The MIGHT Trial - An Exploratory Clinical Trial of IVIG in Anti-HMGCR Immune Mediated Necrotizing Myopathy
Trial Parameters
Brief Summary
This is a randomized, placebo-controlled, double blinded phase 2 exploratory clinical trial of intravenously administered pooled human immunoglobulin (IVIG) in anti-3-hydroxy-3-methylglutaryl-CoA reductase (HMGCR) immune mediated necrotizing myopathy (IMNM). Planned enrollment is 12 individuals with active anti-HMGCR IMNM meeting inclusion and exclusion criteria. Assuming 20% drop-out, the investigators anticipate 10 participants will complete all study assessments. Enrolled participants will be randomized 1:1 to either IVIG 2g/kg or placebo (0.9% sodium chloride at equivalent volume) at weeks 0, 4, and 8. The primary efficacy and co-primary safety and tolerability endpoints will be assessed at week 12. After the randomized phase of the trial, all participants will be offered to continue on to an open-label extension phase in which participants will receive IVIG at weeks 12, 16, and 20. Participants will then return at week 24 for a final non-infusion visit to reassess safety, tolerability, and efficacy outcome.
Eligibility Criteria
Inclusion Criteria: * Age \> 16 years * Anti-HMGCR antibody positive * MMT-8 score \< 142 (range 0-160) * Serum CK \> 5x upper limit of normal * Anti-HMGCR IMNM disease duration \< 36 months at screening * No moderate or severe respiratory or swallowing dysfunction due to anti-HMGCR IMNM at screening * No history of dermatomyositis rash * Must reside in a state with a participating research site Exclusion Criteria: * Oral glucocorticoid (GC) daily dose \> 15mg at screening * Change in oral GC dose \< 2 weeks prior to screening * Prior IVIG treatment for anti-HMGCR IMNM -\>1 oral conventional synthetic DMARD (e.g. methotrexate, mycophenolate mofetil, azathioprine) use at screening * Change in concomitant DMARD dose \< 4 weeks prior to screening * Rituximab \< 6 months prior to screening * Plasma exchange, cyclophosphamide, or biologic immunosuppressive medication \< 3 months prior to screening * Use of statin medication at screening * History of anaphylactic reaction to IVIG * History o