Can immune therapy help rare muscle disorder?
NCT ID NCT06599697
First seen Jun 27, 2026 · Last updated Aug 06, 2026 · Updated 2 times
Summary
This phase 2 trial tests whether intravenous immunoglobulin (IVIG) can help people with a rare autoimmune muscle disease called anti-HMGCR immune-mediated necrotizing myopathy. Twelve participants will receive either IVIG or a placebo every 4 weeks for 12 weeks, then all may receive IVIG in an open-label extension. The main goal is to see if IVIG lowers muscle enzyme levels and is safe.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Intravenous immunoglobulin (IVIG)
- What this could lead to
- If it works, this could point toward a treatment for a rare muscle disease that causes weakness and damage.
- What could go wrong
- This is a very small early-phase trial with only 12 people, so results may not apply widely. IVIG can cause side effects like headache, fever, or allergic reactions.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 12 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Oct 2025
- Expected to finish
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Jun 2027
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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16 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
4 sites. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Johns Hopkins University
NOT_YET_RECRUITINGBaltimore, Maryland, 21218, United States
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University of Alabama at Birmingham
RECRUITINGBirmingham, Alabama, 35233, United States
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University of Pittsburgh
RECRUITINGPittsburgh, Pennsylvania, 15260, United States
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University of Texas Health Science Center at Houston
RECRUITINGHouston, Texas, 77030, United States
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University of Washington
ACTIVE_NOT_RECRUITINGSeattle, Washington, 98195, United States