Romiplostim in Combination With CsA vs. CsA in the Treatment of Newly Diagnosed NSAA

Phase 4

Not yet recruiting

• Conditions: Non Severe Aplastic Anemia
• Interventions: Drug: Romiplostim; Drug: Ciclosporin

• Registration Number: NCT06009497
• Lead Sponsor: Peking Union Medical College Hospital

Brief Summary

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 p...

Detailed Description

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. By binding to the thrombopoietin (TPO) receptor, thrombopoietin receptor agonists (TPO-RAs) can cause co...

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Status Verified: 2023-08
• Study Start: 2023-08
• Study First Submitted: 2023-08-18
• Study First Submitted QC: 2023-08-18
• Last Update Submitted: 2023-08-18
• Study First Posted: 2023-08-24
• Last Update Posted: 2023-08-24
• Primary Completion: 2024-12
• Study Completion: 2025-12

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 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.

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Romiplostim+CsA	Romiplostim	Romiplostim 10 µg/kg, subcutaneous injection, once a week, for at least 3 months. Patients with platelet count ≥50×109/L can stop using, and continue to use with platelet count \< 50×109/L. Ciclosporin 3-5mg/kg/d, adjust the dose to keep trough ciclosporin plasma concentration 100-200ng/ml. Ciclosporin should be used for at least 6 months to evaluate the efficacy. Effective patients will continue to use ciclosporin for at least 1.5 years, followed by a slow reduction.
Romiplostim+CsA	Ciclosporin	Romiplostim 10 µg/kg, subcutaneous injection, once a week, for at least 3 months. Patients with platelet count ≥50×109/L can stop using, and continue to use with platelet count \< 50×109/L. Ciclosporin 3-5mg/kg/d, adjust the dose to keep trough ciclosporin plasma concentration 100-200ng/ml. Ciclosporin should be used for at least 6 months to evaluate the efficacy. Effective patients will continue to use ciclosporin for at least 1.5 years, followed by a slow reduction.
CsA	Ciclosporin	Ciclosporin 3-5mg/kg/d, adjust the dose to keep trough ciclosporin plasma concentration 100-200ng/ml. Ciclosporin should be used for at least 6 months to evaluate the efficacy. Effective patients will continue to use ciclosporin for at least 1.5 years, followed by a slow reduction.

Primary Outcome Measures

Name	Time	Method
complete response rate (CRR)	3, 6 months	Proportion of patients who achieved complete response.
overall response rate (ORR)	3, 6 months	Proportion of patients who achieved complete response and partial response.

Secondary Outcome Measures

Name	Time	Method
adverse event rate	3, 6 months	All adverse events that occur or worsen during treatment, as well as those that occur later but are believed by the investigator to be related to the investigational drug, will be reported.

Trial Locations

Locations (1)

Peking union medical college hospital; 🇨🇳 Beijing, China