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    Summary
    EudraCT Number:2015-005443-14
    Sponsor's Protocol Code Number:NGAM-08
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2017-04-05
    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 ConcernedGermany - PEI
    A.2EudraCT number2015-005443-14
    A.3Full title of the trial
    Prospective, double-blind, randomized, multicenter phase III study evaluating efficacy and safety of three different dosages of NewGam in patients with chronic inflammatory demyelinating poly(radiculo)neuropathy.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Prospective, double-blind, randomized, multicenter phase III study evaluating efficacy and safety of three different dosages of NewGam in patients with chronic inflammatory neuropathy.
    A.4.1Sponsor's protocol code numberNGAM-08
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02638207
    A.5.4Other Identifiers
    Name:INDNumber:14096
    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 Produktionsges.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 Produktionsges.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 Produktionsges.m.b.H.
    B.5.2Functional name of contact pointGlobal Clinical Project Manager
    B.5.3 Address:
    B.5.3.1Street AddressOberlaaer Str. 235
    B.5.3.2Town/ cityVienna
    B.5.3.3Post code1100
    B.5.3.4CountryAustria
    B.5.4Telephone number43610321716
    B.5.5Fax number43610329249
    B.5.6E-mailclinical.department@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 Yes
    D.2.1.1.1Trade name PANZYGA
    D.2.1.1.2Name of the Marketing Authorisation holderOctapharma G.m.b.H.
    D.2.1.2Country which granted the Marketing AuthorisationGermany
    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.3.4Pharmaceutical 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 INNImmunoglobulin G
    D.3.9.1CAS number 308067-58-5
    D.3.9.3Other descriptive nameIMMUNOGLOBULIN G
    D.3.9.4EV Substance CodeSUB20618
    D.3.10 Strength
    D.3.10.1Concentration unit % percent
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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 infusion
    D.8.4Route of administration of the placeboIntravenous drip use (Noncurrent)
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Chronic Inflammatory Demyelinating Poly(radiculo)-neuropathy (CIDP)
    E.1.1.1Medical condition in easily understood language
    Chronic Inflammatory
    neuropathy.
    E.1.1.2Therapeutic area Body processes [G] - Immune system processes [G12]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    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 Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Primary:
    •To provide confirmatory data on the effect of 1.0 g/kg NewGam every three weeks in patients with active CIDP based on the percentage of responders at Week 24, which should corroborate the existing evidence on efficacy of IGIV in CIDP as known from published literature.
    E.2.2Secondary objectives of the trial
    Secondary:
    •To assess the effect of 0.5 g/kg and 2.0 g/kg NewGam every three weeks in patients with active CIDP based on the percentage of responders at Week 24 compared to patients on 1.0 g/kg NewGam every three weeks
    •To evaluate the safety of NewGam administration using various dosages in patients with CIDP
    •To further evaluate the beneficial effect of three NewGam dosages in patients with CIDP by assessing different parameters/scores/scales
    Exploratory:
    •To assess the primary and secondary objectives at three weeks after having provided rescue medication (if applicable)
    •To further evaluate the beneficial effect of NewGam administration in patients with CIDP by additional assessments/scores including quality of life (QoL) measures
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Patients with diagnosis of definite or probable CIDP according to the EFNS/PNS Guideline 2010; including patients with Multifocal Acquired Demyelinating Sensory And Motor Neuropathy (MADSAM) or pure motor CIDP
    2.Patients currently depending on treatment with immunoglobulins or corticosteroids
    3.Patients with active disease, i.e. not being in remission, who are progressive or relapsing prior to trial start or during the Wash-out Phase
    4.Weakness of at least 2 limbs
    5.≥ 18 to < 80 years of age
    6.Adjusted INCAT disability score between 2 and 9 (with a score of 2 coming exclusively from leg disability)
    7.Voluntarily given, fully informed written consent obtained from patient before any study-related procedures are conducted
    E.4Principal exclusion criteria
    1.Unifocal forms of CIDP
    2.Pure sensory CIDP
    3.MMN with conduction block
    4.Patients who previously failed immunoglobulin therapy
    5.Treatment with immunomodulatory/suppressive agents (cyclosporin, methotrexate, mitoxantrone, mycophenolate mofetil or azathioprine) during the six months prior to baseline visit
    6.Patients on or treated with rituximab, alemtuzumab, cyclophosphamide, or other intensive chemotherapeutic regimens, previous lymphoid irradiation or stem cell transplantation during the 12 months prior to baseline visit
    7.Respiratory impairment requiring mechanical ventilation
    8.Myelopathy or evidence of central nervous system demyelination or significant persisting neurological deficits from stroke, or central nervous system (CNS) trauma
    9.Clinical evidence of peripheral neuropathy from another cause such as:
    a.connective tissue disease or systemic lupus erythematosus (SLE)
    b.HIV infection, hepatitis, Lyme disease
    c.cancer (with the exception of basal cell skin cancer)
    d.IgM paraproteinemia with anti-myelin associated glycoprotein antibodies
    10.Diabetic neuropathy
    11.Cardiac insufficiency (New York Heart Association [NYHA] III/IV), cardiomyopathy, significant cardiac dysrhythmia requiring treatment, unstable or advanced ischemic heart disease
