The MIGHT Trial - An Exploratory Clinical Trial of IVIG in Anti-HMGCR Immune Mediated Necrotizing Myopathy
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Intravenously administered pooled human immunoglobulin (IVIG).
- Who it may be relevant to
- Registry conditions: Anti-3-hydroxy-3-methylglutaryl-CoA Reductase (HMGCR) Immune-Mediated Necrotizing Myopathy. Basic parameters: from 16 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
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)
Overview
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.
Interventions
- Biological Intravenously administered pooled human immunoglobulin (IVIG)
IVIG 2g/kg every 4 weeks for 12 weeks (3 doses)
Primary outcome measures
- Percentage change in serum creatine kinase (CK) [Time frame: Week 0 to 12]
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 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Double blind
- Primary purpose
- Treatment
Study locations
United States · 5 centers
- University of Alabama at Birmingham — Birmingham
- Johns Hopkins University — Baltimore
- University of Pittsburgh — Pittsburgh
- University of Texas Health Science Center at Houston — Houston
- University of Washington — Seattle
Identifiers
NCT: NCT06599697 · 300013467 · 1R21AR083566-01A1