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

NCT ID: NCT06004791

Last Updated: 2023-08-22

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE4
• Total Enrollment: 90 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-08-31
• Study Completion Date: 2025-08-31

Brief 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.

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. 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.

Conditions

Aplastic Anemia; Drug Effect

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Herombopag + CsA

Herombopag 10mg/ day, adjust the dose according to the blood image, the course of treatment is at least 3 months, CsA: 3-5 mg/kg/d, adjust the valley concentration 100-200ng/ml, at least 6 months, effective patients continue to use for 1 year, and then gradually reduce the dose.

Group Type: PLACEBO_COMPARATOR

Herombopag + CsA

Intervention Type: DRUG

Herombopag（10mg/d）+CsA(3-5 mg/kg/d, adjust the grain concentration 100-200ng/ml)

rhTPO combined with Herombopag + CsA

Administer rhTPO (15000U, subcutaneously once daily for 7 days, once a month for 3 months),Herombopag 10mg/day, adjust dose according to blood picture for at least 3 months, CsA: 3-5 mg/kg/d, adjust trough concentration 100-200ng/ml for at least 6 months, continue for 1 year if effective, then gradually reduce dose

Group Type: EXPERIMENTAL

rhTPO combined with Herombopag + CsA

Intervention Type: DRUG

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)

Interventions

Herombopag + CsA

Herombopag（10mg/d）+CsA(3-5 mg/kg/d, adjust the grain concentration 100-200ng/ml)

Intervention Type: DRUG

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)

Intervention Type: DRUG

Other Intervention Names

control; experimental

Eligibility Criteria

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 85 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

Peking Union Medical College Hospital

OTHER

Sponsor Role: lead

Responsible Party

Bing Han

Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Locations

Peking union medical college hospital

Beijing, , China

Countries

China

Central Contacts

Bing Han, Doctor

Role: CONTACT

hanbing_li@sina.com

+8601069151235

Other Identifiers

rHCsA

Identifier Type: -

Identifier Source: org_study_id