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Tests treatment safety and results for Myasthenia Gravis
Official title Safety and Efficacy of CD19-BCMA Targeted CAR-T Therapy for Refractory Generalized Myasthenia Gravis
ClinicalTrials.gov ID: NCT06371040
What this study is testing
What is CD19-BCMA Targeted CAR-T Dose 1?
CD19-BCMA Targeted CAR-T Dose 1 is an investigational medicine, being studied as a potential treatment for myasthenia gravis.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 80
You may be able to join if
- 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...
- 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...
- Evaluated by the researcher as refractory MG. Refractory MG is defined as:
You likely can't join if
- 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...
- The researcher believes that there is any medical or mental condition that may harm the research participant or affect the research participant's...
- 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...
- Study participants have clinically relevant active infections (such as sepsis, pneumonia or abscess) or serious infections (resulting in...
- thymoma that underwent thymectomy within 6 months before baseline or was planned to undergo thymectomy during the study, or required chemotherapy...
- Investigator participants have received live attenuated vaccine vaccination within 8 weeks before screening; or plan to receive live vaccine...
See the full eligibility criteria
Who can 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
- 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...
What rules you out
- 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...
- 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...
- 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;
- 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: people 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...
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Xi'an, Shaanxi, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 80 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Xi'an, Shaanxi, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.