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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: EUCTR
Last refreshed on: 7 January 2013
Main ID:  EUCTR2005-002463-88-IT
Date of registration: 21/04/2006
Prospective Registration: Yes
Primary sponsor: ORFAGEN
Public title: fficacy and safety of a human normal immunoglobulin product for intravenous administration (IVIg) in the treatment of dermatomyositis (DM) and polymyositis (PM)
Scientific title: 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.
Date of first enrolment: 17/08/2006
Target sample size: 44
Recruitment status: Not Recruiting
URL:  https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2005-002463-88
Study type:  Interventional clinical trial of medicinal product
Study design:  Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 2  
Phase: 
Countries of recruitment
Austria Czech Republic Germany Hungary Italy
Contacts
Name: CRDPF Langlade   
Address:  3 avenue Hubert Curien 31035 Toulouse cedex 1 France
Telephone: +33534506458
Email: orfagen@orfagen.com
Affiliation:  ORFAGEN
Name: CRDPF Langlade   
Address:  3 avenue Hubert Curien 31035 Toulouse cedex 1 France
Telephone: +33534506458
Email: orfagen@orfagen.com
Affiliation:  ORFAGEN
Key inclusion & exclusion criteria
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, OR Patients with a contra-indication or a major side-effect to prednisone or methotrexate / other IS,ORPatients 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,ORDM 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, ORPatients 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.
Are the trial subjects under 18? no
Number of subjects for this age range: 0
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

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.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
Health Condition(s) or Problem(s) studied
Idiopathic dermatomyositis and polymyositis with insufficiently improved muscle strength under conventional therapy (glucocorticosteroids associated with immunosuppressors).
MedDRA version: 14.1 Level: HLT Classification code 10003821 Term: Muscular autoimmune disorders System Organ Class: 100000004870
Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Intervention(s)

Trade Name: IGVENA*FL 200ML 10G+SET
Pharmaceutical Form: Solution for injection
Concentration unit: g/ml gram(s)/millilitre
Concentration type: equal
Concentration number: .05-

Primary Outcome(s)
Main Objective: to assess the efficacy of the IVIg product as adjunctive treatment to conventional glucocorticosteroids (GS) and immunosuppressors (IS) in DM and PM patients with insufficient improvement of muscle strength.
Primary 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.
Secondary Objective: to assess the overall safety profile of the IVIg product in DM and PM patients.
Timepoint(s) of evaluation of this end point: 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)
Secondary Outcome(s)
Secondary end point(s): - Time course evaluation of muscle strength using BMRC index (run-in period, Period I and Period II). - Physical function recorded by the patients, as measured by the Health Assessment Questionnaire (HAQ) scale. - Global disease activity made by the Investigators, using a visual analog scale (VAS). - Global disease activity made by the patients, using a visual analog scale (VAS). - Serum activity of muscle enzymes: creatine kinase (CK), aldolase, lactate deshydrogenase (LDH). The activity enzymes Aspartate aminotransferase (ASAT) and alanine aminotransferase (ALAT) will be dosed in routine blood laboratory tests. - Measurement outcome as defined by International Myositis Assessment and Clinical Studies Group (IMACS). - Cutaneous signs severity, according to the modified 3-point scale from Göttfried (Göttfried et al., 2000) : 0 = no symptom, 1 = slight heliotrope erythema, scaly eruptions on the knuckles (Gottron’s papules), and/or pruritic hairline and scalp involvement 2 = severe heliotrope erythema, swollen eyelids, splinter hemorrhages, prominent Gottron’s papules, erosions and painful cutaneous ulceration, or necrosis. - Other organ involvement (cardiac, pharyngeal, gastro-intestinal, joint, pulmonary, others) assessed by the Investigators, using clinical and paraclinical examinations. - Consumption of prednisone during the run-in period, Period I and Period II. - Consumption of IS during period II. - Routine blood laboratory tests (haematology, chemistry). - Adverse events.
Timepoint(s) of evaluation of this end point: Timepoints described along with secondary endpoints
Secondary ID(s)
2005-002463-88-DE
ISRCTN87782942
L00133IV301(ORF)
Source(s) of Monetary Support
ORFAGEN
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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