It is a single-center, single-arm, open-labeled clinical trial to evaluate the efficacy and safety of CAR-T cells therapy for Chronic or Refractory Primary Immune Thrombocytopenia (ITP).
This open label and single-arm study aims to evaluate the efficacy and safety of CAR-T cells therapy in patients with Chronic or Refractory Primary Immune Thrombocytopenia (ITP). After enrollment, a leukapheresis procedure will be performed to manufacture chimeric antigen receptor (CAR) modified T cells. Patients will get a 3-5 days lymphodepletion therapy with fludarabine and cyclophosphamide, then the CAR-T cells will be infused by vein. After infusion, subjects will be followed for safety and efficacy evaluation up to 12 weeks. For those with a durable remission 12 weeks after infusion, the follow-up will last for at least 12 months for disease control.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
10
CAR-T cells will be administered by vein. Before CAR-T infusion,patients will get a 3-5 days lymphodepletion therapy with fludarabine and cyclophosphamide.
920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
Kunming, Yunnan, China
RECRUITINGPortion of patients with response (R)
platelet count\>30x10\^9/L and at least 2-fold increase of the baseline count, confirmed on at least 2 separate occasions at least 7 days apart, and absence of bleeding
Time frame: At least 2 weeks after infusion
Portion of patients with complete response (CR)
platelet count\>100x10\^9/L, confirmed on at least 2 separate occasions at least 7 days apart, and absence of bleeding
Time frame: At least 2 weeks after infusion
Portion of patients with relapse
platelet count below 30x10\^9/L or less than 2-fold increase of baseline platelet count or bleeding after treating effectively; platelet counts confirmed on at least 2 separate occasions approximately 1 day apart
Time frame: At least 2 weeks after infusion
Time (in days) from treatment start to response (R)
Time calculated from infusion to the day when the response (R) criteria are achieved
Time frame: At least 2 weeks after infusion
Time (in days) from treatment to complete response (CR)
Time calculated from infusion to the day when the complete response (CR) criteria are achieved
Time frame: At least 2 weeks after infusion
Duration (in days) of response (R)
Time calculated from the day when the response (R) criteria are achieved, to the day when loss of response (R) criteria is achieved
Time frame: At least 2 weeks after infusion
Incidence of adverse events(AE) after infusion
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
The frequency, severity, and laboratory findings of all adverse events/serious adverse events are included. Description, time, classification, and outcome of AE events resulted from the investigational medical product, delivery method, or emergency measures will be recorded in the case report form.
Time frame: Up to 12 months after infusion