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    Summary
    EudraCT Number:2017-002710-31
    Sponsor's Protocol Code Number:SCGAM-02
    National Competent Authority:Czechia - SUKL
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2018-06-01
    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 ConcernedCzechia - SUKL
    A.2EudraCT number2017-002710-31
    A.3Full title of the trial
    DOUBLE-BLIND, RANDOMIZED, PLACEBO- CONTROLLED PHASE III STUDY EVALUATING EFFICACY AND SAFETY OF SUBCUTANEOUS HUMAN IMMUNOGLOBULIN (OCTANORM) IN PATIENTS WITH DERMATOMYOSITIS.
    Dvojitě zaslepená randomizovaná placebem kontrolovaná studie fáze III hodnotící účinnost a bezpečnost subkutánního lidského imunoglobulinu (octanorm) u pacientů s dermatomyozitidou.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    DOUBLE-BLIND, RANDOMIZED, PLACEBO- CONTROLLED PHASE III STUDY EVALUATING EFFICACY AND SAFETY OF SUBCUTANEOUS HUMAN IMMUNOGLOBULIN (OCTANORM) IN PATIENTS WITH DERMATOMYOSITIS.
    A.4.1Sponsor's protocol code numberSCGAM-02
    A.5.4Other Identifiers
    Name:INDNumber:17515
    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 number431610321202
    B.5.5Fax number431610329249
    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 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 % percent
    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 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 placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Dermatomyositis
    E.1.1.1Medical condition in easily understood language
    Subject with Dermatomyositis
    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 10012503
    E.1.2Term Dermatomyositis
    E.1.2System Organ Class 10040785 - Skin and subcutaneous tissue disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of this study is to determine the efficacy of subcutaneous immunoglobulin octanorm in the maintenance treatment of DM patients who have previously responded to IGIV therapy.
    E.2.2Secondary objectives of the trial
    The secondary objectives of this study are:
    •to assess other efficacy outcomes at the end of study (Week 32 or Drop-out Visit;
    •to assess the effect of octanorm on Quality of Life measures;
    •to assess the treatment compliance of home treatment with octanorm;
    to evaluate the safety and tolerability of octanorm in subjects with Dermatomyositis
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Subjects with diagnosis of definite or probable DM according to the Bohan and Peter criteria.
    2.Subjects who have responded to IGIV treatment as assessed by the treating physician and being on a stable dose for at least 3 months on 2 g/kg bodyweight (+/- 10%) prior to study enrolment.
    3.For subjects being on other medication(s) for the treatment of DM (immunosuppressants, corticosteroids): a) subject was on such medication(s) at the start of IGIV treatment in the first place, and b) received such medication(s) for at least 3 months prior to study enrolment and at a stable dose for at least 4 weeks prior to study enrolment at the maximally allowed conditions as per Table 2 (see section 4.2.1).
    4.MMT-8 score ≥144, with at least 3 other CSM to be normal or near normal as per the following criteria: Visual Analogue Scale [VAS] of patient global disease activity
    ≤2 cm, physician’s global disease activity ≤2 cm, extra-muscular disease activity ≤2 cm; no muscle enzyme >4 times upper limit of normal due to myositis, Health Assessment Questionnaire [HAQ] ≤0.25.
    5.Males or females 18 to <80 years of age.
    6.Voluntarily given, fully informed written consent obtained from subject before any study-related procedures are conducted.
    7.Subject must be capable and willing to understand and comply with the relevant aspects of the study protocol
    E.4Principal exclusion criteria
    1-Cancer-associated myositis, defined as the diagnosis of myositis within 2 years of the diagnosis of cancer (except basal or squamous cell skin cancer or carcinoma in situ of the cervix that has been excised and cured at least 1 year for basal or squamous cell skin cancer and 5 years for carcinoma in situ of the cervix must have passed since excision).
    2-Evidence of active malignant disease or malignancies diagnosed within the previous 5 years (including hematological malignancies and solid tumors).
