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Myasthenia GravisPhase 1NCT06688435

A Study to Evaluate the Safety and Preliminary Efficacy of SYS6020 CAR T-cells in Patients With Refractory Generalized Myasthenia Gravis

A Phase 1 study testing SYS6020 injection in people with Myasthenia Gravis, run by CSPC ZhongQi Pharmaceutical Technology Co., Ltd. at 1 site in China.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

This study is a single-arm, open, 2-stage (dose-escalation phase and dose-expansion phase), multi-center, phase I clinical trial to evaluate the safety and tolerance of SYS6020 injection in the participants with refractory systemic myasthenia gravis, and determine the recommended dose (RD) for subsequent studies of… Read more about this study

Who is it for?
Ages 18 to 65 years
Conditions the study is for
Myasthenia Gravis
What is being tested?
SYS6020 injection
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
60 participants
Where
1 siteChina
Study dates
4 Mar 2025to 1 May 2033
Phase
Phase 1
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 65 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.

Who cannot join

  • 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;)
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  • 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.

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

China1 site, 1 recruiting

  • Qilu Hospital, Shandong University

    Jinan

    Chuanzhu Yan, 18561811888, chuanzhuyan@163.com

    Recruiting

Other trials in China

About this study

Official title: A Phase I Study to Evaluate BCMA-targeted Chimeric Antigen Receptor T Cell (SYS6020 Injection) in Patients With Refractory Generalized Myasthenia Gravis

This study is a single-arm, open, 2-stage (dose-escalation phase and dose-expansion phase), multi-center, phase I clinical trial to evaluate the safety and tolerance of SYS6020 injection in the participants with refractory systemic myasthenia gravis, and determine the recommended dose (RD) for subsequent studies of the product, and to preliminarily evaluate the clinical efficacy of the product, as well as to explore the pharmacokinetics and immunogenicity of the product in vivo.

The dose-escalation phase and dose-expansion phase include 7 periods, and they are respectively in sequence as follows: the screening period, apheresis period, pre-dosing assessment, SYS6020 injection infusion, DLT observation period, the primary follow-up period (6 months), and the long-term follow-up period (5 years). The DLT observation period is 28 days after receiving SYS6020 injection. The participants will not undergo lymphodepleting chemotherapy.

Read more

The efficacy and safety profile of the participants will be continuously assessed during the trial. Efficacy measurement includes the MG-ADL, QMG, MGC, MG-QoL 15R scale, MGFA clinical classification, and MGFA post-intervention state (MGFA PIS) grading scales, as well as self-antibodies, etc. Safety measurement includes vital signs, physical examination, laboratory tests, cytokines, and ECG, etc. The adverse events and concomitant therapy will be continuously collected during the trial. In addition, during the study period, blood samples will be collected from participants who have received SYS6020 treatment for PK/PD test, and immunogenicity test.

For the dose-escalation phase, 3 to 5 dose levels are proposed to be explored. The Safety Monitoring Committee (SMC) will discuss the safety data and make a decision if the next SYS6020 injection could be initiated or dose-escalation could be initiated. After the completion of the dose-escalation phase, the recommended doses would be determined for dose-expansion phase. For the dose-expansion phase, further safety and efficacy data will be collected among the participants who will receive the recommended dose of SYS6020 injection.

Being tested
SYS6020 injection
Medical subject headings
Myasthenia Gravis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • SYS6020 injectionBiological

    The SYS6020 is an injection of autologous CAR-T cells that have been temporarily transfected with LNP-mRNA targeting BCMA. Before each dosing, the infusion eligibility of SYS6020 is assessed by the investigator, and the eligible participants will receive 3 dosing SYS6020 injection treatment. The dosing interval is 7 days according to the protocol design.

  • SYS6020 injectionBiological

    The SYS6020 is an injection of autologous CAR-T cells that have been temporarily transfected with LNP-mRNA targeting BCMA. Before each dosing, the infusion eligibility of SYS6020 is assessed by the investigator. The eligible participants will receive SYS6020 injection treatment twice weekly for 3 weeks, for a total of 6 doses.

