Refractory Generalized Myasthenia GravisEarly Phase 1gMGNCT06419166
An Exploratory Clinical Study of GC012F Injection for Refractory gMG
A Early Phase 1 study testing GC012F injection in people with Refractory Generalized Myasthenia Gravis, run by Zhejiang University at 1 site in China.
Trial summary
This study is a single-arm, open-label early exploratory clinical trial designed to evaluate the safety, tolerability, and preliminary efficacy of GC012F injection in subjects with refractory GMG. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Refractory Generalized Myasthenia Gravis
- What is being tested?
- GC012F injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteChina
- Study dates
- 1 Dec 2024to 1 Oct 2027
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
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 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subjects or his/her legal proxy/guardian voluntary signing the ICF, and willing and able to follow the procedure in this study.
- Aged ≥18 years old, no gender limitation;
- Patients with confirmed refractory GMG, and the clinical classification is IIa-IVb (including IIa, IIb, IIIa, IIIb, IVa and IVb) in screening;
- Patients whose MG-ADL score is 5 or more, and the proportion of ocular symptoms is less than 50% in the total score;
- Patients with poor efficacy of conventional treatment and/or no effective treatment means relapse or exacerbation despite conventional hormone, immunosuppressant (e.g., azathioprine, mycophenolate mofetil, tacrolimus, cyclosporin A, cyclophosphamide, etc.), or rituximab treatment;
- Patients who are on corticosteroids, the dose of prednisone should not exceed 20 mg/d (or no more than an equivalent dose of another corticosteroid) during the 3 weeks prior to apheresis, and the dose isn't escalated during 3weeksk prior to apheresis, the dose isn't changed within 4 weeks prior to infusion;
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- Patients with positive MG-specific autoantibodies in the screening period: acetylcholine receptor autoantibody (anti-AChR) titer or muscle-specific tyrosine kinase autoantibody (anti-MuSK) or low-density lipoprotein receptor-associated protein 4 autoantibody (anti-LRP4) or anti-acetylcholine receptor cluster antibody must be higher than the upper limit of the laboratory reference normal value;
- Life expectancy ≥3 months;
- The results of laboratory test during screening period shall meet all following criteria:
- Neu ≥1.0 × 109/L; Hb ≥8.0 g/dL; PLT ≥50 × 109/L;
- ALT ≤3 × ULN; AST ≤3 × ULN; TBIL <2 × ULN (DBIL ≤1.5 × ULN for subjects with Gilbert's syndrome)
- Creatinine clearance (19.3 Appendix 3) ≥30 mL/min;
- APTT ≤1.5 × ULN, PT ≤1.5 × ULN;
- LVEF ≥50% based on echocardiography, no findings of pericardial effusion.
- Women of child-bearing age should:
- Have a negative serum β human chorionic gonadotropin (β-hCG) pregnancy test confirmed by investigators during the screening period;
- Agree to avoid breastfeeding during the study period until at least 1 year after the infusion of GC012F Injection or until two consecutive flow cytometry tests show the absence of CAR-T cells (whichever occurs later).
- Any male subjects who have sexual partners and female subjects with childbearing potential shall agree to take effective contraceptive methods (e.g. oral contraceptive pills, intrauterine device or condoms) from the screening starting until at least 1 year post GC012F Injection infusion or until two consecutive flow cytometry tests show the absence of CAR-T cells (whichever occurs later. Male subjects must agree to use condoms during sexual contact with pregnant females or females with fertility for at least 1 year after the infusion of GC012F Injection, even if a successful vasectomy has been performed;
- Venous access available for blood collection, and no contraindications for leukapheresis.
Who cannot join
- Subjects have a history of severe hypersensitivity or allergy;
- Any contraindication for fludarabine, cyclophosphamide and any component of the investigational product;
- Subjects with any of the following heart diseases:
- Congestive heart failure (New York Heart Association (NYHA) Class III or IV);
- Experienced myocardial infarction or underwent coronary artery bypass grafting (CABG) within 6 months prior to screening period;
- Clinically significant ventricular arrhythmias or a history of unexplained syncope not due to vasovagal reaction or dehydration; or a QTc interval >480 ms during screening;
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- History of severe non-ischemic cardiomyopathy.
