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    Summary
    EudraCT Number:2020-001496-32
    Sponsor's Protocol Code Number:KB070
    National Competent Authority:Slovakia - SIDC (Slovak)
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2020-07-02
    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 ConcernedSlovakia - SIDC (Slovak)
    A.2EudraCT number2020-001496-32
    A.3Full title of the trial
    A Phase III, Open-label, Prospective, Multicenter Study to Assess Efficacy, Safety, and Pharmacokinetics of Kedrion Intravenous Human Normal Immunoglobulin (IVIg) 10% in Pediatric Patients Affected by Primary Immunodeficiency Disease (PID)
    Otvorené, prospektívne, multicentrické klinické skúšanie fázy III na stanovenie účinnosti, bezpečnosti a farmakokinetiky Kedrion - normálny ľudský imunoglobulín na intravenózne použitie (IVIg) 10% u pediatrických pacientov s primárnou imunodeficienciou (PID).
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study, in which all know the treatment assigned to the patient, conducted in more sites, to evaluate the efficaty, safety, and metabolism of Kedrion in venous Human Normal Immunoglobulin (IVIg) 10% in child with Primary Immunodeficiency Disease (PID)
    A.3.2Name or abbreviated title of the trial where available
    PID-PED study
    PID-PED study
    A.4.1Sponsor's protocol code numberKB070
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/246/2017
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorKEDRION S.P.A
    B.1.3.4CountryItaly
    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 supportPharmaceutical company, KEDRION S.p.A
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCromsource S.r.l
    B.5.2Functional name of contact pointClinical Research Department
    B.5.3 Address:
    B.5.3.1Street AddressVia Giorgio De Sandre 3
    B.5.3.2Town/ cityVerona
    B.5.3.3Post code37135
    B.5.3.4CountryItaly
    B.5.4Telephone number39 0458222811
    B.5.5Fax number390458222812
    B.5.6E-mailoriana.zerbini@cromsource.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 nameKIg10
    D.3.2Product code KIg10
    D.3.4Pharmaceutical form Concentrate and solvent 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 Immunoglobulin
    D.3.9.2Current sponsor codeKIg10
    D.3.9.4EV Substance CodeSUB127300
    D.3.10 Strength
    D.3.10.1Concentration unit mg/kg milligram(s)/kilogram
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number200 to 800
    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 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 Yes
    D.3.11.13.1Other medicinal product typePlasma derivate
    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
    Pediatric Patients Affected by Primary Immunodeficiency Disease.
    E.1.1.1Medical condition in easily understood language
    Child with Immunodeficiency Disease.
    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 20.0
    E.1.2Level PT
    E.1.2Classification code 10064859
    E.1.2Term Primary immunodeficiency syndrome
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Primary Efficacy Objective
    To assess the efficacy of KIg10 administered to patients with PID to demonstrate that the rate of acute serious bacterial infections (i.e., the mean number of acute serious bacterial infections per patient-year) is less than 1.0 to provide substantial evidence of efficacy from day 1 to week 51/52.
    Safety Objective
    To assess the safety of KIg10 in the overall study population from day 1 to week 51/52.
    Pharmacokinetic Objectives
    To assess the distribution, metabolism, and elimination of KIg10, total IgG, IgG subclasses, and antigen-specific IgGs at steady state in PID patients treated at different dosing schedules.
    To evaluate trough concentrations of total IgG and compare to trough concentrations prior to the study entry.
    E.2.2Secondary objectives of the trial
    Levels:
    • Of IgG trough before each infusion and at the study termination visit.
    • Of IgG subclasses before infusions 1, 5, 9, and 13 for the 28-day infusion schedule
    and at infusions 1, 7, 11, and 17 for the 21-day infusion schedule.
    • Of Anti-tetanus toxoid, anti-pneumococcal capsular polysaccharide, anti Haemophilus influenza, and anti-measles antibodies before infusions 1, 5, 9, and 13 for the 28-day infusion schedule and before infusions 1, 7, 11, and 17 for the 21-day infusion schedule.
    Occurrence and duration of any infection other than acute serious bacterial infections, of fever episodes, of overall hospitalization, of hospitalization due to infection,
    of patients on antibiotics for the treatment of any kind of infections from day 1 to week 51/52.
    The missed days of work/school/other major activities due to infections, from day 1 to week 51/52.
    QoL using the PedsQL™ questionnaires collected at baseline, at 24 weeks, and at the study termination visit.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Written informed consent/assent obtained from the patient and his/her parent(s) or egally acceptable representative indicating that they understand the purpose of and procedures required for the study and are willing to participate in it.