    12.Severe liver disease (ALAT 3x > normal value)
    13.Severe kidney disease (creatinine 1.5x > normal value)
    14.Hepatitis B, hepatitis C or HIV infection
    15.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
    16.Body mass index (BMI) ≥40 kg/m2
    17.Patients with uncompensated hypothyroidism (abnormally high Thyroid-Stimulating Hormone [TSH] and abnormally low Thyroxine [T4]) or known vitamin B12 deficiency if they don’t receive adequate substitution therapy
    18.Medical conditions whose symptoms and effects could alter protein catabolism and/or IgG utilization (e.g. protein-losing enteropathies, nephrotic syndrome)
    19.Known IgA deficiency with antibodies to IgA
    20.History of severe hypersensitivity, e.g. anaphylaxis or severe systemic response to immuno-globulin, blood or plasma derived products, or any component of NewGam
    21.Known blood hyperviscosity, or other hypercoagulable states
    22.Use of other blood or plasma-derived products within three months prior to Visit 2
    23.Patients with a past or present history of drug abuse or alcohol abuse within the preceding five years prior to baseline visit
    24.Patients unable or unwilling to understand or comply with the study protocol
    25.Participation in another interventional clinical study with IMP treatment currently or during the three months prior to Visit 2
    26.Women who are breast feeding, pregnant, or planning to become pregnant, or are unwilling to use an effective birth control method (such as implants, injectables, combined oral contraceptives, some intrauterine devices (IUDs), sexual abstinence or vasectomized partner) while on study
    E.5 End points
    E.5.1Primary end point(s)
    Primary
    Proportion of responders in the 1.0 g/kg NewGam arm at Week 24 (Termination Visit) relative to baseline (Week 0). A responder being defined as a patient with a decrease 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 and/or legs)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 24
    E.5.2Secondary end point(s)
    Secondary
    1-Proportion of responders in the 0.5 g/kg and 2.0 g/kg NewGam arms at Week 24 relative to baseline compared to the 1.0 g/kg arm, based on the adjusted INCAT disability score
    2- Proportion of responders in the 0.5 g/kg and 2.0 g/kg NewGam arms at Week 24 relative to baseline at Week 0 compared to the 1.0 g/kg arm, based on the grip strength (Martin Vigorimeter) using the previously published minimum clinically important difference (MCID) cut-off of 8 kPa
    3- Proportion of responders in the 0.5 g/kg and 2.0 g/kg NewGam arms at Week 24 relative to baseline at Week 0 compared to the 1.0 g/kg arm, based on the I-RODS scores using the MCID concept related to the varying standard errors (MCID-SE) as recently demonstrated.
    4- Time to first confirmed worsening on the adjusted INCAT disability scale by at least 1 point from the value at baseline (Week 0)
    5 - Mean change from baseline (Week 0) to Termination Visit in
    a.- grip strength of both hands (assessed by Martin Vigorimeter)
    b.- Inflammatory Rasch-built overall disability sum score (I-RODS using the concept of MCID-SE as recently reported; Appendix 1) and number of improvers
    c.- sum of the distal evoked amplitude of 4 right sided and 4 left sided motor nerves (peroneal, tibial, ulnar and median)
    d.- Pain Intensity Numeric Rating Scale (PI-NRS)
    6- Time to first confirmed worsening on the I-RODS scale
    7- Time to 1 point decrease (improvement of disability) in adjusted INCAT disability score
    8-Time to decrease in I-RODS scores

    Exploratory:
    1- Mean change from baseline (Week 0) to Termination Visit in:
    a.- modified Fatigue Severity Scale (FSS; 7-item scale from 0-21 points; see Appendix 2)
    b.- number of improvers by at least 4 points in the MRC sum score (according to the universal rule of minimal clinical important difference)
    c.- SF-36 Health Survey physical composite score (PCS), mental composite score (MCS) and their 8 health domains (see Appendix 3)
    d.- additional NCS analyses (e.g. individual nerve analysis)
    2 - Time to decrease in MRC sum score to or below baseline value after temporary improvement (increase)
    Safety (throughout the entire Wash-out and Dose-evaluation Phases):
    1 - Occurrence of all adverse events (AEs)
    2 - Short term tolerance parameters including vital signs
    3- Physical/neurological examination
    4 - Laboratory parameters (hematology and clinical chemistry) and tests for viral safety
    E.5.2.1Timepoint(s) of evaluation of this end point
    Week 24
    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) 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 No
    E.8.2.4Number of treatment arms in the trial3
    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 EEA26
    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
    Australia
    Bulgaria
    Canada
    Czech Republic
    Denmark
    Germany
    Hungary
    Poland
    Romania
    Russian Federation
    Sweden
    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
    LVLS
    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 months6
    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 months6
    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: 130
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 10
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers Yes
    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 state12
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 57
    F.4.2.2In the whole clinical trial 140
    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 Decision2017-04-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-04-28
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2019-09-05
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