  • SYS6020 injectionBiological

    The SYS6020 is an injection of autologous CAR-T cells that have been temporarily transfected with LNP-mRNA targeting BCMA. Before each dosing, the infusion eligibility of SYS6020 is assessed by the investigator. The eligible participants will receive SYS6020 injection treatment once weekly for 6 weeks, for a total of 6 doses.

Study groups in NCT06688435
Study groupRoleWhat they receive
SYS6020 injection: 2*106CAR+ T-cells per kg(dose level 1)ExperimentalParticipants will undergo leukapheresis to isolate peripheral blood mononuclear cells to manufacture SYS6020 injection, during which no lymphodepleting chemotherapy will be performed. Participants will receive 3 doses treatment of SYS6020 injection, with a dose level of 2*106CAR+ T-cells per kg. If one DLT occurs in three participants, additional 3 ones may be considered to be enrolled after SMC safety data review and make a determination.
Receives: Biological: SYS6020 injection
SYS6020 injection: 6*106CAR+ T-cells per kg(dose level 2)ExperimentalParticipants will undergo leukapheresis to isolate peripheral blood mononuclear cells to manufacture SYS6020 injection, during which no lymphodepleting chemotherapy will be performed. Participants will receive 3 doses treatment of SYS6020 injection, with a dose level of 6*106CAR+ T-cells per kg. If one DLT occurs in three participants, additional 3 ones may be considered to be enrolled after SMC safety data review and make a determination.
Receives: Biological: SYS6020 injection
SYS6020 injection: 1.8*107CAR+ T-cells per kg(dose level 3)ExperimentalParticipants will undergo leukapheresis to isolate peripheral blood mononuclear cells to manufacture SYS6020 injection, during which no lymphodepleting chemotherapy will be performed. Participants receive 3 doses of SYS6020 injection, with a dose level of 1.8*107CAR+ T-cells per kg. If one DLT occurs in three participants, additional 3 ones may be considered to be enrolled after SMC safety data review and make a determination.
Receives: Biological: SYS6020 injection
SYS6020 injection: 3.6*107CAR+ T-cells per kg(dose level 4)ExperimentalParticipants will undergo leukapheresis to isolate peripheral blood mononuclear cells to manufacture SYS6020 injection, during which no lymphodepleting chemotherapy will be performed. Participants receive 3 doses of SYS6020 injection, with a dose level of 3.6*107CAR+ T-cells per kg. If one DLT occurs in three participants, additional 3 ones may be considered to be enrolled after SMC safety data review and make a determination.
Receives: Biological: SYS6020 injection
SYS6020 injection: 5.4*107CAR+ T-cells per kg(dose level 5)ExperimentalParticipants will undergo leukapheresis to isolate peripheral blood mononuclear cells to manufacture SYS6020 injection, during which no lymphodepleting chemotherapy will be performed. Participants receive 3 doses of SYS6020 injection, with a dose level of 5.4*107CAR+ T-cells per kg. If one DLT occurs in three participants, additional 3 ones may be considered to be enrolled after SMC safety data review and make a determination.
Receives: Biological: SYS6020 injection
SYS6020 injection (Expansion stage group A)ExperimentalParticipants will undergo leukapheresis to isolate peripheral blood mononuclear cells to manufacture SYS6020 injection, during which no lymphodepleting chemotherapy will be performed.
Receives: Biological: SYS6020 injection
SYS6020 injection (Expansion stage group B)ExperimentalParticipants will undergo leukapheresis to isolate peripheral blood mononuclear cells to manufacture SYS6020 injection, during which no lymphodepleting chemotherapy will be performed.
Receives: Biological: SYS6020 injection

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. The frequency and the grade of DLT and the incidence of adverse events and serious adverse events. (For the dose-escalation phase)

    For the dose-escalation phase, the primary objective is to evaluate the safety of SYS6020, as measured by the frequency and nature of Dose-limiting toxicity (DLT) and the incidence of all adverse events and serious adverse events. DLT is defined as any adverse event related to SYS6020 that occurs within 28 days after the infusion of SYS6020 and that meets certain criteria.

