Clinical Study on the Safety and Efficacy of PRG-1801 for the Treatment of Recurrent/Refractory Immune Thrombocytopenia (ITP)
This is a single center, open-label, 3+3 dose escalation, early phase 1 study to evaluate the safety, tolerability, and preliminary efficacy of PRG-1801 for patients with relapsed/refractory immune thrombocytopenia (ITP).
• 1\. Age ≥18 years, regardless of gender.
• 2\. Clinically diagnosed with primary immune thrombocytopenia for at least 6 months, with a platelet count \<30×10\^9/L within 48 hours before participating in the study.
• 3\. Positive for anti-platelet glycoprotein autoantibodies (such as GPIIb/IIIa).
• 4\. Previously received first-line and/or second-line ITP treatment (first-line treatment includes: corticosteroids or immunoglobulins; second-line treatment includes thrombopoietin receptor agonists (such as eltrombopag, romiplostim) and/or rituximab, etc.), but the treatment was ineffective (platelet count \<30×10\^9/L after treatment, or platelet count did not increase to twice the baseline value, or there was bleeding), or relapsed after effective treatment (platelet count dropped below 30×10\^9/L after effective treatment, or dropped to less than twice the baseline value, or bleeding symptoms occurred) or difficult to maintain after stopping TPO receptor agonists.
• 5\. Bone marrow examination shows increased or normal megakaryocytes.
• 6\. Basic normal function of important organs:
‣ Echocardiography indicates an ejection fraction ≥50%, and the electrocardiogram shows no significant abnormalities.
⁃ Creatinine clearance rate (CrCl) (Cockcroft-Gault formula) ≥30 mL/min.
⁃ Alanine transaminase (ALT) and aspartate transaminase (AST) ≤3.0× the upper limit of normal (ULN).
⁃ Total bilirubin (TBIL) and alkaline phosphatase (AKP or ALP) ≤2.0×ULN (Gilbert's syndrome ≤ 3.0×ULN).
⁃ Absolute lymphocyte count (ALC) ≥0.5×10\^9/L; absolute neutrophil count (ANC) ≥1×10\^9/L; hemoglobin (Hb) ≥60 g/L; platelet count ≥10×10\^9/L.
⁃ Blood oxygen saturation \>92%.
• 7\. Meet the standards for apheresis or venous blood collection, and have no contraindications to cell collection.
• 8\. Men of reproductive potential and women of childbearing age must agree to use effective contraception from the signing of the informed consent form until 1 year after the use of the study drug. Blood pregnancy tests for women of childbearing age must be negative at screening and before cell infusion, and they must not be breastfeeding.
• 9\. The participant or their guardian agrees to participate in this clinical trial and signs the informed consent form (ICF), indicating their understanding of the purpose and procedures of this clinical trial and their willingness to participate in the study.