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    Summary
    EudraCT Number:2009-017805-13
    Sponsor's Protocol Code Number:NGAM-03
    National Competent Authority:Bulgarian Drug Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2010-12-13
    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 ConcernedBulgarian Drug Agency
    A.2EudraCT number2009-017805-13
    A.3Full title of the trial
    "PROSPECTIVE, PARALLEL GROUP, DOUBLE-BLIND, PLACEBO-CONTROLLED, RANDOMISED, MULTICENTRE, ADAPTIVE, TWO-STAGE PHASE II/III STUDY EVALUATING SAFETY AND EFFICACY OF THREE DIFFERENT DOSAGES OF NEWGAM IN PATIENTS WITH CHRONIC INFLAMMATORY DEMYELINATING POLYRADICULONEUROPATHY (POINT TRIAL)”
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to evaluate efficacy of human immune globulin in patients with CIDP-chronic neuropathy.
    A.3.2Name or abbreviated title of the trial where available
    NGAM-03
    A.4.1Sponsor's protocol code numberNGAM-03
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorOctapharma AG
    B.1.3.4CountrySwitzerland
    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 AG
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationOctapharma Pharmazeutika Produktionsgesellschaft mbH
    B.5.2Functional name of contact pointClinical Research Department
    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+43 1 61032 1716
    B.5.5Fax number+43 1 61032 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 nameNewGam
    D.3.2Product code NewGam
    D.3.4Pharmaceutical form 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 INNImmunoglobuline G
    D.3.9.3Other descriptive nameNewGam
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number86 to 110
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for injection
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)
    E.1.1.1Medical condition in easily understood language
    Acquired immune-mediated inflammatory disorder of the peripheral nerves (affected nerves fail to respond to stimuli). The disorder is causing numbing, tingling, pain and progressive muscle weakness.
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.0
    E.1.2Level PT
    E.1.2Classification code 10057645
    E.1.2Term Chronic inflammatory demyelinating polyradiculoneuropathy
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Stage 1 (Phase 2 dose-finding part):
    • Determination and selection (after a loading dose of 2.0 g/kg NewGam) of one dosage from three NewGam maintenance dosage arms (0.4 g/kg or 1.0 g/kg or 2.0 g/kg given every 21 [±4] days starting at Week 3) in comparison with a placebo arm, based on the percentage of responders (response defined as a decrease, meaning improvement, in the adjusted INCAT disability score by at least 1 point) at Week 24. The selected NewGam dosage and placebo will be employed and compared in Stage 2.

    Stage 2 (Phase 3 confirmatory part):
    • To demonstrate superiority (after a loading dose of 2.0 g/kg NewGam) of the maintenance dosage regimen selected at study Stage 1 (out of 0.4 g/kg, 1.0 g/kg or 2.0 g/kg NewGam) over placebo in patients with CIDP as assessed by the percentage of responders (response defined as a decrease, meaning improvement, in the adjusted INCAT disability score by at least 1 point) at Week 24.
    E.2.2Secondary objectives of the trial
    To evaluate the safety and efficacy of NewGam administration in patients with
    CIDP through other parameters (see Section 3.1.2) at Week 24 (or time of rescue infusion) compared to baseline.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    •Patients diagnosed as having CIDP by a neurologist experienced in neuromuscular diseases based on fulfilment of clinical criteria of the INCAT Group and the definite electrophysiological criteria for CIDP of the European Federation of Neurological Societies/Peripheral Nerve Society (EFNS/PNS); patients with Multifocal Acquired Demyelinating Sensory And Motor neuropathy (MADSAM) or pure motor CIDP will be included provided they fulfil these criteria
    • Worsening of disability and objective increase in weakness or sensory deficit during the 6 months prior to screening
    • ≥ 18 years of age
    • Overall adjusted INCAT disability score between 2-9 (see Appendix 1); a score of 2 has to result exclusively from leg disability
    • Expanded Medical Research Council (MRC) sum score of ≤ 74 (see Appendix 2)
    • Freely given written informed consent from patient
    • Willingness to comply with all aspects of the protocol, including blood sampling, for the duration of the study
    • Women of reproductive age: negative result of pregnancy test (human chorionic gonadotropin [HCG]-based assay in urine) and surgically sterile, sexually inactive, or willing to use adequate contraception for the duration of the trial.
    E.4Principal exclusion criteria
    •Unifocal forms of CIDP
    •Pure sensory CIDP
    •Multifocal motor neuropathy (MMN) with conduction block, defined as a lower motor neuron disorder with motor weakness in an upper limb, without sensory deficit and with proximal conduction block (50% decrease in amplitude/area with proximal compared with distal stimulation) in motor nerves and normal sensory nerve conduction studies (NCS)
    •Treatment of CIDP with immunoglobulins (intravenous or subcutaneous) at any time prior to study entry
    •Steroids of any type equivalent to prednisolone or prednisone > 10 mg/day or equivalent, e.g., > 20 mg every 2 days or plasma exchange (PE) during the last 3 months prior to baseline visit
    •Treatment with azathioprine during 12 months prior to baseline visit unless on a stable dose throughout at least 12 months prior to baseline. Patients who are on a stable dose for at least 12 months prior to baseline should be expected to continue taking the same dose during the trial.
