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Can immune therapy help rare muscle disorder?

NCT ID NCT06599697

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 12 people

The number the study aims to enrol. It can still change while the study runs.

Started

Oct 2025

Expected to finish

Jun 2027

An estimate. End dates often move.

Lead sponsor

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

16 years and older

Sex

Anyone

Healthy volunteers

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

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.

  1. The places running it

    4 sites. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. 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

  • Johns Hopkins University

    NOT_YET_RECRUITING

    Baltimore, Maryland, 21218, United States

  • University of Alabama at Birmingham

    RECRUITING

    Birmingham, Alabama, 35233, United States

  • University of Pittsburgh

    RECRUITING

    Pittsburgh, Pennsylvania, 15260, United States

  • University of Texas Health Science Center at Houston

    RECRUITING

    Houston, Texas, 77030, United States

  • University of Washington

    ACTIVE_NOT_RECRUITING

    Seattle, Washington, 98195, United States