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    Summary
    EudraCT Number:2013-003877-87
    Sponsor's Protocol Code Number:SCGAM-01
    National Competent Authority:Slovakia - SIDC (Slovak)
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2016-03-17
    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 ConcernedSlovakia - SIDC (Slovak)
    A.2EudraCT number2013-003877-87
    A.3Full title of the trial
    CLINICAL PHASE III STUDY TO EVALUATE THE PHARMACOKINETICS, EFFICACY, TOLERABILITY AND SAFETY OF SUBCUTANEOUS HUMAN IMMUNOGLOBULIN
    (OCTANORM 16.5%) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES
    Klinické skúšanie fázy III na zhodnotenie farmakokinetiky, účinnosti, znášanlivosti a bezpečnosti subkutánneho ľudského imunoglobulínu (octanorm 16,5%) u pacientov s primárnou imunodeficienciou.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Clinical trial to confirm and expand information on absorption, distribution, elimination in the body, efficacy, tolerability and safety of a new drug in patients with impaired immune system.
    A.4.1Sponsor's protocol code numberSCGAM-01
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01888484
    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 Pharmazeutika Prod.Ges.m.b.H
    B.1.3.4CountryAustria
    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 Pharmazeutika Prod.Ges.m.b.H
    B.4.2CountryAustria
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationOctapharma Pharmazeutika Prod.Ges.m.b.H
    B.5.2Functional name of contact pointDeputy International Med Director
    B.5.3 Address:
    B.5.3.1Street AddressOberlaaer Strasse 235
    B.5.3.2Town/ cityVienna
    B.5.3.3Post codeA-1100
    B.5.3.4CountryAustria
    B.5.4Telephone number+431610321798
    B.5.5Fax number+43161032 9249
    B.5.6E-maileva.turpel-kantor@Octapharma.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 nameOctanorm 16.5%
    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 INNHuman Normal Immunoglobulin
    D.3.9.1CAS number 308067-58-5
    D.3.9.2Current sponsor codeOctanorm 16.5%
    D.3.9.3Other descriptive nameHUMAN NORMAL IMMUNOGLOBULIN
    D.3.9.4EV Substance CodeSUB14196MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/kg milligram(s)/kilogram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number16.5
    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 Yes
    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
    E.1.1.1Medical condition in easily understood language
    Person with impaired immune system
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    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 first primary objective of the study is to assess the efficacy of octanorm in preventing serious bacterial infections compared with historical control data.
    The second primary objective is to evaluate the pharmacokinetic characteristics of octanorm and to compare the area under the curve with that of IVIG.
    E.2.2Secondary objectives of the trial
    The secondary objectives of the study are:
    • To evaluate the tolerability and safety of octanorm.
    • To determine the PK profile of octanorm.
    • To assess the dosing conversion factor when switching patients from intravenous immunoglobulin treatment.
    • To develop guidance and recommendations to support further adjustments of octanorm dosing based on the total IgG trough level.
    • To assess the effect of octanorm on Quality of Life measures.
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Secondary pharmacokinetic endpoints:
    • PK profiles of total IgG, of IgG subclasses (IgG1, IgG2, IgG3, IgG4), and of antigenspecific antibodies against Haemophilus influenzae, Streptococcus pneumoniae (types 4, 6B, 9V, 14, 18C, 19F, 23F), cytomegalovirus (CMV), tetanus, and measles.
    • Trough levels of serum total IgG (total and subclasses) throughout the study.
    • Trough levels of specific antibodies against Haemophilus influenzae, Streptococcus pneumoniae (types 4, 6B, 9V, 14, 18C, 19F, 23F), CMV, tetanus, and measles throughout the study.
    E.3Principal inclusion criteria
    1. Age of ≥2 years and ≤75 years.
    2. Confirmed diagnosis of PI as defined by ESID and PAGID and requiring immunoglobulin replacement therapy due to hypogammaglobulinaemia or agammaglobulinaemia. The exact type of PI should be recorded.
    3. Patients with at least 6 infusions on regular treatment with any IVIG, thereof a minimum of the last 2 months on the same product prior to entering the study. Constant IVIG dose between 200 and 800 mg/kg body weight (±20% of the mean dose for the last 6 infusions).
    4. Availability of the IgG trough levels of 2 previous IVIG infusions before enrolment, and maintenance of ≥5.0 g/L in the trough levels of these 2 previous infusions.
    5. Negative result on a pregnancy test (HCG-based assay in urine) for women of childbearing potential and use of a reliable method of contraception for the duration of the study.
    6. For adult patients: freely given written informed consent. For minor patients: freely given written informed consent from parents/legal guardians and written informed assent from the child/adolescent in accordance with the applicable regulatory requirements.
    7. Willingness to comply with all aspects of the protocol, including blood sampling, for the duration of the study.
    E.4Principal exclusion criteria
    1.Acute infection requiring intravenous antibiotic treatment within 2 weeks prior to and during the screening period.
    2. Known history of adverse reactions to IgA in other products.
    3. Patients with body mass index ≥40 kg/m2.
    4. Exposure to blood or any blood product or plasma derivatives, other than IVIG treatment for PID within the past 3 months prior to first infusion of octanorm.
    5. Ongoing history of hypersensitivity or persistent reactions to blood or plasma derived products, or any component of the investigational product (such as polysorbate 80).
    6. Requirement of any routine premedication for IgG administration.
    7. History of malignancies of lymphoid cells and immunodeficiency with lymphoma.
    8. Severe liver function impairment (ALAT 3 times above upper limit of normal).
    9. Known protein-losing enteropathies or proteinuria.
    10. Presence of renal function impairment (creatinine >120 μM/L or creatinine >1,35 mg/dL), or predisposition for acute renal failure (e.g., any degree of pre-existing renal insufficiency or routine treatment with known nephritic drugs).
    11. Treatment with oral or parenteral steroids for ≥30 days or when given intermittently or as bolus at daily doses ≥0.15 mg/kg.
    12. Treatment with immunosuppressive or immunomodulatory drugs.
    13. Live viral vaccination (such as measles, rubella, mumps and varicella) within the last 2 months prior to first infusion of octanorm.
    14. Treatment with any investigational medicinal product within 3 months prior to first infusion of octanorm.
    15. Presence of any condition, that is likely to interfere with the evaluation of study medication or satisfactory conduct of the trial.
    16. Known or suspected to abuse alcohol, drugs, psychotropic agents or other chemicals within the past 12 months prior to first infusion of octanorm.
    17. Known or suspected HIV, HCV, or HBV infection.
    18. Pregnant or nursing women.
    19. Planned pregnancy during course of the study.
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint is the rate of SBI (defined as bacteremia/sepsis, bacterial meningitis, osteomyelitis/septic arthritis, bacterial pneumonia, and visceral abscess) per person-year on treatment.