    •Treatment with cyclosporin, methotrexate, mitoxantrone, mycophenolate mofetil, interferon or other immunosuppressive or immunomodulatory drugs during the three months prior to baseline visit
    •Patients ever treated with rituximab, alemtuzumab, cyclophosphamide, or other intensive chemotherapeutic regimens, previous lymphoid irradiation or stem cell transplantation
    •Respiratory impairment requiring mechanical ventilation
    •Myelopathy or clinical evidence of central nervous system (CNS) demyelination, trauma or stroke
    •Clinical evidence of peripheral neuropathy from another cause such as
    -connective tissue disease or systemic lupus erythematosus,
    -HIV infection, hepatitis, Lyme disease,
    -cancer (with the exception of benign skin cancer), lymphoma, malignant plasma cell dyscrasia, Castleman’s disease
    -IgM paraproteinaemia with anti-myelin associated glycoprotein antibodies
    •Known diabetes mellitus
    •Other serious medical condition complicating assessment or treatment including but not limited to
    ocardiac insufficiency (New York Heart Association [NYHA] III/IV), known cardiomyopathy, significant cardiac dysrhythmia requiring treatment, unstable or advanced ischemic heart disease,
    osevere liver disease (ALAT > 3x upper limit of normal for the testing laboratory),
    osevere kidney disease (creatinine > 120 µM),
    ohepatitis B or C or HIV infection
    •Thromboembolic events: patients with a history of deep vein thrombosis (DVT) within the last year prior to baseline visit or pulmonary embolism ever; patients with susceptibility to embolism or DVT
    •Patients with known uncompensated hypothyroidism (abnormally high Thyroid-Stimulating Hormone (TSH), and abnormally low thyroxine (T4)) or known vitamin B12 deficiency
    •Medical conditions whose symptoms and effects could alter protein catabolism and/or IgG utilisation (e.g. protein-losing enteropathies, nephrotic syndrome)
    •Known IgA deficiency with antibodies to IgA
    •History of hypersensitivity, anaphylaxis or severe systemic response to immunoglobulin, blood or plasma derived products, or any component of NewGam
    •Known blood hyperviscosity, or other hypercoagulable states
    •Operation within 3 months prior to baseline visit in which blood products have been used
    •Patients with a past or present history of drug or alcohol abuse
    •Patients unable or unwilling to understand or comply with the study protocol
    •Patients with any condition that would make the patient, in the opinion of the Investigator, unsuitable for the study
    •Live viral vaccination within the last month prior to baseline visit
    •Participation in another interventional clinical study during the 3 months prior to baseline visit
    •Pregnant or nursing women.
    E.5 End points
    E.5.1Primary end point(s)
    Efficacy:
    Response at 24 weeks or at time of rescue NewGam infusion using the adjusted INCAT disability score. Response is defined as a decrease relative to baseline of at least 1 point on the adjusted INCAT disability score, a scale from 0 to 10 (from healthy to unable to make any purposeful movements with arms or legs in which the change from 0 to 1 in the upper limb component is discounted, see Appendix 1)
    The results from the Stage 1 (dose-finding phase II part) of this seamless phase II/III trial will be used to adopt the sample size in the Stage 2 (efficacy phase III part), and also to only continue - in addition to the placebo arm - with the NewGam arm that is the most efficacious as shown by IA.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Primary end point Efficacy: Response at Week 24 or at time of rescue NewGam infusion
    E.5.2Secondary end point(s)
    Efficacy:
    • Mean change from baseline at Week 24 or at time of rescue NewGam infusion in :
    - grip strength of dominant hand or if ambidextrous right hand
    - adjusted INCAT disability score
    - the sum of the distal-evoked amplitude of 4 right sided motor nerves
    - expanded MRC sum score
    • Time to 1 point increase in adjusted INCAT disability score to or above baseline value after temporary 1 or more point decrease

    Safety:
    - Occurrence of all AEs throughout entire 48-week trial period starting with the first infusion
    - Short term tolerance parameters including vital signs throughout entire 48-week trial period
    - Standard ECG (at screening and termination visit)
    - Physical/neurological examination at 12-week intervals throughout entire 48-week trial period
    - Laboratory parameters throughout entire 48-week trial period (except baseline and Week 15, 21, 30 and 42 in Stage 1)
    - Tests for viral safety (at screening and termination visit)
    E.5.2.1Timepoint(s) of evaluation of this end point
    A. Endpoint Efficacy :Mean change from baseline at Week 24 or at time of rescue NewGam infusion
    B.Endpoints safety: Occurrence of all AEs throughout entire 48-weeks (36-weeks in Stage 2); laboratory parameters throughout entire 48-weeks (36-weeks in Stage 2), neurological examination throughout entire 48-weeks (36-weeks in Stage 2).
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    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 Yes
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    three dosage forms
    E.8.2.4Number of treatment arms in the trial4
    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 EEA18
    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
    Bulgaria
    Czech Republic
    India
    Mexico
    Poland
    Romania
    Serbia
    Ukraine
    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
    The end of the study is defined as the last visit of the last patient undergoing the study.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months3
    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 No
    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) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 150
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 25
    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 state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 100
    F.4.2.2In the whole clinical trial 172
    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)
    Patients will continue with the expected normal treatment of CIDP.
    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 Decision2011-01-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-01-27
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2011-11-07
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