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

NCT06004791 | Unknown Phase 4

• 1 other identifier: rHCsA
• Study Type: interventional
• Target: 90
• Locations: 1 country
• Sites: 1

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.

Conditions

Aplastic Anemia; Drug Effect

Outcome Measures

Primary Outcomes (2)

• ORR at 3 Months

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

  Week 14

• ORR at 6 Months

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

  Week 26

Secondary Outcomes (2)

• 3 months of drug safety

  Week 14

• 6 months of drug safety

  Week 26

Study Arms (2)

Herombopag + CsA

PLACEBO COMPARATOR

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.

Drug: Herombopag + CsA

rhTPO combined with Herombopag + CsA

EXPERIMENTAL

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

Drug: rhTPO combined with Herombopag + CsA

Interventions

Herombopag + CsA DRUG

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

Also known as: control

Herombopag + CsA

rhTPO combined with Herombopag + CsA 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)

Also known as: experimental

rhTPO combined with Herombopag + CsA

Eligibility Criteria

• Age: 18 Years - 85 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Peking Union Medical College Hospital: lead

Study Sites (1)

Peking union medical college hospital

Beijing, China

Location

MeSH Terms

Conditions

Anemia, Aplastic

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Central Study Contacts

Bing Han, Doctor

CONTACT

+8601069151235; hanbing_li@sina.com

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: August 16, 2023
• First Posted: August 22, 2023
• Study Start: August 1, 2023
• Primary Completion: August 1, 2024
• Study Completion: August 31, 2025
• Last Updated: August 22, 2023

Record last verified: 2023-08

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Time Frame: always
• Access Criteria: email request

Locations

CN (1)