Primary immune thrombocytopenia
Conditions
Interventions
Sponsors
Qilu Hospital of Shandong University
Eligibility
Sex/Gender
All
Age
1 Years to 18 Years
Inclusion criteria
Inclusion criteria: 1.1 year 10.0 g/dL; 6.(6) Serum creatinine = 1.5 times the upper limit of normal; 7.(7) Total serum bilirubin = 1.5 times the upper limit of normal; 8.(8) Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) = 3.0 times of ULN; 9.(9) Guardian and subject (over 8 years old) have signed informed consent; 10.(10) Treatment with ropristim N01 according to a clinician evaluation follow-up plan.
Exclusion criteria
Exclusion criteria: 1. Have the following history: ? history of bone marrow stem cell disease; ? Previous bone marrow transplantation or peripheral blood stem cell transplantation; (3) Known active or prior malignancy, except for basal cell carcinoma with adequate treatment; ? History of myelodysplastic syndrome; ? History of congenital thrombocytopenia; A history of hepatitis B, hepatitis C or Human Immunodeficiency Virus (HIV); History of systemic lupus erythematosus, Evans Syndrome, or autoimmune neutropenia; History of antiphospholipid antibody syndrome or lupus anticoagulant indication positive; ? History of disseminated intravascular coagulation, hemolytic uremic syndrome or thrombotic thrombocytopenic purpura; ? History of VTE or thrombotic events within the past year; 2.(2) TPO receptor agonists such as romiplostim, itripopal, heptapopal were used within 4 weeks before enrollment; 3.(3) Use of pegylated recombinant human megakaryocyte growth and development factor, recombinant human thrombopoietin (rhTPO), or any other thrombopoietic drug within 4 weeks prior to enrollment; 4.(4) Rituximab (any indication) or 6-mercaptopurine (6-MP) had been used within 8 weeks prior to enrollment, or was expected to be used during the study period; 5.(5) Use of alkylating agents within 8 weeks before the screening visit or anticipated use during the study; 6.(6) Vaccination with a vaccine known to reduce platelet count within 8 weeks prior to screening visit; 7.(7) Known allergy to any E. coli derived recombinant products (e.g., granulocyte colony-stimulating factor, growth hormone, interferon-gamma); 8.(8) To the knowledge of the subject and investigator, will not be able to participate in the research visits or procedures required by the Agreement; 9.(9) any disease that may affect the subject's ability to provide written informed consent and/or comply with all required research procedures; 10.(10) Other factors deemed unsuitable for participation in the study by the investigator.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| complete response rate of one month; | — |
Secondary
| Measure | Time frame |
|---|---|
| Partial response rate of one week;Proportion of patients with platelet response;Platelet durable response time;Proportion of patients with durable platelet response;The time when the platelet response first occur;Effective dose for achieving platelet response for the first time;Platelet peak time and patient proportion;Types and incidence of Treatment Related Adverse Events (TRAEs); | — |
Countries
China
Contacts
Public ContactXiuli Ju
Qilu Hospital of Shandong University
Outcome results
None listed