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Myasthenia Gravis, GeneralizedPhase 1NCT06958939

The Safety and Efficacy of S103 in the Treatment of Refractory Generalized Myasthenia Gravis

A Phase 1 study testing Dose level 1 group, Dose level 2 group and Dose level 3 group in people with Myasthenia Gravis, Generalized, run by Ting Chang, MD at 1 site in China.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

This study is a single-center, open-label, single-arm, dose-exploration study to evaluate the safety and preliminary effectiveness of BCMA CAR-T（S103） 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, Generalized
What is being tested?
Dose level 1 group, Dose level 2 group and Dose level 3 group
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
1 siteChina
Study dates
1 May 2025to 30 Apr 2028
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

  • Age ≥18 years, ≤80 years;
  • MGFA classification type IIa-IVa;
  • Voluntary participation in the study: understanding and awareness of the study and voluntary signing of the informed consent form;
  • Meeting the diagnosis of myasthenia gravis;
  • Assessed by the investigator as meeting the diagnostic criteria for refractory MG: fulfilling one of the following 4 conditions:
  • After adequate dose and duration of at least 2 conventional immunotherapies (including steroid and non-steroid immunosuppressants), the post-intervention status (PIS) is unchanged or worsened.
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  • After adequate dose and duration of at least 2 conventional immunotherapies, the PIS is improved, but the MG-ADL score remains
  • 6 for at least 6 months.
  • After adequate dose and duration of at least 2 conventional immunotherapies, the PIS is remission or improvement, but during regular tapering of immunotherapy, there are still ≥2 exacerbations per year (MG-ADL score ≥6).
  • The investigator considers that, despite current conventional immunotherapy, MG still imposes a significant functional burden on the patient.
  • MG-ADL score ≥6 or QMG score ≥11 at screening and baseline, with ocular muscle score less than 50% of the total score.

Who cannot join

  • Any medical or psychiatric condition that the investigator deems may endanger the study participant or affect their ability to participate in the study; or any condition the investigator considers associated with poor compliance;
  • Females who are breastfeeding or pregnant, or plan to become pregnant at any time during the 12-month period after receiving CAR-T therapy, or have a history of spontaneous or induced abortion within 4 weeks prior to screening;
  • Study participants with clinically relevant active infections (e.g., sepsis, pneumonia, or abscess) or severe infections (requiring hospitalization or antibiotic treatment) within 4 weeks prior to screening;
  • Thymectomy within 6 months prior to baseline or planned thymectomy during the study period, or thymoma requiring chemotherapy and/or radiotherapy at any time;
  • Study participants who received live attenuated vaccines within 8 weeks prior to screening; or plan to receive live vaccines within 8 weeks after treatment;
  • Study participants who received prior rituximab treatment within 6 months before screening;
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  • Treatment with tocilizumab, eculizumab, or efgartigimod within 3 months prior to screening;
  • Intravenous immunoglobulin, plasma exchange, or immunotherapy within 4 weeks prior to screening;
  • Known severe underlying diseases, such as liver or kidney dysfunction, hematologic disorders, prior severe cardiovascular diseases, severe hypertension, diabetes, or poorly controlled blood pressure or blood glucose;
  • Unresected thymoma;
  • Rapid symptom deterioration during the lead-in period, progressing to crisis or pre-crisis state (MGFA IVb-V);
  • Other conditions deemed by the investigator as unsuitable for study participation.

Trial locations and contacts

1 site in 1 country.

China1 site

  • The Second Affiliated Hospital of Air Force Medical University

    Xi'an, Shaanxi, 710038

Other trials in China

About this study

Official title: A Single-center, Single-arm, Dose-exploration Study Evaluating the Safety and Efficacy of SENL103 Autologous T-cell Injection (S103) in the Treatment of Refractory Generalized Myasthenia Gravis

This study is a single-center, open-label, single-arm, dose-exploration study to evaluate the safety and preliminary effectiveness of BCMA CAR-T（S103） in the treatment of refractory, generalized myasthenia gravis. The study is a dose escalation trial in adult, refractory, systemic MG patients. A total of 6-24 MG patients who meet the inclusion criteria are expected to be recruited.

Being tested
Dose level 1 group, Dose level 2 group, Dose level 3 group, Dose level 4 group
Medical subject headings
Myasthenia Gravis

What participants receive

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

  • Dose level 1 groupDrug

    1.0 × 10e6/kg S103 CAR-T cells

  • Dose level 2 groupDrug

    2.0 × 10e6/kg S103 CAR-T cells

  • Dose level 3 groupDrug

    4.0 × 10e6/kg S103 CAR-T cells

  • Dose level 4 groupDrug

    5.0 × 10e6/kg S103 CAR-T cells

Study groups in NCT06958939
Study groupRoleWhat they receive
S103 CAR-TExperimentalThis study adopted a single-arm design, with all patients receiving sequential administration of different doses of S103 CAR-T
Receives: Drug: Dose level 1 group; Drug: Dose level 2 group; Drug: Dose level 3 group; Drug: Dose level 4 group

What is being measured

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

  1. Dose-limiting toxicity

    DLT is defined as the following conditions that occur during the DLT evaluation period, which are not attributed to disease progression or disease-related processes, but are related to the study drug:

    1. All treatments with grade 4 or 5 cytokine release syndrome (CRS);
    2. Grade ≥3 immune effector cell-associated neurotoxicity syndrome (ICANS) possibly related to CAR-T therapy in all treatments;
    3. Grade 3 hematologic toxicity lasting more than 7 days, not caused by the underlying disease (excluding lymphocyte count decrease due to lymphodepletion);
    4. Grade ≥3 organ toxicity reactions (cardiac, skin, gastrointestinal, hepatic, pulmonary, renal/genitourinary) possibly related to CAR-T therapy in all treatments, with cardiac toxicity and renal toxicity reactions ≥ grade 2 considered as DLT;
    5. Treatment-related death;
    6. Other toxicities determined to be DLT after discussion by the SRC.

