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Can engineered immune cells stop a rare muscle disease?

NCT ID NCT07752264

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Aug 07, 2026 · Last updated Aug 07, 2026

Summary

This early-stage trial is testing whether a personalized cell therapy called Eque-cel can safely and effectively treat people with refractory immune-mediated necrotizing myopathy (IMNM), a severe autoimmune condition that causes muscle weakness and damage. The therapy involves reprogramming a patient's own immune cells to target and eliminate specific cells that drive the disease. The study will monitor side effects and measure improvements in muscle strength and function.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Equecabtagene autoleucel (Eque-cel), a personalized CAR-T cell therapy that targets and eliminates BCMA-expressing cells to reset the immune system.
What this could lead to
If successful, this could lead to a new treatment option for people with refractory immune-mediated necrotizing myopathy, potentially offering long-term remission without lifelong immunosuppression.
What could go wrong
This is an early-phase, small trial (27 participants) focused on safety and initial efficacy. CAR-T therapies carry risks like cytokine release syndrome and neurological side effects, and the treatment may not work for everyone.

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 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 27 people

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

Expected to start

Aug 2026

An estimate. Start dates often move.

Expected to finish

Aug 2043

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 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: * 1\. At the time of signing the informed consent form, the age is ≥ 18 years old. * 2.Based on the 2016 ENMC classification criteria, the clinical diagnosis is IMNM. * 3.At the time of screening, the anti-SRP antibody or anti-HMGCR antibody is positive. * 4\. Refractory IMNM that failed or relapsed after standard treatment, and the disease is still active at the time of screening. * 5.The subjects must have appropriate organ functions. * 6.The subjects and their spouses agree to take effective contraceptive measures (excluding safe period contraception) from the time of the subject signing the informed consent form until one year after the CAR-T cell infusion. * 7\. The subjects must agree to sign or personally write and present the informed consent form approved by the ethics committee before starting any screening procedures. Exclusion Criteria: * 1\. Having end-stage myositis with involvement of terminal organs may pose additional risks or interfere with the study assessment. * 2.Presence of irreversible muscle involvement and/or severe atrophy may bring additional risks or interfere with the study assessment. * 3.Uncontrolled interstitial lung disease or any other uncontrolled manifestations of IIM, as judged by the investigator, may require the use of prohibited drugs during the study period. * 4\. Diagnosed with other inflammatory or non-inflammatory myopathies. * 5.Any other known autoimmune disease that the investigator considers to interfere with the accurate assessment of clinical symptoms of IMNM or place the patient at excessive risk. * 6\. Those with a history of solid organ transplantation. * 7\. History of autologous or allogeneic stem cell transplantation. * 8\. Previous history of BCMA-targeted drug treatment. * 9.Having occurred or planned to undergo major surgery or surgical treatment within 4 weeks before enrollment or within 12 weeks after infusion. * 10\. Known primary immunodeficiency (congenital or acquired). * 11\. Having a clear history of mental disorder or a history of substance abuse for mental disorders that cannot be quit. * 12.The subject has uncontrollable active fungal, viral, bacterial or other infections (having persistent infection-related signs/symptoms, without improvement after appropriate anti-infection treatment) or infections requiring intravenous anti-infection drug treatment. * 13.Positive hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) and abnormal peripheral blood hepatitis B virus (HBV) DNA test (defined as HBV DNA quantification ≥ 100IU/ml or ≥ 1000 copies /ml or above the normal reference range of the testing center or positive for qualitative HBV DNA detection) ; hepatitis C virus (HCV) antibody positive and peripheral blood HCV RNA positive (defined as ≥ 1000 IU/mL); human immunodeficiency virus (HIV) antibody positive; cytomegalovirus (CMV) DNA positive (defined as ≥ 1000 IU/mL); Treponema pallidum specific antibody positive and positive for the rapid plasma reagin test for syphilis. * 14.Severe heart disease: including but not limited to unstable angina pectoris and/or myocardial infarction within 12 months of the screening period, any congestive heart failure (NYHA classification ≥ III), and a history of severe arrhythmia. * 15.Severe asthma or chronic obstructive pulmonary disease (COPD). Note: Mild or moderate asthma or COPD patients with stable conditions, after assessment and approval by the investigator and the sponsor, can be enrolled. * 16.Acute cerebrovascular disease events occurred within 6 months before enrollment, including transient ischemic attack or stroke history. * 17.Any serious and/or uncontrolled comorbid diseases as determined by the investigator to potentially interfere with the study assessment. * 18.Malignant tumors within 5 years before screening, excluding cured cervical carcinoma in situ, basal cell or squamous epithelial cell skin cancer, locally advanced prostate cancer after radical surgery, breast duct carcinoma in situ after radical surgery, or thyroid papillary carcinoma after radical surgery. * 19\. Known history of allergy to cyclophosphamide, fludarabine, components of Eque-cel injection or supportive drugs required for toxicity management of CAR-T cell therapy (such as tocilizumab). * 21.Pregnant or lactating women. * 21\. Other situations as determined by the investigator to be unsuitable for enrollment.

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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. 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

  • China-Japan Friendship Hospital

    Beijing, Beijing Municipality, 100029, China

  • Huashan Hospital of Fudan University

    Shanghai, Shanghai Municipality, 200040, China

  • The First Affiliated Hospital of Zhengzhou University

    Zhengzhou, Henan, 450052, China

  • The Second Affiliated Hospital, Zhejiang University School of Medicine

    Hangzhou, Zhejiang, 310009, China