Refractory Myasthenia GravisEarly Phase 1NCT07243366
Allogeneic CD19-CAR-NK Cells in Patients With Refractory Myasthenia Gravis
A Early Phase 1 study testing CD19-CAR-NK Cells Injection in people with Refractory Myasthenia Gravis, run by Guangdong ProCapZoom Biosciences Co., Ltd..
Trial summary
This study is a single-center, prospective, nonrandom, single-arm trial. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Refractory Myasthenia Gravis
- What is being tested?
- CD19-CAR-NK Cells Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- No sites listed yet
- Study dates
- 25 Nov 2025to 30 Nov 2027
- Phase
- Early 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
- Aged 18-65 years old, including the boundary value, no gender restriction;
- MGFA clinical class II-IV;
- Anti-acetylcholine-receptor antibody (AChR-Ab) shows positive;
- Willing to participate in the study, understand and sign the informed consent form (ICF).
- Baseline characteristics - all must be fulfilled:
- Refractory MG patients: Meet the diagnostic criteria in the Chinese Guidelines for the Diagnosis and Treatment of MG (2020 edition). On the basis of typical MG clinical features (fluctuating muscle weakness), a diagnosis can be made if any of the following three points are met, including pharmacological examination, electrophysiological characteristics, and serum anti-AChR antibody testing. Other diseases must also be excluded.
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- Myasthenia Gravis Activities of Daily Living (MG-ADL) score ≥ 6, with ocular sub-score < 50 % of total.
- Diagnostic Criteria: Meeting any one of the following four conditions:
- Following an adequate dose and full course of at least two conventional immunotherapies (including both corticosteroids and non-steroidal immunosuppressants), and at least one complete treatment cycle with a biologic agent (efgartigimod, eculizumab, rituximab, or telitacicept), the post-intervention status (PIS) remains unchanged or worsens.
- Following an adequate dose and full course of at least two conventional immunotherapies (including both corticosteroids and non-steroidal immunosuppressants) and at least one complete treatment cycle with a biologic agent, the PIS improves, yet the MG-ADL score is still ≥6 and persists for at least six months.
- Following an adequate dose and full course of at least two conventional immunotherapies (including both corticosteroids and non-steroidal immunosuppressants) and at least one complete treatment cycle with a biologic agent, the PIS shows remission or improvement; however, during the regular tapering of immunotherapy drugs, the patient experiences ≥2 exacerbations per year with an MG-ADL score ≥6.
- Despite treatment with multiple immunotherapies-including intravenous immunoglobulin (IVIG), plasma exchange, and high-dose intravenous methylprednisolone (IVMP)-as well as aggressive infection control after a myasthenic crisis, the patient remains unable to be weaned from mechanical ventilation for more than 14 days due to respiratory muscle weakness caused by MG.
- Within 30 days before enrollment: glucocorticoids unchanged for 1 month, immunosuppressants for 3 months, pyridostigmine for 2 weeks, and stable MG-ADL score for 1 month.
- Note: 1. "Two conventional immunotherapies" consist of one corticosteroid plus one non-steroidal immunosuppressant. 2. "Adequate dose for a full course" is defined as follows: 1) Corticosteroid: 0.5-1.0 mg · kg-¹ · d-¹ for ≥ 8 weeks. 2) One of the following non-steroidal immunosuppressants taken for the specified minimum duration: A. Azathioprine: 1.5-2.5 mg · kg-¹ · d-¹ in 2-3 divided doses for ≥ 24 weeks. B. Methotrexate: 15 mg once weekly for ≥ 24 weeks. C. Mycophenolate mofetil: 0.75-1.00 g twice daily for ≥ 24 weeks. D. Cyclophosphamide: 400-800 mg intravenously every week OR 100 mg/day orally in two divided doses, with a cumulative dose ≥ 15 g. E. Tacrolimus: 2-3 mg/day with at least one trough level ≥ 4.8 ng/mL for ≥ 12 weeks. F. Cyclosporine: 2-4 mg · kg-¹ · d-¹ in two divided doses, with at least one fasting trough level ≥ 100 ng/mL for ≥ 24 weeks. 3. Failure to complete the full dose and course of any one conventional immunotherapy due to contraindications, comorbidities, or inability to tolerate drug adverse effects is considered equivalent to having an inadequate response after completing a full dose and course of that conventional immunotherapy. 4. Biologic agents include efgartigimod, eculizumab, rituximab, and telitacicept.