    Measured: Day 28

Secondary outcomes (12)

  1. Proportion of subjects who reach MSE (ADL≤1 score, for 4 weeks) at 3 months and 6 months after infusion

    Myasthenia Gravis Activities of Daily Living (MG-ADL) is a questionnaire that includes 8 items: ptosis, diplopia, speech, chewing, swallowing, breathing, and upper and lower limb functions. The scale uses a 4-level scoring system, with a total score of 0-24, and higher scores indicate more severe disability, with an improvement of 2 points or more suggesting clinical improvement.

    Measured: 3 month, 6 month

  2. Changes in ADL score relative to baseline at 28 days, 3 months, 6 months after infusion

    Myasthenia Gravis Activities of Daily Living (MG-ADL) is a questionnaire that includes 8 items: ptosis, diplopia, speech, chewing, swallowing, breathing, and upper and lower limb functions. The scale uses a 4-level scoring system, with a total score of 0-24, and higher scores indicate more severe disability, with an improvement of 2 points or more suggesting clinical improvement.

    Measured: 28 days, 3 months, 6 months

  3. Changes in QMG score relative to baseline at 28 days, 3 months, 6 months after infusion

    Quantitative Myasthenia Gravis scale(QMG), mainly measures the patient's muscle strength and endurance. The QMG assessment includes diplopia, ptosis, facial muscle strength, swallowing, articulation, arm lift on both sides, percentage of predicted forced vital capacity (FVC), head lift, grip strength on both hands, and leg lift on both sides in a supine position, with 13 items in total. Each item is scored from normal (0) to severe (3), with a total score ranging from 0 to 39, and a higher score indicates a more severe disease. A total score change of more than 3 points is considered clinically significant.

    Measured: 28 days, 3 months, 6 months

  4. Changes in MGC score relative to baseline at 28 days, 3 months, 6 months after infusion

    MG Composite (MG-C) combines physician-reported and patient-reported entries. The scale assesses 10 items, with talking, chewing, and swallowing indices self-assessed by study participants, with total scores ranging from 0 to 50. For most patients, a score change of ≥3 points is associated with clinically significant symptom change.

    Measured: 28 days, 3 months, 6 months

  5. Changes in QOL15 score relative to baseline at 28 days, 3 months, 6 months after infusion

    15-item MG-specific QOL scale, MG-QOL15, is a short survey questionnaire completed by participants, with 15 items covering three aspects: physical, psychological, and social. It aims to assess the "patient's perspective" of "quality of life" related to MG, using a 5-level scoring method (from mild to severe, 0-4 points), with a maximum score of 60 points, and higher scores indicating poorer quality of life.

    Measured: 28 days, 3 months, 6 months

  6. Median time to re-infusion to achieve MSE status

    Measured: 6 months

  7. Changes of blood pressure

    Measured: 28 days, 3 months, 6 months

  8. Changes of pulse rate

    Measured: 28 days, 3 months, 6 months

  9. Changes of weight

    Measured: 28 days, 3 months, 6 months

  10. Changes in myasthenia gravis-specific autoantibody titers relative to baseline at 28 days, 3 months, 6 months after infusion

    Measured: 28 days, 3 months, 6 months

  11. Changes in immunoglobulins relative to baseline at 28 days, 3 months, 6 months after infusion

    Measured: 28 days, 3 months, 6 months

  12. Incidence and grading of severe adverse events (SAE) from 28 days to 2 years after re-infusion

    Measured: 2 years

Timeline

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

  1. First posted6 May 2025
  2. Study start1 May 2025
  3. Primary completion30 Apr 2026
  4. Study completion30 Apr 2028 (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
Dose 1 1.0 × 10e6/kg S103 CAR-T cells Dose 2 2.0 × 10e6/kg S103 CAR-T cells Dose 3 4.0 × 10e6/kg S103 CAR-T cells Dose 4 5.0 × 10e6/kg S103 CAR-T cells
Masking
None (open label)
Enrolment
10 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06958939
Sponsor's protocol ID
CART-20250321
Status last verified
May 2026
First submitted
13 Apr 2025
Last updated
27 May 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Yes: Experimental data may be shared with the consent of the principal investigator

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Dose level 1 group, Dose level 2 group and Dose level 3 group in people with Myasthenia Gravis, Generalized, 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 11 inclusion and 12 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: under way, not looking for participants, as of the sponsor's last update on 27 May 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?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Dose-limiting toxicity (time frame: Day 28). 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 NCT06958939.

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 27 May 2026.

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