group of disorders causedby a defect in the tissues, cells, &/or proteins of the immune system; and leading to increased susceptibility to infections.
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.0
    E.1.2Level SOC
    E.1.2Classification code 10005329
    E.1.2Term Blood and lymphatic system disorders
    E.1.2System Organ Class 10005329 - Blood and lymphatic system disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To determine the efficacy of Gammaplex by measuring the number of serious acute bacterial infections during treatment with Gammaplex over a 12 month period.
    E.2.2Secondary objectives of the trial
    To assess the safety and tolerability of Gammaplex and to compare the data collected from adult subjects with PID from the GMX01 study.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. The subject is between the ages of or is equal to 2 and 16 years of age, of either sex, belonging to any ethnic group, and above a minimum weight of 10 kg. This weight is based on the amount of blood required for testing.
    2. The subject has a primary immunodeficiency disease, which has as a significant component of hypogammaglobulinemia and/or antibody deficiency (e.g. common variable immunodeficiency, X-linked and autosomal forms of agammaglobulinemia, hyper-IgM syndrome, Wiskott-Aldrich Syndrome). N.B. Isolated deficiency of a single IgG subclass, or of specific antibodies without hypogammaglobulinemia per se, does not qualify for inclusion.
    3. Subjects require the following before their first infusion of Gammaplex®:
    • Documented IGIV dose(s) and treatment intervals for the last 2 consecutive routine IGIV treatments. The previous doses should also meet the following conditions before study entry:
    • Have not changed by ± 50% of the mean dose for at least 3 months
    • Be between 300 and 800 mg/kg/infusion
    • Be given every 21-28 days, inclusive
    • Be a licensed or investigational product (Phase III or IIIb).
    • Documented previous IgG trough levels for the last 2 consecutive routine IGIV treatments:
    • Maintained at least 300 mg/dL above baseline serum IgG levels (defined as before initiation of any gamma globulin treatment for that subject)
    • Must be greater or equal to 600 mg/dL
    E.4Principal exclusion criteria
    1. Has not been treated with IGIV (treatment naive subject)
    2. The subject has a history of any severe anaphylactic reaction to blood or any blood-derived product.
    3. The subject is known to be intolerant to any component of Gammaplex, such as sorbitol (i.e. intolerance to fructose).
    4. The subject has selective IgA deficiency, history of reaction to products containing IgA, or has a history of antibodies to IgA.
    5. Subjects who have completed the study and subjects who have withdrawn cannot participate in the study for a second time.
    6. The subject is currently receiving, or has received, any investigational agent, other than an immune serum globulin (ISG) preparation that is being evaluated in a Phase III or IIIb study, within the prior 3 months.
    7. The subject has been exposed to blood or any blood product or derivative within the last 6 months, other than a commercially available IGIV or other forms of commercially available and licensed ISG. If an unlicensed ISG product that is in Phase III or IIIb has been given, the subject cannot be infused with Gammaplex until 20 days after the last dose was given.
    8. The subject is pregnant or is nursing.
    9. The subject, at screening, has levels greater than 2.5 times the upper limit of normal as defined at the central laboratory of any of the following: (Alanine transaminase (ALT); Aspartate transaminase (AST) Lactate dehydrogenase (LDH)).
    10. The subject has a severe renal impairment (defined as serum creatinine greater than 2 times the upper limit of normal or BUN greater than 2.5 times the upper limit of normal for the range of the laboratory doing the analysis); the subject is on dialysis; the subject has a history of acute renal failure.
    11. The subject is known to abuse alcohol, opiates, psychotropic agents, or other chemicals or drugs, or has done so within the past 12 months.
    12. The subject has a history of DVT, or thrombotic complications of IGIV therapy.
    13. The subject suffers from any acute or chronic medical condition (e.g. renal disease or predisposing conditions for renal disease, or protein losing state) that, in the opinion of the investigator, may interfere with the conduct of the study.
    14. The subject has an acquired medical condition, such as, chronic or recurrent neutropenia (ANC < 1000 x 109/L) or AIDS known to cause secondary immune deficiency, or is post or recovering from hematopoietic stem cell transplantation.
    15. The subject is receiving the following medication: Systemic long-term corticosteroids (i.e. not intermittent or burst, daily, >1 mg of prednisone equivalent/kg/day).
    16. The subject is receiving Immunosuppressive or Immunomodulatory drugs.
    17. The subject has non-controlled arterial hypertension.
    18. The subject has anemia (hemoglobin <10 g/dL) at screening.
    E.5 End points
    E.5.1Primary end point(s)
    The number of serious acute bacterial infections per subject per year, and will be based on the total of all of the following events as defined by the FDA: bacterial pneumonia, bacteremia or sepsis, osteomyelitis/septic arthritis, visceral abscess, bacterial meningitis
    E.5.1.1Timepoint(s) of evaluation of this end point
    At each infusion visit (every 21/28 days), the safety visit (following the first infusion) and at both follow-up visits (the 1st 10-14 days after the last infusion, and the second 3 months after the last infusion).
    E.5.2Secondary end point(s)
    • Number and proportion of subjects from Week 15 onwards who maintain trough IgG levels at least as high as the average of the 2 previous trough levels before the first Gammaplex infusion.
    • Number of days of school missed because of infection per subject year
    • Number and days of hospitalizations because of infection per subject year
    • Number of visits to physicians for acute problems and/or number of visits to hospital emergency rooms per subject year
    • Other infections documented by fever or a positive result on a radiograph and/or culture per subject year
    • Number of infectious episodes per subject per year
    • Number of days on therapeutic and prophylactic antibiotics
    Safety
    • Number of adverse events and adverse drug reactions (data collected via daily diary cards)
    • Significant changes in vital signs, safety laboratory parameters (including kidney and liver function), virology, physical examination and medical history during the study.
    he following PK variables for total IgG levels will be determined:
    PK
    • Cmax – peak concentration in plasma;
    • tmax – time to reach the peak concentration in plasma;
    • AUC(0-28) – area under the plasma concentration-time curve from time 0 (infusion start time) to Day 28;
    • t1/2 – terminal half-life;
    • Cl – systemic clearance;
    • Vd – volume of distribution;
    • Kelim – elimination rate constant;
    • MRT–mean residence time.
    Trough levels of IgG, IgG subclasses and three specific antibodies will be measured prior to certain infusions.
    Other Safety
    Number and percent of adverse events (AEs); vital signs; clinical laboratory tests and Direct Coombs’ Test; transmission of viruses; physical examination.

    E.5.2.1Timepoint(s) of evaluation of this end point
    At each infusion visit (every 21/28 days), the safety visit (following the first infusion) and at both follow-up visits (the 1st 10-14 days after the last infusion, and the second 3 months after the last infusion).
    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) 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 No
    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
    Chile
    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 years1
    E.8.9.2In all countries concerned by the trial months11
    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: 25
    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: 12
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 13
    F.1.2Adults (18-64 years) No
    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 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 No
    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
    Young children from 2 years upwards.
    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 25
    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 will receive 13 to 17 infusions of Gammaplex (i.e. 12 months on either a 21- or 28-day schedule with a 3 month follow-up). Efficacy, safety, PK & trough levels of Gammaplex will be assessed. The use of antipyretics, antihistamines, analgesics and anti-emetics before infusions is permitted, but corticosteroids are not allowed.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    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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