Herombopag Added to Cyclosporine in Non Severe Aplastic Anemia

NCT ID: NCT05660785

Last Updated: 2025-08-05

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 54 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-12-01
• Study Completion Date: 2025-01-28

Brief Summary

This is a prospective, multicenter, single-arm, phase 2 trial. The aim of this study is to evaluate the efficacy and safety of herombopag combined with cyclosporine for patients with non severe aplastic anemia (NSAA).

Detailed Description

This study aims to improve the 24 weeks response rate. The sample size is calculated based on Simon's two-stage design. The first stage of the study enrolled a cohort of 15 patients. If after 24 weeks at least 9 patients achieved a response, then enrollment was expanded to a total of 43 patients. The null hypothesis was unaccepted if more than 26 of 43 patients achieved the response. Accounting for a 20% dropout rate, the estimated final sample size was 54 patients.

Conditions

Non Severe Aplastic Anemia; Untreated

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

CsA + Herombopag

Herombopag combined with cyclosporine

Group Type: EXPERIMENTAL

Herombopag

Intervention Type: DRUG

Hetrombopag is a TPO receptor agonist approved in China in 2021 for idiopathic thrombocytopenic purpura (ITP) and second-line severe aplastic anemia (SAA). Indications of chemotherapy-induced thrombocytopenia (CIT), pediatric/juvenile ITP and naive severe aplastic anemia are under development. Hetrombopag was granted Orphan Drug Designation by FDA for the treatment of CIT.

Cyclosporine A is a calcineurin inhibitor, which has an effect on reducing T-cell proliferation and activation, can reverse pancytopenia and alleviate transfusion requirements in NSAA.

Interventions

Herombopag

Hetrombopag is a TPO receptor agonist approved in China in 2021 for idiopathic thrombocytopenic purpura (ITP) and second-line severe aplastic anemia (SAA). Indications of chemotherapy-induced thrombocytopenia (CIT), pediatric/juvenile ITP and naive severe aplastic anemia are under development. Hetrombopag was granted Orphan Drug Designation by FDA for the treatment of CIT.

Cyclosporine A is a calcineurin inhibitor, which has an effect on reducing T-cell proliferation and activation, can reverse pancytopenia and alleviate transfusion requirements in NSAA.

Intervention Type: DRUG

Other Intervention Names

Cyclosporine A

Eligibility Criteria

Inclusion Criteria

• Willing and able to comply with the requirements for this study and written informed consent.
• Male or female age ≥ 18 years
• Diagnosis of untreated non severe aplastic anemia.
• Platelet counts < 50 x 10^9/L at least 2 times consecutively (time interval ≥ 1 week)

Exclusion Criteria

• Receive immunosuppressive therapy more than 4 weeks before enrollment
• Treatment with TPO-RA within 1 week before enrollment
• Inherited bone marrow failure syndromes
• Bone marrow fibrosis grade ≥ 2
• The presence of hemolytic PNH clone
• The presence of clonal karyotypic abnormalities (del(20q), +8 and -Y are not included in this category)
• Previously treated with TPO-RA ≥ 4 weeks
• Previously received immunosuppressive therapy ≥ 12 weeks
• Ferritin > 1000 ng/ml (The increased level of Ferritin led by infection is not included in this category)
• Have an allergy to eltrombopag or any other part of this medicine.
• History of radiotherapy and chemotherapy for malignant solid tumors
• Cytopenia caused by other non-hematologic diseases, including liver cirrhosis, active rheumatic connective tissue disease, and persistence of infectious diseases, etc
• Abnormal liver function: ALT or AST > 3 ULN, or TBil > 1.5 ULN after treatment.
• Abnormal kidney function: Creatinine clearance < 30 ml/min, or serum creatinine (sCr) >1.5 ULN
• Patients with diabetic nephropathy, neuropathy, or eye disease
• Patients with poorly controlled hypertension or cardiac arrhythmia
• Patients with congestive heart failure and the NYHA grade ≥ 3 historically or currently, and LVEF < 45% within 4 weeks before enrollment
• History of arteriovenous thrombosis within 1 year before enrollment
• Participation in another clinical trial within 4 months before the start of this trial
• Pregnant or breast-feeding patients
• Patients considered to be ineligible for the study by the investigator for reasons other than the above

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Jiangsu Hengrui Pharmaceutical Co., Ltd.

INDUSTRY

Sponsor Role: collaborator

Institute of Hematology & Blood Diseases Hospital, China

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Tangshan Central Hospital

Tangshan, Hebei, China

Zhoukou Central Hospital

Zhoukou, Henan, China

The Second Affilated Hospital of Shandong First Medical University

Tai’an, Shandong, China

Regenerative Medicine Center

Tianjin, Tianjin Municipality, China

Countries

China

Other Identifiers

IIT2022049

Identifier Type: -

Identifier Source: org_study_id