MIGHT — IVIG for anti‑HMGCR immune‑mediated necrotizing myopathy
The MIGHT Trial - An Exploratory Clinical Trial of Intravenous Immunoglobulin (IVIG) in Anti-3-Hydroxy-3-Methylglutaryl-CoA Reductase (HMGCR) Immune Mediated Necrotizing Myopathy (IMNM)
This will test whether intravenous immunoglobulin (IVIG) helps people aged 16 and older with active anti‑HMGCR immune‑mediated necrotizing myopathy.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 12 (estimated) |
| Ages | 16 Years and up |
| Sex | All |
| Sponsor | University of Alabama at Birmingham Academic / other |
| Drugs / interventions | Rituximab, methotrexate, cyclophosphamide |
| Locations | 5 sites (Birmingham, Alabama and 4 other locations) |
| Trial ID | NCT06599697 on ClinicalTrials.gov |
What this trial studies
This randomized, double‑blind, placebo‑controlled phase 2 trial will compare IVIG 2 g/kg versus saline placebo given at weeks 0, 4, and 8 in 12 participants with active anti‑HMGCR IMNM. Primary efficacy and co‑primary safety/tolerability endpoints are assessed at week 12, and all participants may join an open‑label extension receiving IVIG at weeks 12, 16, and 20 with a final visit at week 24. Key inclusion criteria include anti‑HMGCR antibody positivity, serum CK >5× upper limit of normal, MMT‑8 <142, disease duration under 36 months, and residence in a participating state; major exclusions include prior IVIG for IMNM, current statin use, recent biologic/plasma exchange, or high‑dose glucocorticoids. The small planned sample size (12, anticipating ~10 completers) makes this an exploratory effort to generate preliminary safety and efficacy signals.
Who should consider this trial
Good fit: People aged 16 and older with active anti‑HMGCR IMNM (anti‑HMGCR antibody positive, CK >5× ULN, MMT‑8 <142), disease duration under 36 months, not on high‑dose glucocorticoids or prior IVIG, and who reside in a participating state are ideal candidates.
Not a fit: Patients with long-standing disease (>36 months), prior IVIG for IMNM, current statin use, significant respiratory or swallowing dysfunction, or on high-dose glucocorticoids are unlikely to be eligible and may not benefit from this specific protocol.
Why it matters
Potential benefit: If successful, IVIG could improve muscle strength and reduce muscle enzyme levels, offering a new treatment option for people with anti‑HMGCR IMNM.
How similar studies have performed: Case series and observational reports suggest IVIG can help immune‑mediated necrotizing myopathies including anti‑HMGCR cases, but randomized controlled evidence in this population is limited.
Eligibility criteria
Show full inclusion / exclusion 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 of angina pectoris, myocardial infarction, transient ischemic attack, or stroke \< 12 months prior to screening * Females of child-bearing potential who are pregnant, breastfeeding, or are unwilling to practice a highly effective method of contraception during the study * Wells Criteria for DVT score of 2 or more at screening * Wells Criteria for PE score of 4 or more at screening * Weight \>120kg * History of cancer (excluding non-melanomatous skin cancer) \< 5 years prior to screening * History of pulmonary embolism or deep venous thromboembolism \< 3 years prior to screening * History of hyperviscosity or hypercoagulable state * Currently receiving anti-coagulation therapy (vitamin K antagonists, non-vitamin K oral anticoagulants \[e.g. dabigatran, rivaroxaban, apixaban\], parenteral anticoagulants \[e.g. fondaparinux\]. Note that oral anti-platelet agents are allowed (e.g. aspirin, clopidogrel, ticlopidine). * Glomerular filtration rate (GFR) \<60mL/min at the time of screening * Any medical condition which, in the investigator's judgment, makes participation in the clinical trial unadvisable or which would interfere with evaluation of the study treatment.
Where this trial is running
Birmingham, Alabama and 4 other locations
- University of Alabama at Birmingham — Birmingham, Alabama, United States (Recruiting)
- Johns Hopkins University — Baltimore, Maryland, United States (Not_yet_recruiting)
- University of Pittsburgh — Pittsburgh, Pennsylvania, United States (Not_yet_recruiting)
- University of Texas Health Science Center at Houston — Houston, Texas, United States (Active_not_recruiting)
- University of Washington — Seattle, Washington, United States (Active_not_recruiting)
Study contacts
- Principal investigator: James Andrews, MD — University of Alabama at Birmingham
- Study coordinator: James Andrews, MD
- Email: jaandrews@uabmc.edu
- Phone: (205) 934-1564
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.