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IRCT20221129056655N2 IRCT 2023-09-23 Shiraz University of Medical Sciences Romiplostim in treatment-naïve acquired aplastic anemia Assessment of efficacy and safety of Romiplostim in immunosuppressive-naïve children with severe acquired aplastic anemia: a phase II/III open-label study 2023-06-22 anticipated 23 Complete https://irct.ir/trial/67137 interventional Randomization: N/A, Blinding: Not blinded, Placebo: Not used, Assignment: Single, Purpose: Treatment. 2-3 Acquired aplastic anemia. Intervention group: Romiplostim (N-Plate, Amgen company) is administered subcutaneously at a fixed dose of 10 mcg/kg weekly for 4 weeks (weeks 1-4). The drug is administered on day 1 of IST (horse ATG 40 mg/kg/day on days 1-4 plus cyclosporine 10 mg/kg/day PO divided into two equal daily doses). The dosage is titrated to steps of 10, 15, and 20 mcg/kg once weekly for up to 27 weeks (weeks 5-27). The romiplostim dose is adjusted depending on platelet response and toxicity. The dose is increased by one step every 4 weeks until a platelet response is achieved. If the platelet count is >200 ≥ 109/L, the dose is reduced by one step. The romiplostim dose is tapered with the intent to discontinue when trilineage hematopoiesis is achieved.Trilineage hematopoiesis is defined as a platelet count of >50 × 109/L, hemoglobin concentration of >10 g/dL and neutrophil count of >1×109/L maintained for 8 weeks with the same romiplostim dose without transfusion.. Yes - There is a plan to make this available What will be shared: Patients' data will be shared anonymously. When: The data will be accessible after the results are published. To whom: Academic investigators will have access to the data upon their request. Conditions: Researchers can use data for research purposes with the permission of the principal investigator. Where to obtain: Text to the PI (Mohammadreza Bordbar) to the following phone number: 09177072149 or send an email to the following address: mbordbar53@gmail.com How to obtain: The request will be sent to the Ethics Committee of the University. If there is no ethical concern, they will be sent to them. Usually it takes 30 working days. Comments:

public Mohammadreza Bordbar

Amir Oncology Hospital- Farhangshahr Street

Shiraz Iran (Islamic Republic of) 71879-15998 +98 71 3632 3532 mbordbar53@gmail.com Shiraz University of Medical Sciences scientific Mohammadreza Bordbar

Amir Oncology Hospital- Farhangshahr Street

Shiraz Iran (Islamic Republic of) 71879-15998 +98 71 3632 3532 mbordbar53@gmail.com Shiraz University of Medical Sciences Iran (Islamic Republic of) Children 1 month up to the age of 18 years with severe and very severe acquired aplastic anemia who are eligible to be treated with IST (ATG plus cyclosporine) 1 month 18 years Both 1. Patients with inherited aplastic anemia 2. Previously treated with ATG, cyclosporine 3. Diagnosed as having acute myeloid leukemia (AM)L or myelodysplastic syndrome (MDS) 4. Concurrent active infection not adequately responding to appropriate therapy 5. Having active malignancies, or having a history of the treatment of malignancies within 5 years prior to informed consent 6. Concurrent paroxysmal nocturnal hemoglobinuria 7. History of chromosome aberrations discovered in bone marrow cells 8. Bone marrow fibrosis based on reticulin stain 9. Active or latent HIV infection 10. Active or latent HBV infection 11. Active or latent HCV infection 12. Active or latent tuberculous infection 13. Active or latent visceral leishmaniasis 14. Planned hematopoietic stem cell transplantation during the study D61.3 Idiopathic aplastic anemia Treatment - Drugs Intervention group: Romiplostim (N-Plate, Amgen company) is administered subcutaneously at a fixed dose of 10 mcg/kg weekly for 4 weeks (weeks 1-4). The drug is administered on day 1 of IST (horse ATG 40 mg/kg/day on days 1-4 plus cyclosporine 10 mg/kg/day PO divided into two equal daily doses). The dosage is titrated to steps of 10, 15, and 20 mcg/kg once weekly for up to 27 weeks (weeks 5-27). The romiplostim dose is adjusted depending on platelet response and toxicity. The dose is increased by one step every 4 weeks until a platelet response is achieved. If the platelet count is >200 ≥ 109/L, the dose is reduced by one step. The romiplostim dose is tapered with the intent to discontinue when trilineage hematopoiesis is achieved.Trilineage hematopoiesis is defined as a platelet count of >50 × 109/L, hemoglobin concentration of >10 g/dL and neutrophil count of >1×109/L maintained for 8 weeks with the same romiplostim dose without transfusion. Complete or partial hematologic response at the end of study. Timepoint: At week 27 of the study. Method of measurement: Check CBC. The time to achieve complete or partial hematologic response. Timepoint: weekly. Method of measurement: CBC. The need to platelet or blood transfusion. Timepoint: Week 27. Method of measurement: The proportion of patients with transfusion independence or reduction in transfusion needs among patients who had received a transfusion within 8 weeks prior to the first romiplostim administration. Shiraz University of Medical Sciences Approved 2023-07-05 Ethics Committee of Shiraz University of Medical Sciences 7th floor- Central building of Shiraz University of Medical Sciences- Zand Blvd- Shiraz Shiraz Fars Iran (Islamic Republic of)