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    The EU Clinical Trials Register currently displays   38876   clinical trials with a EudraCT protocol, of which   6392   are clinical trials conducted with subjects less than 18 years old.
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    EudraCT Number:2016-004766-26
    Sponsor's Protocol Code Number:NL58747
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2016-12-21
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedNetherlands - Competent Authority
    A.2EudraCT number2016-004766-26
    A.3Full title of the trial
    Intravenous immunoglobulins as early treatment in newly diagnosed idiopathic inflammatory myopathies (IMMEDIATE): a pilot study.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Early treatment with intravenous immunoglobulins for newly diagnosed myositis patients
    Vroege behandeling met intraveneuze immunoglobuline bij nieuw gediagnosticeerde patiënten met myositis
    A.3.2Name or abbreviated title of the trial where available
    A.4.1Sponsor's protocol code numberNL58747
    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 SponsorAcademic Medical Center
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportCSL Behring
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAcademic Medical Center
    B.5.2Functional name of contact pointAnneke J. van der Kooi
    B.5.3 Address:
    B.5.3.1Street AddressMeibergdreef 9
    B.5.3.2Town/ cityAmsterdam
    B.5.3.3Post code1105AZ
    B.5.4Telephone number+31(0)205663847
    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. name Privigen®
    D. of the Marketing Authorisation holderCSL Behring GmbH, Marburg, Germany
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 namePrivigen®
    D.3.2Product code J06BA02
    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 INNHuman normal immunoglobulin
    D.3.9.3Other descriptive nameHUMAN NORMAL IMMUNOGLOBULIN (IV)
    D.3.9.4EV Substance CodeSUB12041MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/l milligram(s)/litre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    idiopathic inflammatory myopathies
    E.1.1.1Medical condition in easily understood language
    auto-immune inflammation/disease of the muscles
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    aim of exploring efficacy of early intravenous immunoglobulin treatment for patients with idiopathic inflammatory myopathies
    E.2.2Secondary objectives of the trial
    aim of exploring safety of early intravenous immunoglobulin treatment for patients with idiopathic inflammatory myopathies, and feasibility with respect to a future phase 3 trial
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Adult patients (age ≥ 18 years)

    • Subacute-onset of disease (disease duration of ≤ 9 months) of muscle symptoms.

    • Biopsy proven IIMs (see for diagnostic criteria Hoogendijk et al. 2004, note: ASS is considered a separate entity, but new criteria in which it has been included, has yet to be published).
    o Dermatomyositis
    o Non-specific myositis/antisynthetase syndrome
    o Immune-mediated necrotizing myopathy

    • Minimal disability of:
    o MMT score reduction of 10% or lower and,
    o 2 other of the core set measures score abnormalities defined as:
    - Physician Global Activity 2 cm or more
    - Patient Global Activity 2 cm or more
    - Health Assessment Questionnaire 0.25 or more
    - Muscle enzymes more than 1.5 times upper limit of normal
    - Extra-Muscular Activity 2 cm or more
    E.4Principal exclusion criteria
    • IVIg treatment related:
    o Subjects who have received systemic immunosuppressive/immunomodulatory medication within the last 6 months with the execption of prednisone dosed as follows:
     daily dose of 20mg or lower
     used for 2 weeks or less
     no evident clinical response

    o history of thrombotic episodes within the 2 years prior to enrolment
    o known allergic reactions or other severe reactions to any blood-derived product
    o known IgA deficiency and anti-IgA serum antibodies
    o pregnancy (wish).

    • Conditions that are likely to interfere with:
    o compliance (legal incompetent and/or incapacitated patients are excluded) or,
    o evaluation of efficacy (e.g. due to severe pre-existing disability as result of any other disease than IIM or language barrier).

    • Lack of informed consent (IC)
    E.5 End points
    E.5.1Primary end point(s)
    The number of patients with clinical significant improvement, defined as ≥40 points improvement on a continuous, weighted score of 6 core set measures (as developed by the International Myositis Assessment and Clinical Studies group) after 9 weeks of treatment.
    E.5.1.1Timepoint(s) of evaluation of this end point
    9 weeks after start of IVIg treatment
    E.5.2Secondary end point(s)
    Efficacy of IVIg (measured 9 weeks after treatment)
    1. Time to at least moderate improvement on the CIS
    2. Minimal improvement (20-40 points) on each of the 6 IMACS CSMs
    a. Physician Global Activity
    b. Patient Global Activity
    c. Manual Muscle Testing
    d. Health Assessment Questionnaire
    e. Muscle enzymes
    f. Extra-Muscular Activity
    3. Moderate improvement or more (≥40 points) on each of the 6 IMACS CSMs
    a. Physician Global Activity
    b. Patient Global Activity
    c. Manual Muscle Testing
    d. Health Assessment Questionnaire
    e. Muscle enzymes
    f. Extra-Muscular Activity
    4. The number of deterioration patients needing rescue therapy
    5. Significant improvement in Academic Medical Center Linear Disability Scale (ALDS)
    6. Significant improvement in Modified Rankin Scale (MRS)
    7. Significant improvement of dysphagia (if present) on the Amyotrofic Lateral Sclerosis Severity Scale Swallowing
    8. Significant improvement in dynamometric muscle strength
    9. Significant improvement on the Rasch modified MRC Sum Score (Rasch-MRC)
    10. Significant improvement on the EuroQol Group Health Questionnaire (EQ-5D-5L)
    11. The number of participants with significant decrease of T2 weighted (T2)/short tau inversion recovery (STIR) hyperintensity of muscles and fascia on MRI
    12. The number of patients with significant change of size and echo intensity of muscles and fascia on ultrasound (US)
    13. The number of patients with change of highly expanded B-cell clones
    14. RNA and RBM20 expression before and after IVIg treatment
    15. Galectin-9 and CXCL10 levels before and after IVIg treatment

    Safety of IVIg (measured during the total duration of the study)
    The number of serious adverse events (SAEs)

    Feasibility of a future trial (assessed at end report)
    1. Process
    a. recruitment rate
    b. retention rate
    2. Resources
    a. Estimation of total time investment
    b. Estimation of total financial investment
    3. Management:
    a. Potential human/personnel concerns
    b. Potential data concerns
    c. Potential logistics concerns
    E.5.2.1Timepoint(s) of evaluation of this end point
    efficacy 9 weeks after start IVIg treatment, safety during total duration of the study, feasibility at end reports
    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 Yes
    E.6.13.1Other scope of the trial description
    feasibility with respect to an eventual future phase 3 trial
    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. trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    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 No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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, or in case of unacceptable preliminary results, defined as:
    • 100% deterioriation and/or SAEs rate in the first 4 inclusions (≥4 study subjects)
    • ≥80% deterioriation and/or SAEs rate in the first 5 inclusions (≥4 study subjects)
    • ≥60% deterioriation and/or SAEs rate in the first 6 inclusions (≥4 study subjects)
    • ≥50% deterioriation and/or SAEs rate after the first 7 inclusions (≥4 study subjects)
    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 months
    E.8.9.1In the Member State concerned days
    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: 15
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 5
    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 state20
    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)
    Participants will be followed and treated to the discretion of the treating physician (= standard care).
    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 Decision2016-12-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-03-08
    P. End of Trial
    P.End of Trial StatusOngoing
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