A single arm, open-label pilot study is designed to determine the safety and effectiveness of CD19 CAR NK cells (KN5501) in patients with refractory immune thrombocytopenia. 9 patients are planned to be enrolled in the dose-escalation trial (9×10\^9 cells, 13.5×10\^9 cells). The primary objective of the study is to evaluation of the safety and feasibility of KN5501 for the treatment of relapsed/refractory B-cell related autoimmune diseases. The secondary objective is to evaluate evaluation of KN5501 for the treatment of refractory immune thrombocytopenia. The exploratory objective is to evaluate expansion, persistence and ability to deplete CD19 positive B cells of KN5501 in patients with refractory immune thrombocytopenia.
Age: ≥ 18 years old and ≤ 65 years old, male or female, subjects voluntarily participate in this clinical study and sign the Informed Consent Form (ICF);
Diagnosis of immune thrombocytopenia (UTP) (according to American Society of Hematology 2019 guidelines and International ITP Working Group);
Platelet count is decreased in routine blood tests at least two consecutive times, with no obvious abnormalities in the morphology of blood cells on microscopic examination of peripheral blood smears;
Spleen is not enlarged during screening;
Subjects with known severe allergic reactions, hypersensitivity, contraindication to any medications during the trial (cyclophosphamide, fludarabine, tozumabs), or subjects with a history of severe allergic reactions;
Subjects with active infection receiving intravenous (IV) antibiotic treatment, or received intravenous (IV) antibiotic treatment within one week prior to anti-CD19 CAR NK Cell (KN5501) infusion;
Subjects with acquired and congenital immunodeficiency diseases;
Subjects with grade III or IV heart failure (NYHA classification);
Single group
anti-CD19 CAR NK cells (KN5501)
DrugPatients will receive Fludarabine (25 mg/m2 per day) and Cyclophosphamide (1000mg/m2 per day) on day -3. Doses of 9×10\^9 cells, 13.5×10\^9 anti-CD19 CAR NK cells (KN5501) will infused in each group using the dose-escalation strategy
Incidence of Dose Limiting Toxicity (DLTs)
To characterize the Dose Limiting Toxicities (DLTs) of anti-CD19 CAR NK Cells for refractory immune thrombocytopenia
Treatment Emergent Adverse Events (TEAEs)
To characterize the Treatment Emergent Adverse Events (TEAEs) of anti-CD19 CAR NK Cells for refractory immune thrombocytopenia
Objective Response Rate of subjects
Objective Response is assessed according to definitions of International ITP Working Group. Objective Response Rate is defined as proportion of patients with PLT content.
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Changzhou No.2 People's Hospital
Lead sponsor
Rui Therapeutics Co., Ltd
Collaborator