    2. Confirmed clinical diagnosis of a PID as defined by 2017 International Union of Immunological Societies (IUIS) Phenotypic Classification for Primary Immunodeficiencies (Bousfiha A, 2018) and The European Society for Immunodeficiencies (ESID) Registry
    Working Definitions for the Clinical Diagnosis of Inborn Errors of Immunity (Seidel MG at al., 2019) and requiring treatment with IVIg. Documented agammaglobulinemia (defined as the total absence of one or more classes of antibodies) or hypogammaglobulinemia
    (defined as low levels of one or more classes [ie, at least 2 standard deviations under the mean level per age]). (NOTE: IVIg treatment is generally requested in the absence of IgG independently from whether other antibodies are absent).
    3. Male or female, age from 2 up to 16 years and 11 months, at the time of screening, as the patient must be under 18 years (< 18) at the time of study termination visit.
    4. Received 200 to 800 mg/kg of a commercially available IVIg therapy in the range of 1- or 28-day intervals (±3 or ±4 days, respectively) for at least 3 infusion cycles prior to screening. (NOTE: Other IVIgs will be prohibited after ICF signature and until study end, week 51/52).
    5. At least 2 documented IgG trough levels while receiving an IVIg, of = 6 g/L obtained at 2 infusion cycles within 12 months (1 must be within 6 months) prior to ICF ignature.
    6. Patient and his/her parent(s)/legal guardian(s) are willing to comply with all requirements of the protocol.
    7. Females of child-bearing potential with a negative pregnancy test (serum or urine) and who agree to employ adequate birth control measures during the study, such as
    a. sexual abstinence, to be evaluated in relation to the preferred and usual lifestyle of the subject;
    b. male or female condom with or without spermicide;
    c. cap, diaphragm or sponge with spermicide;
    d. progestogen-only oral hormonal contraception, if already used in the past on medical prescription
    The adequate birth control measures should be maintained throughout the study under parental control.
    8. Authorization to access personal health information.
    9. Patients previously participating in a clinical trial with another experimental IVIg may be enrolled if they have received stable commercially available IVIg therapy for at least 3 infusion cycles (21 or 28 days) prior to screening.
    10. Patients currently on treatment with any subcutaneous immunoglobulin (SCIG) can be nrolled if they are switched to stable commercially available IVIg therapy for at least 3 infusion cycles (21 or 28 days) prior to screening.
    11. Males or females with a body weight greater than or equal to 15 kg (≥15 kg).
    E.4Principal exclusion criteria
    Newly diagnosed PID and naïve to IgG replacement therapy.
    Dysgammaglobulinemia (defined as a deficiency in one or more classes of antibodies, but not severe enough to require substitutive therapy) or isolated IgG subclass deficiency, or profound primary T cell deficiency (defined as the absence or severe reduction of T
    lymphocytes [CD3+ <300 cell/mm3] and an absent or particularly low proliferative response [10% of the lower normal range] to phytohaemagglutinin P [PHA]).
    History of severe or serious reactions or hypersensitivity to IVIg or other injectable forms of IgG.
    History of thrombotic events including deep vein thrombosis, cerebrovascular accident, pulmonary embolism, transient ischemic attacks, or myocardial infarction, as defined by at least 1 event in patient’s lifetime.
    IgA deficiency with documented antibodies to IgA.
    Received blood products that have not undergone viral inactivation measures within 12 months prior to ICF signature.
    Significant protein losing enteropathy, nephrotic syndrome, or lymphangiectasia.
    An acute infection as documented by culture or diagnostic imaging and/or a body temperature =38.5° C (=101.3° F) within 7 days prior to screening.
    Acquired immunodeficiency syndrome (AIDS) and/or hepatitis B/C active disease at ICF signature.
    Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 2.5 times of the upper limit of normal for the laboratory designated for the study.
    Using an implanted venous access device.
    Profound anemia, defined according to the patient's age as shown in table Dallman PR, 1979 or persistent severe neutropenia (= 500 neutrophils per mm3) or persistent lymphopenia of less than 500 cells per microliter.
    A severe chronic condition such as renal failure, congestive heart failure (New York Heart Association III/IV), cardiomyopathy, cardiac arrhythmia associated with thromboembolic events (e.g., atrial fibrillation), unstable or advanced ischemic heart
    disease, hyperviscosity, or any other condition that the Investigator believes is likely to interfere with evaluation of the study drug or with satisfactory conduct of the trial.
    History of a malignant disease other than properly treated carcinoma in situ of the cervix or basal cell or squamous cell carcinoma of the skin within 24 months prior to ICF signature.
