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.
1. Thrombocytopenia caused by myelodysplastic syndromes, early aplastic anemia, atypical aplastic anemia, thrombotic thrombocytopenic purpura, etc.
2. Bone marrow examination during the screening period suggests myelofibrosis MF≥2 (European consensus scoring standard Thieleja2005) or bone marrow examination indicates the presence of primary diseases other than ITP that can cause thrombocytopenia.
3. Allergic history to any component in the cell product.
Congestive heart failure of NYHA class III or IV.
Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
Lead sponsor
Shenzhen Pregene Biopharma Co., Ltd.
Collaborator