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Myasthenia GravisPhase 1NCT06371040

Safety and Efficacy of CD19-BCMA Targeted CAR-T Therapy for Refractory Generalized Myasthenia Gravis

A Phase 1 study testing CD19-BCMA Targeted CAR-T Dose 1 and CD19-BCMA Targeted CAR-T Dose 2 in people with Myasthenia Gravis, run by Ting Chang, MD 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
Search all trials

Trial summary

This study is a single-center, open-label, single-arm, dose-exploration study to evaluate the safety and preliminary effectiveness of CD19-BCMA CAR-T in the treatment of refractory, generalized myasthenia gravis. The study is a dose escalation trial in adult, refractory, systemic MG patients. Read more about this study

Who is it for?
Ages 18 to 80 years
Conditions the study is for
Myasthenia Gravis
What is being tested?
CD19-BCMA Targeted CAR-T Dose 1 and CD19-BCMA Targeted CAR-T Dose 2
Could I receive a placebo?
No, everyone receives an active treatment
How many people
9 participants
Where
1 siteChina
Study dates
12 Jul 2024to 1 Dec 2026
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 80 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Study participants will be selected for this study only if they meet all of the following criteria:
  • Age ≥18 years old and ≤80 years old;
  • The subject signs the informed consent form, is willing and able to comply with the protocol, complete the research assessment and return for follow-up;
  • To be diagnosed as a patient with systemic MG, the patient is required to have positive myasthenia-related antibodies (AChR-Ab, Musk-Ab or LRP4) on the basis of typical myasthenic symptoms;
  • Evaluated by the researcher as refractory MG. Refractory MG is defined as:
  • Treatment failed after receiving at least 2 immunosuppressants
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  • Definition of treatment failure: 1) Persistent weakness and impairment of daily activities; 2) MG aggravation and/or crisis during treatment; 3) Intolerance to immunotherapy due to side effects or comorbidities;
  • Repeated plasma exchange (PE) or intravenous immune globulin (IVIg) treatment is required to control symptoms;
  • The researchers believe that despite the current routine immunotherapy for patients, MG still imposes a large functional burden on patients.
  • MGFA classification IIa~IVa at screening and baseline;
  • QMGS score ≥11 points or MG-ADL score ≥5 points at screening and baseline, of which the eye score accounts for no more than 50%;
  • Male study participants must agree to take contraceptive measures during the treatment period and within 1 year after receiving study treatment, and are prohibited from donating sperm throughout the study period;
  • If you are a woman of childbearing potential (WOCBP), you must agree to take contraceptive measures during treatment and for at least 1 year after receiving study treatment. Participants must have a negative serum pregnancy test result during screening; a negative urine pregnancy test result must be confirmed before receiving CART for the first time.

Who cannot join

  • Prior to screening and the baseline visit, study participants will not be eligible for inclusion in the study if they meet any of the following criteria:
  • The researcher believes that there is any medical or mental condition that may harm the research participant or affect the research participant's ability to participate in this study; or any condition that the researcher believes is related to poor compliance;
  • Women who are lactating or pregnant, or women who plan to become pregnant at any time within 12 months after receiving CART treatment, or who have a history of spontaneous abortion or induced abortion within 4 weeks before screening;
  • Study participants have clinically relevant active infections (such as sepsis, pneumonia or abscess) or serious infections (resulting in hospitalization or requiring antibiotic treatment) within 4 weeks before screening;
  • thymoma that underwent thymectomy within 6 months before baseline or was planned to undergo thymectomy during the study, or required chemotherapy and/or radiotherapy at any time;
  • Investigator participants have received live attenuated vaccine vaccination within 8 weeks before screening; or plan to receive live vaccine vaccination within 8 weeks after treatment;
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  • Study participants have received rituximab treatment within 6 months before screening;
  • Have received tocilizumab or eculizumab treatment within 3 months before screening;
  • Have received intravenous human immunoglobulin, plasma exchange, or immunotherapy within 4 weeks before screening;
  • Those with known serious underlying diseases, such as liver and kidney damage, blood diseases, previous severe cardiovascular disease, severe hypertension, diabetes, and poor blood pressure and blood sugar control;
  • Unresected thymoma (Note: Subjects with benign thymoma resected more than one year before screening are eligible. Benign is defined as no known metastasis on pathological examination and no intracystic or extracystic Extension. Imaging studies must be performed during the screening period to assess thymic status).
  • Any of the following laboratory abnormalities occur during the screening period (one repeat measurement can be performed during the screening period before randomization to confirm the results)
  • Elevated liver enzymes (aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > 3 times the upper limit of normal (ULN)).
  • Total bilirubin>1.5 times ULN
  • Estimated glomerular filtration rate (eGFR) <45 mL/min/1.73 m2
  • Abnormal PT or INR, or prolonged APTT >1.5 times ULN
  • Neutrophil count <1000cell/ul
  • Platelet count <50000/mm3
  • Hemoglobin<8.0g/dl
  • Those with a high-risk history of tuberculosis infection or acquired tuberculosis infection;
  • Known immunodeficiency diseases, including human immunodeficiency virus (HIV) infection;
  • Positive for hepatitis B surface antigen (HBsAg) during the screening period;
  • Receive blood transfusion treatment 4 weeks before screening or during the screening period;
  • Symptoms worsen rapidly during the lead-in period and enter crisis or pre-crisis state (MGFA IVb-V)
  • Other circumstances in which the researcher deems it inappropriate to participate in the study.

