This is an open-label, single-arm, dose-escalation study designed to evaluate the safety, tolerability, recommended subsequent dose, pharmacokinetic profile, and preliminary efficacy of JCXH-213 in adult patients with primary immune thrombocytopenia (ITP) who are refractory or relapsed after treatment with corticosteroids and second-line therapies.
The study consists of a screening period, treatment period, end-of-treatment visit, safety follow-up, and study withdrawal visit. Two dose groups are planned: 2 mg and 4 mg, with 2 patients to be enrolled in each dose group. During the treatment period, JCXH-213 at the assigned dose will be administered once every other day for a total of 7 doses. Assessments for safety and efficacy will be conducted according to the study schedule. After the last dose, patients will undergo an end-of-treatment visit and safety follow-up until the end of the study.
Voluntarily sign the informed consent form (ICF) and be expected to complete the follow-up visits and treatment required by the study protocol.
Age ≥ 18 years and ≤ 65 years at screening, male or female.
Clinical diagnosis of primary immune thrombocytopenia (ITP) for no less than 3 months, according to the American Society of Hematology 2011 Evidence-Based Practice Guideline (Neunert et al. 2011) or the International Consensus Report on the Investigation and Management of Primary Immune Thrombocytopenia (Provan et al. 2010), as applicable.
At least one second-line ITP therapy includes: rituximab and/or recombinant human thrombopoietin (rhTPO, e.g., TPO), thrombopoietin receptor agonists (TPO-RA, e.g., eltrombopag, hetrombopag, avatrombopag, romiplostim).
Diagnosis of autoimmune hemolytic anemia, any secondary or hereditary thrombocytopenic disorders, including leukemia, lymphoma, multiple myeloma, aplastic anemia, myelodysplastic syndrome, Evans syndrome, common variable immunodeficiency, systemic lupus erythematosus, liver cirrhosis, antiphospholipid syndrome, pseudothrombocytopenia, or drug-induced thrombocytopenia (e.g., due to quinine, heparin, antibiotics, or antiepileptic drugs).
History of any thromboembolic event or extensive/severe bleeding within 1 year prior to the first dose of study drug, such as hemoptysis, major upper gastrointestinal bleeding, intracranial hemorrhage, or presence of sepsis or other irregular bleeding.
Receipt of anti-CD20 monoclonal antibody (e.g., rituximab) or other medicinal treatments (including cyclophosphamide and vindesine) within 3 months prior to the first dose of study drug.
Receipt of the following medicinal treatments within 4 weeks prior to the first dose of study drug: azathioprine, danazol, dapsone, cyclosporine A, tacrolimus, or sirolimus.