Can engineered immune cells stop a rare muscle disease?
NCT ID NCT07752264
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
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Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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About 27 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Aug 2026
An estimate. Start dates often move.
- Expected to finish
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Aug 2043
An estimate. End dates often move.
- Lead sponsor
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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
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18 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: * 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.
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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. 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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China-Japan Friendship Hospital
Beijing, Beijing Municipality, 100029, China
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Huashan Hospital of Fudan University
Shanghai, Shanghai Municipality, 200040, China
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The First Affiliated Hospital of Zhengzhou University
Zhengzhou, Henan, 450052, China
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The Second Affiliated Hospital, Zhejiang University School of Medicine
Hangzhou, Zhejiang, 310009, China