Trial locations and contacts

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

Zhe Ruan contact

8618682932643

ruanzhe573291596@126.com

China1 site, 1 recruiting

  • Tangdu Hospital, The Fourth Military Medical University

    Xi'an, Shaanxi, 710038

    Ting Chang, 02984778845, changting1981@163.com

    Zhe Ruan, ruanzhe573291596@126.com

    Recruiting

Other trials in China

About this study

Official title: Evaluate the Safety and Efficacy of CD19-BCMA Targeted CAR-T Therapy for Refractory, Generalized Myasthenia Gravis: A Single-center, Open-label, Single-arm, Dose-finding Study

This study is a single-center, open-label, single-arm, dose-exploration study to evaluate the safety and preliminary effectiveness of CD19-BCMA CAR-T in the treatment of refractory, generalized myasthenia gravis. The study is a dose escalation trial in adult, refractory, systemic MG patients. The Keyboard method will be used to perform dose escalation to explore the maximum tolerated dose (MTD). A total of 12 MG patients who meet the inclusion criteria are expected to be recruited.

Being tested
CD19-BCMA Targeted CAR-T Dose 1, CD19-BCMA Targeted CAR-T Dose 2
Keywords
Generalized Myasthenia Gravis, CAR-T Therapy
Medical subject headings
Myasthenia Gravis

What participants receive

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

  • CD19-BCMA Targeted CAR-T Dose 1Drug

    5.0 e5/ kg CD19-BCMA CAR-T positive T cells

  • CD19-BCMA Targeted CAR-T Dose 2Drug

    1.5 e6/ kg CD19-BCMA CAR-T positive T cells

  • CD19-BCMA Targeted CAR-T Dose 2Drug

    5 e6/ kg CD19-BCMA CAR-T positive T cells

Study groups in NCT06371040
Study groupRoleWhat they receive
CD19-BCMA Targeted CAR-TExperimentalNot described
Receives: Drug: CD19-BCMA Targeted CAR-T Dose 1; Drug: CD19-BCMA Targeted CAR-T Dose 2

What is being measured

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

  1. Frequency, type, and severity of adverse events

    Frequency, type, and severity of adverse events (according to the National Cancer Institute Common Terminology Criteria for Adverse Events CTCAE V5.0) occurring within 4 weeks after CD19-BCMA CAR-T infusion

    Measured: From Baseline (Day 1) to Safety Follow-Up Visit (up to 4 weeks)

Secondary outcomes (11)

  1. Frequency, type, and severity of abnormal laboratory indicators related to treatment

    Frequency, type, and severity of abnormal laboratory indicators related to treatment (according to the National Cancer Institute Common Terminology Criteria for Adverse Events CTCAE V5.0)

    Measured: From Baseline (Day 1) up fo Follow-Up (up to 24 weeks)

  2. Changes of blood pressure

    Measured: From Baseline (Day 1) up fo Follow-Up (up to 24 weeks)