- Accompanied by other uncontrolled malignancies. Subjects with the following conditions should be excluded: early-stage tumors that have received radical treatment (carcinoma in situ or grade 1 tumors, or non-ulcerated primary melanoma with a depth <1 mm and no involvement of lymph nodes), basal cell skin cancer, skin squamous cell carcinoma, cervical carcinoma in situ, or breast cancer in situ that has received potential radical treatment;
- Severe underlying medical conditions, such as:
- Fungal, bacterial, viral or other infections or suspected fungal, bacterial, viral or other infections that cannot be controlled or require general intravenous administration;
- Significant clinical evidence of dementia or mental status changes;
- History of any central nervous system (CNS) or neurodegenerative diseases, (e.g., epilepsy, seizures, paralysis, aphasia, stroke, severe brain injury, dementia, Parkinson's disease, psychiatric disorders).
- Positive results in any of the following tests:
- HIV antibody positive;
- HBsAg positive; or HBcAb positive and HBV-DNA above the lower limit of detection of the analytical method;
- HCV antibody positive with HCV RNA above the lower limit of detection of the analysis method; or known history of hepatitis C without completion of antiviral therapy for ≥24 weeks;
- Syphilis antibody positive.
- Received therapy of non-hormonal immunosuppressants within 3 weeks prior to apheresis;
- Major surgery within 2 weeks prior to leukapheresis or surgery plan during the study (except for local anesthesia surgery, but not performed within 2 weeks after infusion);
- Receipt of a live-attenuated vaccine within 4 weeks prior to leukapheresis;
- Intravenous injection of immunoglobulins or therapy of plasma exchange (PE);
- Receipt of other biologics for MG within 3 weeks prior to apheresis or within 8 weeks prior to infusion;
- Participation in any other clinical trial within 4 weeks prior to signing ICF, or the date of signing the ICF still within 5 half-lives of the drug from the last dose in the last clinical trial (whichever is longer);
- Thymectomy within 12 months prior to ICF signing;
- Pregnant women or lactating women who do not agree to abstain from breastfeeding, men and women who have a fertility plan during participation in this study or within 1 year after receiving study treatment;
- Any situation that may hinder subjects' participation in the entire trial or confuse the results, or any situation in which investigators believe that participation in this study is not in the subject's best interests.
Trial locations and contacts
1 site in 1 country.
China1 site
The First Affiliated Hospital,College of Medicine, Zhejiang University
Hanzhou, Zhejiang, 310003
About this study
Official title: An Exploratory Clinical Study of GC012F Injection for the Treatment of Refractory Generalized Myasthenia Gravis
This study is a single-arm, open-label early exploratory clinical trial designed to evaluate the safety, tolerability, and preliminary efficacy of GC012F injection in subjects with refractory GMG. Additionally, the study aims to assess the pharmacokinetic (PK), pharmacodynamic (PD) characteristics, and immunogenicity of GC012F injection in subjects.
Read the detailed description
This study is a single-arm, open-label early exploratory clinical trial designed to evaluate the safety, tolerability, and preliminary efficacy of GC012F injection in subjects with refractory GMG. Additionally, the study aims to assess the pharmacokinetic (PK), pharmacodynamic (PD) characteristics, and immunogenicity of GC012F injection in subjects.
The trial consists of several phases: screening period, apheresis day, baseline period, lymphodepletion period, pre-infusion assessment period, GC012F infusion period, safety and efficacy follow-up period, long-term follow-up period, and study discontinuation visit (if applicable).
Qualified subjects will undergo apheresis and receive the infusion after the production of CAR-T products. Subjects will undergo lymphodepletion before CAR-T cell infusion and assessment before infusion. Subjects meeting the cell infusion criteria will receive CAR-T cell infusion according to the dose specified in the protocol. Dose adjustments may occur based on safety and clinical efficacy for subjects in the same group or subsequent trial groups.
