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    The EU Clinical Trials Register currently displays   40628   clinical trials with a EudraCT protocol, of which   6628   are clinical trials conducted with subjects less than 18 years old.
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    EudraCT Number:2005-002463-88
    Sponsor's Protocol Code Number:L00133 IV 301 (ORF)
    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:2009-10-05
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedCzechia - SUKL
    A.2EudraCT number2005-002463-88
    A.3Full title of the trial
    Efficacy and safety of a human normal immunoglobulin product for intravenous administration (IVIg) in the treatment of dermatomyositis (DM) and polymyositis (PM): prospective, randomised, double-blind, placebo-controlled study.
    A.4.1Sponsor's protocol code numberL00133 IV 301 (ORF)
    A.5.1ISRCTN (International Standard Randomised Controlled Trial) NumberISRCTN87782942
    A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorOrfagen
    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 support
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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 Ig Vena
    D. of the Marketing Authorisation holderKedrion S.p.A
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/03/169 and EU/3/03/170
    D.3 Description of the IMP
    D.3.1Product nameIg VENA (10g/200mL)
    D.3.2Product code L0133
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeL0133
    D.3.9.3Other descriptive nameHuman normal immunoglogulin (IVIg)
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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) Information not present in EudraCT
    D. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product Information not present in EudraCT
    D. ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D. on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    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 Information not present in EudraCT
    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 use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Idiopathic DM and PM with insufficiently improved muscle strenght under conventional therapy (Glucocorticosteroids associated with immunosuppressors)
    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
    To assess the efficacy of the IVIg product as adjunctive treatment to conventional glucocorticosteroids (GS) and immunosuppressors (IS) in dermatomyositis (DM) and polymyositis (PM) patients with insufficient improvement of muscle strength.
    E.2.2Secondary objectives of the trial
    To assess the overall safety profile of the IVIg product in DM and PM patients.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    -Male or female patients of at least 18 years of age

    -Patients fulfilling the diagnostic criteria (definite or probable) of the European Neuro-Muscular Committee (ENMC) for idiopathic DM and PM.

    -Patients with an active DM or PM disease who received conventional therapies for at least 14 weeks: prospective or clearly-documented retrospective administration of oral prednisone 1mg/Kg per day for at least 4 weeks, with or without IS, followed by IS at stable dose (methotrexate MTX 15-50 mg per week, or other IS) and prednisone for at least 10 weeks,

    Patients with a contra-indication or a major side-effect to prednisone or methotrexate / other IS,

    Patients under bitherapy (prednisone and MTX / other IS) with a documented deterioration of their BMRC score, as follows:·
    - Of at least 18 points if BMRC at the beginning of the run-in period over 56·
    - Of at least 12 points if BMRC at the beginning of the run-in period between 40.5 and 56 included·
    - Of at least 8 points if BMRC at the beginning of the run-in period below 40.5,

    DM patients under bitherapy (prednisone and MTX / other IS) having a documented deterioration of their cutaneous signs, as follows:·
    - Cutaneous signs aggravated (score from 0 or 1 to 2)·
    - Cutaneous signs of 2 at two consecutive visits,

    Patients under bitherapy (prednisone and MTX / other IS) with an onset of visceral involvement (e.g. pharyngeal, pulmonary, cardiac).

    -Patients with no significant improvement of muscle strength under conventional therapy, as follows:·
    *Less of 8 points if BMRC score over 56 at the beginning of the run-in period ·
    *Less of 12 points if BMRC score between 40.5 and 56 at the beginning of the run-in period ·
    *Less of 18 points if BMRC score below 40.5 at the beginning of the run-in period.

    -Patients with BMRC index between 24 and 72 at baseline.

    - For French centres, patients affiliated to the French Security System.

    -Patients able to follow instructions.

    -Written informed consent from the patients.
    E.4Principal exclusion criteria
    - Pregnant women, nursing mothers and women of childbearing potential with no reliable contraception.
    -Patients who do not fulfill the ENMC diagnostic criteria (definite or probable) of idiopathic DM and PM.
    -Patients with a diagnosis of paraneoplasic DM or PM.
    -Juvenile DM and PM (age less than 18 years).
    -DM patients with no muscle involvement.
    -Patients with life expectancy of less than 3 months.
    -Patients whose muscle strength is responsive to conventional therapy, i.e. with the following BMRC improvement of at least:
    · 18 points of their BMRC index at baseline compared to the beginning of the run-in period if BMRC below 40,5 at first run-in period assessment
    · 12 points of their BMRC index at baseline compared to the beginning of the run-in period if BMRC between 40.5 and 56 included at first run-in period assessment
    · 8 points of their BMRC index at baseline compared to the beginning of the run-in period if BMRC over 56 at first run-in period assessment
    - Patients with an BMRC index of less than 24 or more than 72.
    - Patients having received a bolus of methylprednisone within 3 weeks prior to study entry.
    - Patients with a known allergy to one of the ingredients of the IVIg test product.
    - Patients with decompensated cardiac insufficiency or any other intercurrent condition that may alter the study conduct.
    - Patients with positive Coomb’s test at baseline.
    - Patients who refuse to participate in the study.
    - Patients who are not able to follow instructions.
    - Patients who are protected by the law (guardianship, trusteeship).
    - Patients who have participated in a study within the 3 months prior to study run-in period.
    - Patients who refuse to give written informed consent.
    E.5 End points
    E.5.1Primary end point(s)
    Muscle strength intensity, as defined by the BMRC and modified by Cherin (Cherin et al., 1994), which assigns the grades 0 to 11 to indicate the level of muscle power in 8 muscle groups (neck flexors, trapezius, deltoid, biceps, psoas, maximus and medius gluteus, and quadriceps). BMRC index will be determined by an independent physician/physiotherapist masked to treatment allocation and study visits.
    Treatment response will be defined as an improvement from baseline of BMRC score at the end of Period I (84 days or earlier in case of significant deterioration as described above) as follows:
    -Patients with baseline BMRC score between 24 and 40 included: at least 18 points improvement, i.e. an average improvement of more than 2 grades / muscle group (i.e. from patients having contraction with moving initiation to patients having at least moving in the plan).
    -Patients with baseline BMRC score between 40.5 and 56 included: at least 12 points improvement, i.e. an average improvement of at least 1.5 grades / muscle group (i.e. from patients having moving initiation in the plan, to patients having at least moving against gravity).
    -Patients with baseline BMRC score between 56.5 and 72 included: at least 8 points improvement, i.e. an average improvement of at least 1 grade / muscle group (i.e. from patients having an incomplete moving against gravity to patients having at least a moving initiation against resistance).
    Patients prematurely switched from Period I to Period II for BMRC index aggravation will be considered as non-responders.
    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. 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 Yes
    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 Yes
    E. trial design description
    period I : double blind; period II : open
    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.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    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 EEA20
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    E.8.7Trial has a data monitoring committee No
    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
    Provided in the protocol.
    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 months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months0
    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.1.3Elderly (>=65 years) Yes
    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 40
    F.4.2.2In the whole clinical trial 44
    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)
    Provided in the protocol.
    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 Decision2009-11-03
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2009-12-16
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2011-09-06
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