Early treatment with intravenous immunoglobulins for newly diagnosed myositis patients

Phase 1

• Conditions: idiopathic inflammatory myopathies; Therapeutic area: Diseases [C] - Immune System Diseases [C20]

• Registration Number: EUCTR2016-004766-26-NL
• Lead Sponsor: Academic Medical Center

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2016-12-21
• Last Update Posted: 29 January 2018

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 20

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).
oDermatomyositis
oNon-specific myositis/antisynthetase syndrome
oImmune-mediated necrotizing myopathy

•Minimal disability of:
oMMT score reduction of 10% or lower and,
o2 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

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 15
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 5

Exclusion Criteria

•IVIg treatment related:
oSubjects 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

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

•Conditions that are likely to interfere with:
ocompliance (legal incompetent and/or incapacitated patients are excluded) or,
oevaluation 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)

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: aim of exploring efficacy of early intravenous immunoglobulin treatment for patients with idiopathic inflammatory myopathies;Secondary Objective: 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;Primary 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.;Timepoint(s) of evaluation of this end point: 9 weeks after start of IVIg treatment