- Conditions
- Refractory Generalized Myasthenia Gravis
- Being tested
- GC012F 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.
GC012F injectionDrug
Each subject will receive GC012F injection (CD19-BCMA CAR-T cells) once by intravenous infusion on Day 0.
Other Name: CD19-BCMA CAR-T cells
| Study group | Role | What they receive |
|---|---|---|
| Refractory gMG subjects | Experimental | Refractory Generalized Myasthenia Gravis Receives: Drug: GC012F injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of DLT
DLT
Measured: Within 28 days after GC012F injection infusion
Frequency and severity of abnormal findings in electrocardiograms
The electrocardiography shall be measured after 5 minutes of rest, and assessments included as follows: Heart rate, RR interval, PR interval, QT interval, QRS wave, QT interval and other indexes.
Measured: Within 96 weeks after GC012F injection infusion
Frequency and severity of abnormal findings of adverse events.
Any untoward medical event that occurs after a subject has administered an investigational product, which may be manifested as a symptom, sign, disease or laboratory abnormality but does not necessarily have a causal relationship with the investigational product.
Measured: Within 96 weeks after GC012F injection infusion
Frequency and severity of abnormal findings in physical examinations
The full physical examination at least includes assessments of skin, mucosa, lymph nodes, head, neck, chest, abdomen, spine/limbs, and nervous system. A full physical examination needs to be completed only during the screening period, and the physical examination can be performed in subsequent visit as per changes in signs and symptoms.
Measured: Within 96 weeks after GC012F injection infusion
Frequency and severity of abnormal findings in laboratory tests
Laboratory tests include blood test，Coagulation function ，Infectious disease detection and tests recommended by the Investigator
Measured: Within 96 weeks after GC012F injection infusion
Frequency and severity of abnormal findings in vital signs
Vital signs shall be measured after 5 minutes of rest, and assessments included as follows: Temperature, oxygen saturation, heart rate, respiratory rate, resting systolic and diastolic blood pressure.
Measured: Within 96 weeks after GC012F injection infusion
RP1D
Recommended phase I dose
Measured: 2 years after GC012F injection infusion
MTD
maximum tolerated dose
Measured: 2 years after GC012F injection infusion
Secondary outcomes (9)
PK parameters of CAR-T cells in peripheral blood after GC012F infusion (Cmax);
PK results of GC012F
Measured: Within 96 weeks after GC012F injection infusion
PK parameters of CAR-T cells in peripheral blood after GC012F infusion (Tmax);
PK results of GC012F
Measured: Within 96 weeks after GC012F injection infusion
PK parameters of CAR-T cells in peripheral blood after GC012F infusion ( AUC);
PK results of GC012F
Measured: Within 96 weeks after GC012F injection infusion
Levels of cytokines [IL-6、IL-10、IFN-γ、TNF-α、MCP-1(as applicable)], lymphocyte subsets, and soluble BCMA in peripheral blood after GC012F infusion;
PK results of GC012F
Measured: Within 28 days after GC012F injection infusion
Disease activity indices:MG-ADL
Efficacy results of GC012F
Measured: Within 96 weeks after GC012F injection infusion
Disease activity indices:MGC
Efficacy results of GC012F
Measured: Within 96 weeks after GC012F injection infusion
Disease activity indices:QMG
Efficacy results of GC012F
Measured: Within 96 weeks after GC012F injection infusion
Disease activity indices: MG-QoL 15r
Efficacy results of GC012F
Measured: Within 96 weeks after GC012F injection infusion
Disease activity indices: post-intervention status according to the Myasthenia Gravis Foundation of America (MGFA) classification
Efficacy results of GC012F
Measured: Within 96 weeks after GC012F injection infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 May 2024
- Study start1 Dec 2024 (estimated)
- Primary completion1 Jun 2027 (estimated)
- Study completion1 Oct 2027 (estimated)
Why it stopped: The study stopped early, before enrolling its first participant.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Refractory Generalized Myasthenia GravisStudies of the same condition, open and under way.
