A Prospective, Randomized, Controlled Study of rhTPO in Combination With Herombopag + CsA vs Herombopag + CsA for the Treatment of Primary TD-NSAA

Aplastic Anemia Clinical Trial

Official title:

A Prospective, Randomized, Controlled Study of rhTPO in Combination With Herombopag + CsA vs Herombopag + CsA for the Treatment of Primary Transfusion-dependent NSAA

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06004791
• Other study ID #: rHCsA
• Status: Not yet recruiting
• Phase: Phase 4
• Start date: August 2023
• Est. completion date: August 31, 2025

Study information

• Verified date: August 2023
• Source: Peking Union Medical College Hospital
• Contact: Bing Han, Doctor
• Phone: +8601069151235
• Email: hanbing_li[@]sina.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Aplastic anemia (AA) is a group of clinical syndromes. Treatment options are very limited. The results of a previous clinical study showed good efficacy and a high safety profile of herombopag in improving thrombocytopenia, but this result needs to be supported by more data. In our study, patients who were willing to participate in this study and were diagnosed with transfusion-dependent non-heavy aplastic anemia were randomized to the rhTPO combined with herombopag + cyclosporine group and given rhTPO (at a dose of 1500 U by subcutaneous injection once daily for 7 d, 28 d for 3 courses) +Herombopag(10 mg/day for 3 months) + cyclosporine (3-5 mg/kg/d for 3 months). -5 mg/kg/d for at least 6 months) and herombopag + cyclosporine (10 mg/day for 3 months) + cyclosporine (3-5 mg/kg/d for at least 6 months) in the herombopag+ cyclosporine group to observe the efficacy and safety.

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. The prevalence of AA in China is 7.4 per 1 million. It peaks in the 15-25 and 60+ age groups and is more common in men than women. Treatment options are very limited. The results of a previous clinical study showed good efficacy and a high safety profile of herombopag in improving thrombocytopenia, but this result needs to be supported by more data. In our study, patients who were willing to participate in this study and were diagnosed with transfusion-dependent non-heavy aplastic anemia were randomized to the rhTPO combined with herombopag + cyclosporine group and given rhTPO (at a dose of 1500 U by subcutaneous injection once daily for 7 d, 28 d for 3 courses) +Herombopag(10 mg/day for 3 months) + cyclosporine (3-5 mg/kg/d for 3 months). -5 mg/kg/d for at least 6 months) and Herombopag + cyclosporine (10 mg/day for 3 months) + cyclosporine (3-5 mg/kg/d for at least 6 months) in the herombopag+ cyclosporine group to observe the efficacy and safety.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 90
• Est. completion date: August 31, 2025
• Est. primary completion date: August 2024
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years to 85 Years

Eligibility

Inclusion Criteria:

1. Age =18 years old.

2. Clearly diagnosed 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 or 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.

Related Conditions & MeSH terms

• Anemia, Aplastic
• Aplastic Anemia
• Drug Effect

Intervention

Drug:
• Herombopag + CsA
Herombopag(10mg/d)+CsA(3-5 mg/kg/d, adjust the grain concentration 100-200ng/ml)
• rhTPO combined with Herombopag + CsA
rhTPO (15000U, subcutaneous injection, once a day for 7 days, once a month for 3 months),Herombopag(10mg/d)+CsA(3-5 mg/kg/d, adjust the grain concentration 100-200ng/ml)

Locations

Country	Name	City	State
China	Peking union medical college hospital	Beijing

Sponsors (1)

Lead Sponsor	Collaborator
Peking Union Medical College Hospital

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	ORR at 3 Months	After 3 months of treatment, ORR was calculated by measuring platelets, hemoglobin, neutrophils, and transfusion independence	Week 14
Primary	ORR at 6 Months	After 6 months of treatment, ORR was calculated by measuring platelets, hemoglobin, neutrophils, and transfusion independence	Week 26
Secondary	3 months of drug safety	Assessing the incidence and severity of adverse events; All adverse events that occur or worsen within 3 months of treatment will be reported	Week 14
Secondary	6 months of drug safety	Assessing the incidence and severity of adverse events; All adverse events that occur or worsen within 6 months of treatment will be reported	Week 26