Romiplostim in Combination With Ciclosporin Versus Ciclosporin in the Treatment of Newly Diagnosed Non-severe Aplastic Anemia

Romiplostim-1

Aplastic anemia (AA) is a group of clinical syndromes caused by a significant decrease in bone marrow hematopoietic tissue from different etiologies, resulting in hematopoietic failure. Treatment options for patients with aplastic anemia are very limited. In a phase II/III, multicenter, open-label study exploring the efficacy and safety of romiplostim, the primary endpoint showed an overall response rate of 84% \[95% CI 66-95%\] at week 27. However, there are no prospective clinical data exploring whether romiplostim combined with ciclosporin (CsA) can further improve efficacy than ciclosporin monotherapy in newly diagnosed NSAA. Therefore, we aimed to compare the efficacy and safety of romiplostim in combination with CsA versus CsA monotherapy.

2023-08-01

2024-12-01

2025-12-01

Not yet recruiting

Early phase I

44

Inclusion Criteria: 1. Age ≥18 years old. 2. Clearly diagnosed as untreated NSAA. 3. At least one of the following conditions was met at the time of enrollment: hemoglobin \<90 g/L. Platelet \<30×109/L, neutrophils \<1.0×109/L. 4. Baseline liver and kidney function (ALT, AST, Cr) was less than 2 times the normal value. 5. No active infection; Not pregnant or breastfeeding. 6. Agree to sign the consent form. 7. The Eastern Cancer Collaboration Group (ECOG) score was 0-2. Exclusion Criteria: 1. Pancytopenia caused by other causes, such as myelodysplastic syndrome (MDS). 2. There is cytogenetic evidence of clonal hematologic bone marrow diseases (MDS, AML). 3. PNH clone ≥50%. 4. Had received hematopoietic stem cell transplantation (HSCT) before enrollment. 5. Immunosuppressive therapy such as ATG or cyclosporine use for more than 2 weeks. 6. Infection or bleeding that is not controlled by standard treatment. 7. Allergic to recombinant TPO or Hitrepopar. 8. Active HIV, HCV, HBV infection or cirrhosis, or portal hypertension. 9. Any concomitant malignancy or local basal cell carcinoma of the skin within 5 years. 10. Previous history of thromboembolic events, heart attack or stroke (including antiphospholipid antibody syndrome), and current use of anticoagulants. 11. Women who are pregnant or nursing (lactation). 12. Have participated in other clinical trials within 3 months

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE4'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 44, 'type': 'ESTIMATED'}}

2023-08-24