    Measured: 12 months

  2. Response rate at 6 months after dosing （For the dose-expansion phase)

    Response is defined as a ≥3-point reduction from baseline in the MG-ADL total score at Month 6

    Measured: 6months

Secondary outcomes (14)

  1. The proportion of MG-ADL score sdecline≥2

    The proportion of participants achieving a decline of 2 points or more in MG-ADL from baseline to 6 months and 12 months after dosing (as dose-escalation phase endpoints only);

    Measured: 6 months, 12 months

  2. the proportion of QMG score decline≥2

    The proportion of participants achieving a decline of 2 points or more in QMG from baseline to 6 months and 12 months after dosing (as dose-escalation phase endpoints only);

    Measured: 6 months, 12 months

  3. The proportion of MGC score decline≥3

    The proportion of participants achieving a decline of 3 points or more in MGC from baseline to 6 months and 12 months after dosing (as dose-escalation phase endpoints only); The proportion of participants achieving a decline of 3 points or more in MGC from baseline to 6 months and 12 months after dosing (as dose-expansion phase endpoints only)

    Measured: 6 months, 12 months

  4. The mean change of MG-ADL score and QMG score decline ≥3

    The proportion of participants achieving a decline of 3 points or more in both MG-ADL and QMG from baseline to 6 months and 12 months after dosing (as dose expansion phase endpoints only);

    Measured: 6 months, 12 months

  5. The mean change of QMG score

    the mean change of QMG score from baseline to 6 months and 12 months after dosing (as two-phase joint-endpoints)

    Measured: 6 months, 12 months

  6. The mean change of MG QoL-15R score

    the mean change of MG QoL-15R from baseline to 6 months and 12 months after dosing (as two-phase joint-endpoints)

    Measured: 6 months, 12 months

  7. The mean change of MGC score

    the mean change of MGC from baseline to 6 months and 12 months after dosing (as two-phase joint-endpoints)

    Measured: 6 months, 12 months

  8. The mean change of MGFA clinical classification

    the mean change of MGFA clinical classification from baseline to 6 months and 12 months after dosing (as two-phase joint-endpoints)

    Measured: 6 months, 12 months

  9. The mean change of MGFA PIS grading

    the mean change of MGFA PIS grading from baseline to 6 months and 12 months after dosing (as two-phase joint-endpoints)

    Measured: 6 months, 12 months

  10. The mean change of hand grip strength

    the mean change of hand grip strength from baseline to 6 months and 12 months after dosing (as two-phase joint-endpoints)

    Measured: 6 months, 12 months

  11. The mean change of vital capacity

    the mean change of vital capacity from baseline to 6 months and 12 months after dosing (as two-phase joint-endpoints)

    Measured: 6 months, 12 months

  12. The proportion of participants with concentration titer change of myasthenia gravis-specific autoantibody

    the proportion of participants whose AChR-Ab or MuSK-Ab concentrations titer is declined by ≥50% from baseline to 12 weeks after dosing (as two phase joint-endpoints)

    Measured: 12 weeks

  13. The incidence of AE and SAE

    the incidence of all the adverse events (AE) and serious adverse events (SAE) during 12 months after dosing. (as two phase joint-endpoints)

    Measured: 12 months

  14. the pharmacokinetic parameters of SYS6020 injection

    the concentrations and pharmacokinetic parameters of SYS6020 CAR+ cells, as well as the gene copies of CAR targeting BCMA.

    Measured: 12 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted14 Nov 2024
  2. Study start4 Mar 2025
  3. Primary completion1 Dec 2028 (estimated)
  4. Study completion1 May 2033 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Model description
This is an open-label, two-phase (dose-escalation and dose-expansion), multicenter, phase I clinical study. The dose-escalation phase adopts a sequential design model, including 5 arms for 5 dose level. The arms of dose-expansion phase depends on the number of recommended dose, which may be 1-2 recommended dose.
Masking
None (open label)
Enrolment
60 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06688435
Sponsor's protocol ID
SYS6020-004
Status last verified
Aug 2026
First submitted
6 Nov 2024
Last updated
12 Aug 2026
Results posted
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing SYS6020 injection in people with Myasthenia Gravis, run by CSPC ZhongQi Pharmaceutical Technology Co., Ltd. at 1 site in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 12 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

The frequency and the grade of DLT and the incidence of adverse events and serious adverse events. (For the dose-escalation phase) (time frame: 12 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06688435.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Aug 2026.

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