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

Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1
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. 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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 80
Healthy Volunteers: f
View:

• 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 pregnancy test result during screening; a negative urine pregnancy test result must be confirmed before receiving CART for the first time.

Locations
Other Locations
China
Tangdu Hospital, The Fourth Military Medical University
RECRUITING
Xi'an
Contact Information
Primary
Zhe Ruan
ruanzhe573291596@126.com
8618682932643
Time Frame
Start Date: 2024-07-12
Estimated Completion Date: 2026-12-01
Participants
Target number of participants: 9
Treatments
Experimental: CD19-BCMA Targeted CAR-T
Related Therapeutic Areas
Sponsors
Leads: Ting Chang, MD

This content was sourced from clinicaltrials.gov