  3. Changes of pulse rate

    Measured: From Baseline (Day 1) up fo Follow-Up (up to 24 weeks)

  4. Changes of weight

    Measured: From Baseline (Day 1) up fo Follow-Up (up to 24 weeks)

  5. Changes of Myasthenia Gravis Activities of Daily Living (MG-ADL) scores

    Changes in MG-ADL scores [0-24 point] from baseline at 24 weeks after CD19-BCMA CAR-T infusion

    Measured: From Baseline (Day 1) up fo Follow-Up (up to 24 weeks)

  6. Changes of Quantitative Myasthenia Gravis (QMG) scores

    Changes in QMG scores [0-39 point] from baseline at 24 weeks after CD19-BCMA CAR-T infusion

    Measured: From Baseline (Day 1) up fo Follow-Up (up to 24 weeks)

  7. Changes of Myasthenia Gravis Composite (MGC) scores

    Changes in MGC scores [0-50 point] from baseline at 24 weeks after CD19-BCMA CAR-T infusion

    Measured: From Baseline (Day 1) up fo Follow-Up (up to 24 weeks)

  8. Proportion of subjects who achieved improvement

    Proportion of subjects who achieved improvement at 24 weeks after CD19-BCMA CAR-T infusion and sustained it for at least 4 weeks (clinical improvement difined as a ≥2-point reduction in the total MG-ADL score [0-24 point])

    Measured: From Baseline (Day 1) up fo Follow-Up (up to 24 weeks)

  9. Time to achieve clinical improvement

    Time to achieve clinical improvement (clinical improvement difined as a ≥2-point reduction in the total MG-ADL score [0-24 point])

    Measured: From Baseline (Day 1) up fo Follow-Up (up to 24 weeks)

  10. Changes of myasthenia gravis-specific autoantibody titers

    Changes from baseline in myasthenia gravis-specific autoantibody titers over 24 weeks after CD19-BCMA CAR-T infusion

    Measured: From Baseline (Day 1) up fo Follow-Up (up to 24 weeks)

  11. Changes of immunoglobulins

    Changes in immunoglobulins (IgG, IgM, IgA, IgE) within 24 weeks after CD19-BCMA CAR-T infusion

    Measured: From Baseline (Day 1) up fo Follow-Up (up to 24 weeks)

Timeline

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

  1. First posted17 Apr 2024
  2. Study start12 Jul 2024
  3. Primary completion1 Nov 2025 (estimated)
  4. Study completion1 Dec 2026 (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
Not applicable
Intervention model
Sequential
Model description
CD19-BCMA Targeted CAR-T Dose 1 5.0 e5/ kg CD19-BCMA CAR-T positive T cells CD19-BCMA Targeted CAR-T Dose 2 1.5 e6/ kg CD19-BCMA CAR-T positive T cells CD19-BCMA Targeted CAR-T Dose 2 5 e6/ kg CD19-BCMA CAR-T positive T cells
Masking
None (open label)
Enrolment
9 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06371040
Sponsor's protocol ID
V1.0, CART-20230619
Status last verified
Sep 2025
First submitted
17 Mar 2024
Last updated
15 Sep 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Yes: Experimental data may be shared with the consent of the principal investigator

Publications

  • Ruan Z, Ning F, Zhang W, Li S, Su Y, Tang Y, Cao X, Huang X, Song N, Li Z, Li H, Luan X, Yang N, Li Y, Paul F, Liu Q, Chang T. Single-cell profiling of immune reset in patients with refractory generalized myasthenia gravis receiving autologous CD19/BCMA CAR-T cell therapy. Med. 2026 Apr 10;7(4):101026. doi: 10.1016/j.medj.2026.101026. Epub 2026 Feb 25.

    PubMed 41747738

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing CD19-BCMA Targeted CAR-T Dose 1 and CD19-BCMA Targeted CAR-T Dose 2 in people with Myasthenia Gravis, run by Ting Chang, MD 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 13 inclusion and 25 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 15 Sep 2025. 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?

Contact Zhe Ruan (8618682932643, ruanzhe573291596@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Frequency, type, and severity of adverse events (time frame: From Baseline (Day 1) to Safety Follow-Up Visit (up to 4 weeks)). 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 NCT06371040, which also lists 1 linked publication.

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 15 Sep 2025.

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