- More trials run by Zhejiang UniversityThe sponsor's other registered studies.
- 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
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Zhejiang University
- Sponsor type
- University, hospital or other
- Collaborators
- Gracell Biotechnologies (Shanghai) Co., Ltd.
- Responsible party
- Principal investigator, He Huang, Zhejiang University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06419166
- Sponsor's protocol ID
- TXB2024002
- Acronym
- gMG
- Status last verified
- Jun 2025
- First submitted
- 25 Apr 2024
- Last updated
- 27 Jun 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 27 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Exploratory Clinical Study of GC012F Injection for the Treatment of Refractory Generalized Myasthenia Gravis
- ClinicalTrials.gov ID
- NCT06419166
- Acronym / sponsor ID
- gMG / TXB2024002
- Conditions
- Refractory Generalized Myasthenia Gravis
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: The study stopped early, before enrolling its first participant.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Zhejiang UniversityUniversity, hospital or other
- Collaborators
- Gracell Biotechnologies (Shanghai) Co., Ltd.
- Study start
- 1 Dec 2024 (estimated)
- Primary completion
- 1 Jun 2027 (estimated)
- Study completion
- 1 Oct 2027 (estimated)
- First posted
- 17 May 2024 (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-arm, open-label early exploratory clinical trial designed to evaluate the safety, tolerability, and preliminary efficacy of GC012F injection in subjects with refractory GMG. Additionally, the study aims to assess the pharmacokinetic (PK), pharmacodynamic (PD) characteristics, and immunogenicity of GC012F injection in subjects.
Detailed description
This study is a single-arm, open-label early exploratory clinical trial designed to evaluate the safety, tolerability, and preliminary efficacy of GC012F injection in subjects with refractory GMG. Additionally, the study aims to assess the pharmacokinetic (PK), pharmacodynamic (PD) characteristics, and immunogenicity of GC012F injection in subjects.
The trial consists of several phases: screening period, apheresis day, baseline period, lymphodepletion period, pre-infusion assessment period, GC012F infusion period, safety and efficacy follow-up period, long-term follow-up period, and study discontinuation visit (if applicable).
Qualified subjects will undergo apheresis and receive the infusion after the production of CAR-T products. Subjects will undergo lymphodepletion before CAR-T cell infusion and assessment before infusion. Subjects meeting the cell infusion criteria will receive CAR-T cell infusion according to the dose specified in the protocol. Dose adjustments may occur based on safety and clinical efficacy for subjects in the same group or subsequent trial groups.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Refractory gMG subjects | Experimental | Drug: GC012F injectionRefractory Generalized Myasthenia Gravis |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (19 listed)
- Subjects or his/her legal proxy/guardian voluntary signing the ICF, and willing and able to follow the procedure in this study.
- Aged ≥18 years old, no gender limitation;
- Patients with confirmed refractory GMG, and the clinical classification is IIa-IVb (including IIa, IIb, IIIa, IIIb, IVa and IVb) in screening;
- Patients whose MG-ADL score is 5 or more, and the proportion of ocular symptoms is less than 50% in the total score;
- Patients with poor efficacy of conventional treatment and/or no effective treatment means relapse or exacerbation despite conventional hormone, immunosuppressant (e.g., azathioprine, mycophenolate mofetil, tacrolimus, cyclosporin A, cyclophosphamide, etc.), or rituximab treatment;
Key exclusion criteria (26 listed)
- Subjects have a history of severe hypersensitivity or allergy;
- Any contraindication for fludarabine, cyclophosphamide and any component of the investigational product;
- Subjects with any of the following heart diseases:
- Accompanied by other uncontrolled malignancies. Subjects with the following conditions should be excluded: early-stage tumors that have received radical treatment (carcinoma in situ or grade 1 tumors, or non-ulcerated primary melanoma with a depth <1 mm and no involvement of lymph nodes), basal cell skin cancer, skin squamous cell carcinoma, cervical carcinoma in situ, or breast cancer in situ that has received potential radical treatment;
- Severe underlying medical conditions, such as:
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 |
|---|---|---|
| GC012F injectionEach subject will receive GC012F injection (CD19-BCMA CAR-T cells) once by intravenous infusion on Day 0. Other Name: CD19-BCMA CAR-T cells | Drug | Refractory gMG subjects |
Primary outcome
Incidence of DLT
Time frame: Within 28 days after GC012F injection infusion
DLT
Frequency and severity of abnormal findings in electrocardiograms
Time frame: Within 96 weeks after GC012F injection infusion
The electrocardiography shall be measured after 5 minutes of rest, and assessments included as follows: Heart rate, RR interval, PR interval, QT interval, QRS wave, QT interval and other indexes.
