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

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Primary Purpose

Status

Not yet recruiting

Phase

Phase 4

Locations

China

Study Type

Interventional

Intervention

Herombopag + CsA

rhTPO combined with Herombopag + CsA

About this trial

This is an interventional treatment trial for Aplastic Anemia

Eligibility Criteria

18 Years - 85 Years (Adult, Older Adult)All SexesAccepts Healthy Volunteers

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

Sites / Locations

Peking union medical college hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Placebo Comparator

Experimental

Arm Label

Herombopag + CsA

rhTPO combined with Herombopag + CsA

Arm Description

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.

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

Outcomes

Primary Outcome Measures

ORR at 3 Months

After 3 months of treatment, ORR was calculated by measuring platelets, hemoglobin, neutrophils, and transfusion independence

ORR at 6 Months

After 6 months of treatment, ORR was calculated by measuring platelets, hemoglobin, neutrophils, and transfusion independence

Secondary Outcome Measures

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

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

Full Information

NCT ID

NCT06004791

First Posted

August 16, 2023

Last Updated

August 16, 2023

Sponsor

Peking Union Medical College Hospital

1. Study Identification

Unique Protocol Identification Number

NCT06004791

Brief Title

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

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

Study Type

Interventional

2. Study Status

Record Verification Date

August 2023

Overall Recruitment Status

Not yet recruiting

Study Start Date

August 2023 (Anticipated)

Primary Completion Date

August 2024 (Anticipated)

Study Completion Date

August 31, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Peking Union Medical College Hospital

4. Oversight

Studies a U.S. FDA-regulated Drug Product

No

Studies a U.S. FDA-regulated Device Product

No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Aplastic Anemia, Drug Effect

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 4

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Randomized

Enrollment

90 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Herombopag + CsA

Arm Type

Placebo Comparator

Arm Description

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.

Arm Title

rhTPO combined with Herombopag + CsA

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

Herombopag + CsA

Other Intervention Name(s)

control

Intervention Description

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

Intervention Type

Drug

Intervention Name(s)

rhTPO combined with Herombopag + CsA

Other Intervention Name(s)

experimental

Intervention Description

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)

Primary Outcome Measure Information:

Title

ORR at 3 Months

Description

After 3 months of treatment, ORR was calculated by measuring platelets, hemoglobin, neutrophils, and transfusion independence

Time Frame

Week 14

Title

ORR at 6 Months

Description

After 6 months of treatment, ORR was calculated by measuring platelets, hemoglobin, neutrophils, and transfusion independence

Time Frame

Week 26

Secondary Outcome Measure Information:

Title

3 months of drug safety

Description

Assessing the incidence and severity of adverse events; All adverse events that occur or worsen within 3 months of treatment will be reported

Time Frame

Week 14

Title

6 months of drug safety

Description

Assessing the incidence and severity of adverse events; All adverse events that occur or worsen within 6 months of treatment will be reported

Time Frame

Week 26

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

85 Years

Accepts Healthy Volunteers

Eligibility Criteria

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

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Bing Han, Doctor

Phone

+8601069151235

Email

hanbing_li@sina.com

Facility Information:

Facility Name

Peking union medical college hospital

City

Beijing

Country

China

12. IPD Sharing Statement

Plan to Share IPD

Yes

IPD Sharing Plan Description

individual participant data would be accepted upon request

IPD Sharing Time Frame

always

IPD Sharing Access Criteria

email request

Aplastic Anemia, Drug Effect

About this trial

Eligibility Criteria

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

1. Study Identification

2. Study Status

3. Sponsor/Collaborators

4. Oversight

5. Study Description

6. Conditions and Keywords

7. Study Design

8. Arms, Groups, and Interventions

10. Eligibility

12. IPD Sharing Statement

Learn more about this trial