None listed
Conditions
Interventions
Study subjects will be treated with intravenous immunoglobulin (Privigen®). Initial dose of IVIg is 2 g/kg over 2-4 days, followed by 3 infusions of 1 g/kg on 1-2 days every 3 weeks
Sponsors
Department of Neurology, Academic Medical Center Amsterdam
Eligibility
Inclusion criteria
Inclusion criteria: • Adult patients (age ≥ 18 years) • Treatment naïve patients • Subacute-onset of disease (disease duration of ≤ 9 months) • 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). o Dermatomyositis o Polymyositis/overlap myositis/antisynthetase syndrome o Immune-mediated necrotizing myopathy
Exclusion criteria
Exclusion criteria: • IVIg treatment related: o Subjects who have received clinical relevant immunosuppressive medication (e.g. plasmapheresis, biologicals, immune therapy etc.) within the last 6 months o history of thrombotic episodes within the 2 years prior to enrolment o known allergic reactions or other severe reactions to any blood-derived product o known IgA deficiency and anti-IgA serum antibodies o pregnancy (wish). • Conditions that are likely to interfere with: o compliance (legal incompetent and/or incapacitated patients are excluded) or, o evaluation of efficacy (e.g. due to severe pre-existing disability as result of any other disease than IIM). • Lack of informed consent (IC)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary objective is to determine the number of participants with clinical significant improvement (ACR/EULAR Total Improvement Score (TIS) of at least 40) at 9 weeks after start of IVIg treatment. | — |
Secondary
| Measure | Time frame |
|---|---|
| Efficacy of IVIg 1. Time to at least moderate improvement on the TIS 2. Minimal improvement (20%-40%) on each of the 6 IMACS group CSMs 3. Moderate improvement or more (=40%) on each of the 6 IMACS group CSMs 4. The number of deteriorating patients needing rescue therapy 5. Changes in Academic Medical Center Linear Disability Scale (ALDS) 6. Changes in Modified Rankin Scale (MRS) 7. Improvement of dysphagia (if present) 8. Improvement of dynamometric muscle strength 9. Improvement on the Rasch modified MRC Sum Score (Rasch-MRC) 10. Improvement on the EuroQol Group Healt Questionnaire (EQ-5D-5L). 11. The number of participants with significant decrease of abnormalities of muscles and fascia on MRI 12. The number of patients with significant change of size and echo intensity of muscles and fascia on ultrasound (US) 13. Changes in the B-cell repertoire after IVIg treatment 14. RNA and RBM20 expression before and after IVIg treatment 15. Galectin-9 and CXCL10 levels before and after IVIg treatment Safety of IVIg (measured during the total duration of the study) The number of serious adverse events (SAEs) Feasibility of a future trial (assessed at end report) 1. Process: recruitment potential 2. Resources: time and budget estimation 3. Management: exploration of organizational issues | — |
Contacts
Public ContactAnneke Van der Kooi
Amsterdam UMC, University of Amsterdam, Amsterdam Neuroscience
Outcome results
None listed