Who cannot join
- Received IVMP, IVIG, or TPE within the 2 months before the current visit;
- Unable to cooperate in completing the MG-ADL, or QMG, or Quantitative QMG questionnaire.
- Judged by a senior clinician to have any of the following: 1)Severe or chronic urinary-tract infection; 2)History of recurrent infections; 3)Previous allergy to any human-derived biologic drug; 4)Depression, suicidal ideation, or other psychiatric disorder;
- Patients with active infection, such as herpes zoster, HIV, active tuberculosis, or active hepatitis.
- Rituximab within 6 months, eculizumab within 3 months, or efgartigimod within 1 month before enrollment.
- Receipt of any live vaccine within 3 months before screening or planned vaccination during the study.
- Subjects deemed unsuitable for participation in this trial by the investigator (e.g., severe psychiatric illness).
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Exploratory Clinical Trial of Allogeneic CD19-CAR-NK Cells in Patients With Refractory Myasthenia Gravis
This study is a single-center, prospective, nonrandom, single-arm trial.
Read the detailed description
This study is a single-center, prospective, nonrandom, single-arm trial. To evaluate the clinical safety and efficacy of allogeneic CD19-CAR-NK Cells in patients with refractory myasthenia gravis.
- Conditions
- Refractory Myasthenia Gravis
- Being tested
- CD19-CAR-NK Cells 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.
CD19-CAR-NK Cells InjectionDrug
Also called CD19-CAR-NK Cells
Allogeneic CD19-CAR-NK cells will be administered intravenously at a fixed dose, once every two weeks for a total treatment duration of 24 weeks.
| Study group | Role | What they receive |
|---|---|---|
| CD19-CAR-NK Cells Group | Experimental | Allogeneic CD19-CAR-NK cells will be administered intravenously at a fixed dose, once every two weeks for a total treatment duration of 24 weeks. Receives: Drug: CD19-CAR-NK Cells Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events
Incidence and severity of adverse events from subjects during the trial.
Measured: through study completion, an average of 1 year
Secondary outcomes (5)
Myasthenia Gravis Activities of Daily Living Scores
Changes in myasthenia gravis activities of daily living scores from baseline at every 2-week on-treatment visit and each follow-up, including mean change and symptom fluctuation.
Measured: at every 2-week on-treatment visit (up to 1 month) and each follow-up (up to 1 year)
Quantitative Myasthenia Gravis Scores
Changes in quantitative myasthenia gravis scores from baseline at every 2-week on-treatment visit and each follow-up, including mean change and symptom fluctuation.
Measured: at every 2-week on-treatment visit (up to 1 month) and each follow-up (up to 1 year)
Myasthenia Gravis Foundation of America Post-intervention Status
Changes in myasthenia gravis foundation of America post-intervention status from baseline at every 2-week on-treatment visit and each follow-up.
Measured: at every 2-week on-treatment visit (up to 1 month) and each follow-up (up to 1 year)
AChR-antibody titers
Changes in AChR-antibody titers from baseline at every 2-week on-treatment visit and each follow-up.
Measured: at every 2-week on-treatment visit (up to 1 month) and each follow-up (up to 1 year)
lymphocyte subsets
Changes in lymphocyte subsets from baseline at every 2-week on-treatment visit and each follow-up.
