Efficacy and safety of L0133 in the treatment of dermatomyositis and polymyositis: prospective, randomised, double-blind, placebo-controlled study
- Conditions
- Dermatomyositis (DM) and polymyositis (PM)Skin and Connective Tissue DiseasesSystemic connective tissue disorders
- Registration Number
- ISRCTN87782942
- Lead Sponsor
- Orfagen (France)
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- All
- Target Recruitment
- 44
Current inclusion criteria as of 14/08/2009:
1. Male or female patients of at least 18 years of age
2. Patients fulfilling the diagnostic criteria (definite or probable) of the European Neuromuscular Committee (ENMC) for idiopathic DM and PM
3.1. Patients with an active DM or PM disease who received conventional therapies for at least 14 weeks: oral prednisone 1 mg/kg per day for at least 4 weeks, with or without immunosuppressors (IS), followed by IS at stable dose and prednisone for at least 10 weeks, or
3.2. Patients with a contra-indication or a major side-effect to prednisone or methotrexate/other IS, or
3.3. Patients under biotherapy with a documented deterioration of their British Medical Research Council (BMRC) score, or
3.4. DM patients under biotherapy having a documented deterioration of their cutaneous signs, or
3.5. Patients under biotherapy with an onset of visceral involvement
4. Patients with no significant improvement of muscle strength under conventional therapy
5. Patients with BMRC index between 24 and 72 at baseline
Previous inclusion criteria as of 18/01/2008:
1. Male or female patients of at least 18 years of age
2. Patients fulfilling the diagnostic criteria (definite or probable) of the European Neuromuscular Committee (ENMC) for idiopathic DM and PM
3. Patients with an active DM or PM disease who received conventional therapies for at least 14 weeks: oral prednisone 1 mg/Kg per day, immunosuppressors at stable dosage
4. Patients with no significant improvement of muscle strength under conventional therapy, i.e. with an improvement of their muscle British Medical Research Council (BMRC) index of less than 18 points at baseline compared to the beginning of the run-in period
5. Patients with BMRC index between 32 and 64 at baseline
Initial inclusion criteria:
1. Male or female patients of at least 18 years of age
2. Patients fulfilling the diagnostic criteria (definite or probable) of the European Neuromuscular Committee (ENMC) for idiopathic DM and PM
3. Patients with an active DM or PM disease who received conventional therapies for at least 18 weeks: oral prednisone 1 mg/Kg per day, Methotrexate 15 mg per week
4. Patients with no significant improvement of muscle strength under conventional therapy, i.e. with an improvement of their muscle British Medical Research Council (BMRC) index of less than 18 points at baseline compared to the beginning of the run-in period
5. Patients with BMRC index between 32 and 64 at baseline
Current exclusion criteria as of 14/08/2009:
1. Pregnant women, nursing mothers and women of childbearing potential with no reliable contraception
2. Patients who do not fulfil the ENMC diagnostic criteria (definite or probable) of idiopathic DM and PM
3. Patients with a diagnosis of paraneoplasic DM or PM
4. Juvenile DM and PM (age less than 18 years)
5. DM patients with no muscle involvement
6. Patients with life expectancy of less than three months
7. Patients whose muscle strength is responsive to conventional therapy, i.e. with an 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 if BMRC between 40.5 and 56 included at first run-in period assessment and 8 points if BMRC over 56 at first run-in period assessment
8. Patients with an BMRC index of less than 24 or more than 72
9. Patients having received a bolus of methylprednisone within three weeks prior to study entry
10. Patients with a known allergy to one of the ingredients of the IVIg test product
11. Patients with decompensated cardiac insufficiency or any other inter-current condition that may alter the study conduct
12. Patients with positive Coomb's test at baseline
Previous exclusion criteria as of 18/01/2008:
1. Pregnant women, nursing mothers and women of childbearing potential with no reliable contraception
2. Patients who do not fulfil the ENMC diagnostic criteria (definite or probable) of idiopathic DM and PM
3. Patients with a diagnosis of paraneoplasic DM or PM
4. Juvenile DM and PM (age less than 18 years)
5. DM patients with no muscle involvement
6. Patients with life expectancy of less than three months
7. Patients with severe forms of DM and PM: pharyngeal, cardiac or pulmonary involvement
8. Patients without conventional treatments as first-line therapy for at least 14 weeks: oral prednisone 1 mg/Kg per day, immunosuppressors at stable dosage
9. Patients whose muscle strength is responsive to conventional therapy, i.e. with an improvement of at least 18 points of their BMRC index at baseline compared to the beginning of the run-in period
10. Patients with a BMRC index of less than 32 or more than 64
11. Patients having received a bolus of methylprednisone within three months prior to study entry
12. Patients with a known allergy to one of the ingredients of the IVIg test product
13. Patients with decompensated cardiac insufficiency or any other inter-current condition that may alter the study conduct
14. Patients with positive Coomb's test at baseline
Initial exclusion criteria:
1. Pregnant women, nursing mothers and women of childbearing potential with no reliable contraception
2. Patients who do not fulfil the ENMC diagnostic criteria (definite or probable) of idiopathic DM and PM
3. Patients with a diagnosis of paraneoplasic DM or PM
4. Juvenile DM and PM (age less than 18 years)
5. DM patients with no muscle involvement
6. Patients with life expectancy of less than three months
7. Patients with severe forms of DM and PM: pharyngeal, cardiac or pulmonary involvement
8. Patients without conventional treatments as first-line therapy: prednisone 1 mg/Kg daily Methotrexate 15 mg per week)
9. Patients whose muscle strength is responsive to conventional therapy, i.e. with an improvement of at least 18 points of their BMRC index at baseline compared to the beginning of the run-in period
10. Patients with a BMRC index of less than
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 1. Muscle strength intensity, as defined by the BMRC.<br>2. Treatment response will be defined as an improvement from baseline of BMRC score at the end of Period I.
- Secondary Outcome Measures
Name Time Method 1. Time course evaluation of muscle strength using BMRC index (run-in period, Period I and Period II).<br>2. Physical function recorded by the patients, as measured by the Health Assessment Questionnaire (HAQ) scale.<br>3. Visual Analogue Scale (VAS) global disease activity made by the Investigators and the patients<br>4. Serum activity of muscle enzymes - Measurement outcome as defined by International Myositis Assessment and Clinical Studies Group (IMACS).<br>5. Cutaneous signs severity, according to the modified three-point scale from Göttfried <br>6. Other organ involvement (cardiac, pharyngeal, gastro-intestinal, joint, pulmonary, others) assessed by the Investigators, using clinical and paraclinical examinations.<br>7. Consumption of prednisone during the run-in period, Period I and Period II.<br>8. Consumption of IS during period II.<br>9. Routine blood laboratory tests (haematology, chemistry).<br>10. Adverse events.