Can engineered immune cells tame a debilitating autoimmune disease?
NCT ID NCT06688435
First seen Aug 12, 2026 · Last updated Aug 13, 2026 · Updated 1 time
Summary
This phase 1 trial is testing an experimental therapy called SYS6020 for people with refractory generalized myasthenia gravis, a chronic autoimmune condition that causes severe muscle weakness. SYS6020 uses a patient's own immune cells, engineered to target BCMA, a protein involved in producing the antibodies that drive the disease. The study aims to see if this treatment is safe and tolerable, and whether it can improve symptoms, with participants receiving multiple infusions and being followed for up to five years.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- SYS6020 injection, a personalized CAR-T cell therapy targeting BCMA
- What this could lead to
- If successful, this could offer a new treatment option for people with refractory myasthenia gravis who have not responded to standard therapies.
- What could go wrong
- This is an early-phase trial with a small number of participants, so safety and effectiveness are not yet established. CAR-T therapies can cause serious 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
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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About 60 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Mar 2025
- Expected to finish
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May 2033
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 to 65 years
- 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\) The ages ≥18 and ≤ 70 years old; * 2\) Diagnosed as generalized myasthenia gravis (GMG), the clinical classification of MGFA II-IVa; * 3\) Diagnosed as refractory myasthenia gravis (refractory MG) ; * 4\) QMG score \>11 in the screening period and before apheresis; * 5\) Positive acetylcholine receptor antibody (AChR-Ab) and/or muscle-specific receptor tyrosine kinase (MuSK) antibody in the screening period; * 6\) The daily dose of concomitant glucocorticoid therapy must not exceed 20mg prednisone or equivalent within 7 days prior to apheresis; * 7\) Participants have a thorough understanding of this clinical trial and voluntarily sign a written informed consent form. Exclusion Criteria: * 1\) Have been known to have allergic reactions, hypersensitivity, intolerance or contraindications to SYS6020(including its active ingredient and excipient dextran 40) or the drugs potentially used in the study, or who have had a previous history of severe allergic reactions; * 2\) Participants with major chronic diseases that are not well-controlled and considered to increase the participant's risk potentially by the investigator; * 3\) Participants with other autoimmune diseases that require systemic treatment. Participants with stable autoimmune thyroid diseases who have a normal thyroid function and are at a stable therapeutic dose are allowed to be enrolled. * 4\) Participants with a severe recurrent infection during the screening period, or any active infection that the investigator considers may affect the patient's participation; * 5)Participants with a history of positive HIV; participants with positive HBsAg; participants with positive HBcAb and with HBV-DNA above the measurable limit;(Note: participants with positive HBV-DNA or HCV-RNA results within 6 months prior to ICF are excluded); * 6\) Participants with a history of malignant tumors within the past 5 years or with current active malignant tumors. (Participants with successfully treated localized tumors, as well as participants with WHO histological type A, AB, B1 or B2thymoma, with no evidence of recurrence or metastasis for at least 1 year after complete resection assessed by the investigator , are allowed to be enrolled;) * 7\) Any serious respiratory system disease. * 8\) Participants with a history of serious cardiovascular disease, such as severe cardiac rhythm or conduction abnormalities. * 9\) Abnormal laboratory findings with clinical significance, including ALT, AST\>3\*ULN; Scr\>1.5\*ULN; INR\>1.5\*ULN, and so on. . * 10\) Individuals with potential disease conditions (including laboratory abnormalities) which are considered of clinical significance by the investigator; individuals with alcohol dependence or drug abuse . * 11\) Individuals with a current psychotic disorder that interferes with adherence. * 12\) Participants with a history of primary immunodeficiency disease, organ or hematopoietic stem cell/bone marrow transplantations before screening; or those planning to undergo a transplantation during the trial; * 13\) Participants with a history of ≥ Grade 2 (CTCAE 5.0 standard) bleeding within 30 days before screening, or those requiring long-term continuous treatments with anticoagulant drugs. * 14\) Participants who have received any CAR-T therapy or gene therapy before. * 15\) Participants who have received intravenous injection of human immunoglobulin (IVIG) or plasmapheresis (PE), plasma separation, or hemodialysis within 1 month before apheresis. * 16\) Participants who have used calcineurin inhibitors, or cyclophosphamide or neonatal Fc receptor antagonists within 3 weeks before apheresis. Participants who have used targeted B-cell biological agents such as rituximab within 3 months before apheresis. Participants who started receiving eculizumab treatment within 8 weeks before the first dosing; * 17\) Any situations that the investigator believes that the participant is not suitable for this clinical trial for any other reasons.
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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
1 site. 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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Qilu Hospital, Shandong University
RECRUITINGJinan, China
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