Frequency and severity of abnormal findings of adverse events.
Time frame: Within 96 weeks after GC012F injection infusion
Any untoward medical event that occurs after a subject has administered an investigational product, which may be manifested as a symptom, sign, disease or laboratory abnormality but does not necessarily have a causal relationship with the investigational product.
Frequency and severity of abnormal findings in physical examinations
Time frame: Within 96 weeks after GC012F injection infusion
The full physical examination at least includes assessments of skin, mucosa, lymph nodes, head, neck, chest, abdomen, spine/limbs, and nervous system. A full physical examination needs to be completed only during the screening period, and the physical examination can be performed in subsequent visit as per changes in signs and symptoms.
Frequency and severity of abnormal findings in laboratory tests
Time frame: Within 96 weeks after GC012F injection infusion
Laboratory tests include blood test，Coagulation function ，Infectious disease detection and tests recommended by the Investigator
Frequency and severity of abnormal findings in vital signs
Time frame: Within 96 weeks after GC012F injection infusion
Vital signs shall be measured after 5 minutes of rest, and assessments included as follows: Temperature, oxygen saturation, heart rate, respiratory rate, resting systolic and diastolic blood pressure.
RP1D
Time frame: 2 years after GC012F injection infusion
Recommended phase I dose
MTD
Time frame: 2 years after GC012F injection infusion
maximum tolerated dose
9 secondary outcomes
PK parameters of CAR-T cells in peripheral blood after GC012F infusion (Cmax);
Time frame: Within 96 weeks after GC012F injection infusion
PK parameters of CAR-T cells in peripheral blood after GC012F infusion (Tmax);
Time frame: Within 96 weeks after GC012F injection infusion
PK parameters of CAR-T cells in peripheral blood after GC012F infusion ( AUC);
Time frame: Within 96 weeks after GC012F injection infusion
Levels of cytokines [IL-6、IL-10、IFN-γ、TNF-α、MCP-1(as applicable)], lymphocyte subsets, and soluble BCMA in peripheral blood after GC012F infusion;
Time frame: Within 28 days after GC012F injection infusion
Disease activity indices:MG-ADL
Time frame: Within 96 weeks after GC012F injection infusion
Disease activity indices:MGC
Time frame: Within 96 weeks after GC012F injection infusion
Disease activity indices:QMG
Time frame: Within 96 weeks after GC012F injection infusion
Disease activity indices: MG-QoL 15r
Time frame: Within 96 weeks after GC012F injection infusion
Disease activity indices: post-intervention status according to the Myasthenia Gravis Foundation of America (MGFA) classification
Time frame: Within 96 weeks after GC012F injection infusion
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jun 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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing GC012F injection in people with Refractory Generalized Myasthenia Gravis, run by Zhejiang University 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 19 inclusion and 26 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: withdrawn, as of the sponsor's last update on 27 Jun 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?
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?
The study was withdrawn before enrolling participants, so there are no results.
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?
Incidence of DLT (time frame: Within 28 days after GC012F injection infusion). The study lists 8 primary outcomes in all. 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 27 Jun 2025.