Measured: at every 2-week on-treatment visit (up to 1 month) and each follow-up (up to 1 year)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Nov 2025
- Study start25 Nov 2025 (estimated)
- Primary completion30 Jun 2027 (estimated)
- Study completion30 Nov 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Refractory Myasthenia GravisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Guangdong ProCapZoom Biosciences Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07243366
- Sponsor's protocol ID
- PLKR-CAR-NK-I-101
- Status last verified
- Sep 2025
- First submitted
- 9 Sep 2025
- Last updated
- 21 Nov 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Exploratory Clinical Trial of Allogeneic CD19-CAR-NK Cells in Patients With Refractory Myasthenia Gravis
- ClinicalTrials.gov ID
- NCT07243366
- Acronym / sponsor ID
- PLKR-CAR-NK-I-101
- Conditions
- Refractory Myasthenia Gravis
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Guangdong ProCapZoom Biosciences Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 25 Nov 2025 (estimated)
- Primary completion
- 30 Jun 2027 (estimated)
- Study completion
- 30 Nov 2027 (estimated)
- First posted
- 21 Nov 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is a single-center, prospective, nonrandom, single-arm trial.
Detailed description
This study is a single-center, prospective, nonrandom, single-arm trial. To evaluate the clinical safety and efficacy of allogeneic CD19-CAR-NK Cells in patients with refractory myasthenia gravis.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CD19-CAR-NK Cells Group | Experimental | Drug: CD19-CAR-NK Cells InjectionAllogeneic CD19-CAR-NK cells will be administered intravenously at a fixed dose, once every two weeks for a total treatment duration of 24 weeks. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- Aged 18-65 years old, including the boundary value, no gender restriction;
- MGFA clinical class II-IV;
- Anti-acetylcholine-receptor antibody (AChR-Ab) shows positive;
- Willing to participate in the study, understand and sign the informed consent form (ICF).
- Baseline characteristics - all must be fulfilled:
Key exclusion criteria (7 listed)
- Received IVMP, IVIG, or TPE within the 2 months before the current visit;
- Unable to cooperate in completing the MG-ADL, or QMG, or Quantitative QMG questionnaire.
- Judged by a senior clinician to have any of the following: 1)Severe or chronic urinary-tract infection; 2)History of recurrent infections; 3)Previous allergy to any human-derived biologic drug; 4)Depression, suicidal ideation, or other psychiatric disorder;
- Patients with active infection, such as herpes zoster, HIV, active tuberculosis, or active hepatitis.
- Rituximab within 6 months, eculizumab within 3 months, or efgartigimod within 1 month before enrollment.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CD19-CAR-NK Cells InjectionAlso: CD19-CAR-NK CellsAllogeneic CD19-CAR-NK cells will be administered intravenously at a fixed dose, once every two weeks for a total treatment duration of 24 weeks. | Drug | CD19-CAR-NK Cells Group |
Primary outcome
Adverse Events
Time frame: through study completion, an average of 1 year
Incidence and severity of adverse events from subjects during the trial.
5 secondary outcomes
Myasthenia Gravis Activities of Daily Living Scores
Time frame: at every 2-week on-treatment visit (up to 1 month) and each follow-up (up to 1 year)
Quantitative Myasthenia Gravis Scores
Time frame: at every 2-week on-treatment visit (up to 1 month) and each follow-up (up to 1 year)
Myasthenia Gravis Foundation of America Post-intervention Status
Time frame: at every 2-week on-treatment visit (up to 1 month) and each follow-up (up to 1 year)
AChR-antibody titers
Time frame: at every 2-week on-treatment visit (up to 1 month) and each follow-up (up to 1 year)
lymphocyte subsets
Time frame: at every 2-week on-treatment visit (up to 1 month) and each follow-up (up to 1 year)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Nov 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing CD19-CAR-NK Cells Injection in people with Refractory Myasthenia Gravis, run by Guangdong ProCapZoom Biosciences Co., Ltd..
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 14 inclusion and 7 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: opening soon, as of the sponsor's last update on 21 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
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 Jiexing Chen (183 0200 2029, jxchen@procapzoom.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse Events (time frame: through study completion, an average of 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 21 Nov 2025.