    History of pharmacoresistant epilepsy or multiple episodes of migraine (defined as at least 1 episode within 6 months of ICF signature) not controlled by medication.
    Patient must not be receiving the following medication from at least 30 days prior to ICF signature:
    Steroids, oral or parenteral, at a daily dose of = 0.15 mg/kg/day of prednisone or equivalent).
    Other immunosuppressive drugs (including monoclonal antibodies) or chemotherapy.
    Females who are pregnant, breast feeding or planning a pregnancy during the course of the study. Women who become pregnant during the study will be withdrawn from the study.
    Participated in another clinical study within 30 days prior to ICF signature.
    Active drug or alcohol abuse or history of drug or alcohol abuse within the 6 months before screening.
    Direct relative of an employee of the CRO, the study site, or Kedrion.
    Previously treated under this protocol.
    Unable to provide informed consent.
    Patients with any condition which, in the opinion of the Investigator, might interfere with the evaluation of the study objectives or the patient’s participation in this trial.
    Patients with Hypersensitivity to the active substance or to any of the excipients
    E.5 End points
    E.5.1Primary end point(s)
    Incidence rate (i.e., the mean number of acute serious bacterial infections per patientyear) of acute serious bacterial infections (bacterial pneumonia, bacteremia/sepsis, bacterial meningitis, visceral abscess, osteomyelitis/septic arthritis) according to
    pre-specified criteria). Primary analysis of the primary endpoint will be performed on the FAS. Primary efficacy will be analyzed using a Poisson model using as offset the length of the observation period per patient. The estimate and 1-sided 99% upper confidence limit
    will be reported. Efficacy will be claimed if this limit is less than 1.0.
    E.5.1.1Timepoint(s) of evaluation of this end point
    All visits
    E.5.2Secondary end point(s)
    1. Serum IgG trough levels before each infusion of KIg10 and at the study termination visit (week 51/52).
    2. IgG subclasses levels (IgG1, IgG2, IgG3, IgG4) before infusions 1, 5, 9 and 13 for the 28-day infusion schedule, and before infusions 1, 7, 11 and 17 for the 21-day infusion schedule.
    3. Frequency of patients with total IgG below 6 g/L criteria.
    4. Anti-tetanus toxoid antibody level (quantitative assay) before infusions 1, 5, 9 and 13 for the 28-day infusion schedule and before infusions 1, 7, 11 and 17 for the 21-day infusion schedule.
    5. Anti-pneumococcal capsular polysaccharide antibody level (quantitative assay) before infusions 1, 5, 9 and 13 for the 28-day infusion schedule and before infusions 1, 7, 11 and 17 for the 21-day infusion schedule.
    6. Anti-measles antibody level (quantitative assay) before infusions 1, 5, 9 and 13 for the 28-day infusion schedule and before infusions 1, 7, 11 and 17 for the 21-day infusion schedule.
    7. Anti-Haemophilus influenza type b antibody level (quantitative assay) before infusions 1, 5, 9 and 13 for the 28-day infusion schedule and before infusions 1, 7, 11 and 17 for the 21-day infusion schedule.
    8. Incidence rate (i.e., the mean number per patient-year) of any infection other than acute serious bacterial infections from day 1 to week 51/52.
    9. Duration of any infection other than acute serious bacterial infections from day 1 to week 51/52.
    10.Incidence rate (i.e. the mean number per patient-year) of fever episodes, from day 1 to week 51/52.
    11.Duration of fever episodes, from day 1 to week 51/52.
    12.Overall hospitalization days from day 1 to week 51/52.
    13.Days of hospitalizations due to infection from day 1 to week 51/52.
    14.Incidence rate (i.e. the mean number per patient-year) of patient on antibiotics
    for the treatment of any kind of infection from day 1 to week 51/52.
    15.Duration of patients on antibiotics for the treatment of any kind of infection from day 1 to week 51/52.
    16.Days of missed work/school/other major activities due to infections from day 1 to week 51/52.
    17.PedsQL™ score at baseline, week 24, and study termination visit.
    E.5.2.1Timepoint(s) of evaluation of this end point
    All visits
    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) 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.8.2.4Number of treatment arms in the trial1
    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 Yes
    E.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA12
    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
    Hungary
    Italy
    Portugal
    Russian Federation
    Slovakia
    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
    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 months10
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    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 Yes
    F.1.1Number of subjects for this age range: 30
    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: 15
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 15
    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 No
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 22
    F.4.2.2In the whole clinical trial 30
    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
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2020-10-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-07-27
    P. End of Trial
    P.End of Trial StatusOngoing
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