Monotherapy IVIG Gamunex-C for HMG-CoA Reductase Auto-Antibody Positive Necrotizing Myopathy Treatment (The MIGHT Trial)
Stopped: No Participants Enrolled
United States0Started 2022-05
Plain-language summary
This is a phase 2, pilot, randomized, placebo-controlled trial of Gamunex-C IVIG as mono-therapy for HMGCoA reductase auto-antibody positive (HMGCR) necrotizing myopathy. The trial will test the feasibility and initial efficacy of Gamunex-C IVIG mono-therapy in HMGCR necrotizing myopathy.
Who can participate
Age range
18 Years – 120 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
A subject must meet all of the following inclusion criteria at screening to be eligible for participation in this study:
* Anti-HMGCR positive. Patients will be screened by commercially-available ELISA.
* Age ≥ 18 years
* Demonstrable proximal muscle weakness: score of \<135 on the Proximal Manual Muscle Strength Testing 8-Muscle Group Assessment (MMT-8) (range 0-160).
* Serum creatinine kinase (CK) more than 5 times the upper limit of normal
* Muscle biopsy will not be required for eligibility in order to minimize the time to enrollment and initiation of treatment. Muscle biopsy will be obtained whenever possible as part of the standard of care.
* Subjects must be willing and able to provide written informed consent.
Exclusion Criteria:
A subject meeting any of the following exclusion criteria at screening is NOT eligible for participation in this study:
* Disease duration greater than 36 months.
* Participants taking oral or intravenous glucocorticoids where the dose has changed within 4 weeks of screening.
* Exposure to immunoglobulin treatment (IV, IM, or SubQ) in the prior 3 months
* Exposure to plasma exchange (PEX) in the prior 3 months
* Exposure to other immunosuppressive medications (e.g. methotrexate, leflunomide, azathioprine, mycophenolate mofetil) in the prior 6 months
* Exposure to rituximab or any monoclonal antibody in the prior 12 months
* Currently taking a statin medication
* History of dermatomyositis rash (either biopsy-proven, o…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
ACR/EULAR 2016 Clinical Response Criteria for Myositis