Idiopathic DM and PM with insufficiently improved muscle strength under conventional therapy (Glucocorticosteroids associated with immunosuppressors).
Conditions
Interventions
Trade Name: Ig VENA
Product Name: Ig VENA (10g/200mL)
Product Code: L0133
Pharmaceutical Form: Solution for infusion
Current Sponsor code: L0133
Other descriptive name: Human normal immunoglobulin (IV
Sponsors
Orfagen
Eligibility
Sex/Gender
All
Inclusion 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, OR 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, OR 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, OR 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. Are the trial subjects under 18? no Number of subjects for this age range: 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
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 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.;Secondary Objective: To assess the overall safety profile of the IVIg product in DM and PM patients;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 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. | — |
Countries
Austria, Czech Republic, Germany, Hungary, Italy
Outcome results
None listed