    The primary endpoint with respect to the PK investigations is the AUC from time 0 (start of the infusion) to the end of the nominal dosing period, standardized to 1 week (AUCτ), at steady-state conditions.
    E.5.1.1Timepoint(s) of evaluation of this end point
    End of the study
    E.5.2Secondary end point(s)
    Secondary efficacy endpoints:
    • The annual rate of all infections of any kind or seriousness.
    • Non-serious infections (total and by category).
    • Time to resolution of infections.
    • Use of antibiotics (number of days and annual rate).
    • Hospitalizations due to infection (number of days and annual rate).
    • Episodes of fever.
    • Days missed from work/school/kindergarten/day care due to infections and their treatment.
    • QoL assessments using the CHQ-PF50 from parent or guardian of patients <14 years of age and the SF-36 Health Survey in patients ≥14 years of age.

    Secondary pharmacokinetic (PK) endpoints:
    • PK profiles of total IgG, of IgG subclasses (IgG1, IgG2, IgG3, IgG4), and of antigen-specific antibodies against Haemophilus influenzae, Streptococcus pneumoniae (types 4, 6B, 9V, 14, 18C, 19F, 23F), cytomegalovirus (CMV), tetanus, and measles.
    • Trough levels of serum total IgG (total and subclasses) throughout the study.
    • Trough levels of specific antibodies against Haemophilus influenzae, Streptococcus pneumoniae (types 4, 6B, 9V, 14, 18C, 19F, 23F), CMV, tetanus, and measles throughout
    • IVIG to octanorm DCF (based on the area under the concentration-time curve [AUCτ]).

    Secondary safety endpoints:
    • Occurrence of all treatment emergent adverse events (TEAEs) throughout the entire 65-week treatment period starting with the first infusion of octanorm.
    • Occurrence of temporally associated TEAEs.
    • Proportion of infusions with at least 1 temporally associated adverse event (AE).
    • Occurrence of suspected adverse reactions (SARs).
    • TEAEs by speed of infusion.
    • Local injection site reactions.
    • Vital signs (blood pressure, pulse, body temperature, respiratory rate).
    • Laboratory parameters (hematology, clinical chemistry, markers for intravascular hemolysis, and tests for viral safety).
    E.5.2.1Timepoint(s) of evaluation of this end point
    End of the study
    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 Yes
    E.6.13.1Other scope of the trial description
    Tolerability
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Yes
    E.7.1.1First administration to humans Yes
    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.8.3 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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA15
    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
    Canada
    Czech Republic
    Hungary
    Poland
    Russian Federation
    Slovakia
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    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 years3
    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: 36
    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: 16
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 20
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 20
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 8
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Younger children
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state14
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 26
    F.4.2.2In the whole clinical trial 64
    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)
    After end of the study participation, subjects will be treated at the discretion of their physician, with other most suitable standard therapies and medical care, currently local available for his/her condition
    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 Decision2017-01-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-05-11
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2020-06-09
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