    3-Subjects with overlap myositis connective tissue disease associated DM, inclusion body myositis, polymyositis, juvenile dermatomyositis or drug-induced myopathy.
    4-Subjects with immune-mediated necrotizing myopathy with absence of typical DM rash.
    5-Subjects with generalized, severe musculoskeletal conditions other than DM that prevent a sufficient assessment of the subject by the physician.
    6-Subjects who received blood or plasma-derived products (other than IGIV) or plasma exchange within the last 3 months before enrolment.
    7-Subjects with administration of permitted concomitant medications exceeding the maximally allowed conditions as per section 4.2.1
    8-Subjects with administration of forbidden concomitant medications within the washout periods as defined in Table 3
    9-Subjects starting or planning to start a physical therapy–directed exercise regimen during the trial. Subjects on stable physical therapy for >4 weeks are allowed but the regimen should remain the same throughout the trial.
    10-Cardiac insufficiency (New York Heart Association III/IV).
    11-Severe liver disease, with signs of ascites and hepatic encephalopathy.
    12-Severe kidney disease (as defined by estimated glomerular filtration rate (eGFR)
    < 30 mL/min/1.73 m2).
    13-Known active or chronic hepatitis B, hepatitis C or HIV infection. Past hepatitis B or C infection that has been cured is allowed.
    14-Subjects with a history of deep vein thrombosis within the last year prior to study enrolment or pulmonary embolism.
    15-Body mass index >40 kg/m2 and/or body weight >120 kg.
    16-Medical conditions whose symptoms and effects could alter protein catabolism and/or IgG utilization.
    17-Known IgA deficiency with antibodies to IgA.
    18-History of hypersensitivity, anaphylaxis or severe systemic response to immuno- globulin, blood or plasma derived products or any component of octanorm 16.5% such as polysorbate 80 or to sodium chloride.
    19-Known blood hyperviscosity, or other hypercoagulable states.
    20-Subjects with a history of drug abuse within the past 5 years prior to study enrolment.
    21-Participating in another interventional clinical study with investigational treatment within 3 months prior to study enrolment except study GAM10-08.
    22-Women who are breast feeding, pregnant, or planning to become pregnant during study.
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of patients with clinically important deterioration during the treatment period up to Week 32. A patient with clinically important deterioration is defined as a patient with 1) MMT-8 worsening ≥ 6 points (scale of 150) OR CDASI (Total Activity Score)worsening ≥ 5 points, AND 2) a Physician’s Global Disease Activity VAS worsening 2 cm.
    E.5.1.1Timepoint(s) of evaluation of this end point
    End of the study
    E.5.2Secondary end point(s)
    •Mean change from baseline (Week 0-defined as end of IGIV therapy) to end of the treatment period (Week 32 or Drop-Out Visit) in:
    oModified Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI);
    oSix Myositis CSM: MMT-8, Physician’s Global Disease Activity, Patient’s Global Disease Activity, Extra-Muscular Disease Activity, Muscle enzymes, Health Assessment Questionnaire (HAQ);
    oSF-36v2 Health Survey.
    •Mean change in TIS from Baseline (Week 0) to end of treatment period (Week 32 or Drop-Out Visit).
    •Time to clinically important deterioration during the treatment period.
    •Number and type of deviations from protocol requirements relating to home treatment (dosing, timing).
    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 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 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 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 trial2
    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 concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA17
    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
    Russian Federation
    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 years1
    E.8.9.1In the Member State concerned months11
    E.8.9.1In the Member State concerned days19
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months11
    E.8.9.2In all countries concerned by the trial days19
    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: 70
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 44
    F.4.2.2In the whole clinical trial 78
    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 each subject will be treated at the discretion of their physician, with other most suitable standard therapies and medical care, currently available locally 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 Decision2018-06-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-06-27
    P. End of Trial
    P.End of Trial StatusCompleted
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