A Study to Evaluate the Safety and Efficacy of Hetrombopag Olamine in Severe Aplastic Anemia （SAA） Patient

Conditions

Severe Aplastic Anemia

Keywords

SAA

Brief summary

This is a Multicentre, Open-label, single-arm, Phase II Study to Evaluate the Safety and Efficacy of Hetrombopag Olamine in Patients With Severe Aplastic Anemia. 55 adult patients with SAA will be enrolled in the study. Treatment with Hetrombopag will be started at 7.5 mg/day and uptitrated according to the platelet count. The primary objective of the study is to assess the safety and efficacy of Hetrombopag in patients with SAA.

Fengkui Zhang, Xin Zhao, Hong Chang, Bing Han, Sujun Gao et al. (29 authors)

Blood2024-11-051 citations

10.1182/blood-2024-206377

Iron chelation of hetrombopag in aplastic anemia: a post hoc analysis of a phase II study.

Yang W, Zhao X, He G, Chang H, Han B, Gao S, Wang S, Chen T, Li F, Wang Y, Ge X, Fu R, Ge Z, Li Y, Liu H, Liu X, Miao M, Zhang L, Zhang F.

2022-10-115 citations

10.1007/s00277-022-04968-8

A multicenter phase II study on the efficacy and safety of hetrombopag in patients with severe aplastic anemia refractory to immunosuppressive therapy

Guangxin Peng, Guangsheng He, Hong Chang, Sujun Gao, Xinjian Liu et al. (19 authors)

Therapeutic Advances in Hematology2022-01-0129 citations

10.1177/20406207221085197

A Multicenter, Open-Label, Single-Arm, Phase 2 Study to Evaluate the Efficacy and Safety of Hetrombopag in Patients with Severe Aplastic Anemia (SAA)

Fengkui Zhang, Guangxin Peng, Guangsheng He, Hong Chang, Sujun Gao et al. (21 authors)

Blood2020-11-057 citations

10.1182/blood-2020-137319

Interventions

DRUGHetrombopag Olamine

once daily

Study design

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

No

Inclusion criteria

1. Previous diagnosis of refractory severe aplastic anemia, not meeting partial response criteria after at least one treatment course of immunosuppressive therapy, not suitable or unwilling to do hematopoietic stem cell transplantation. 2. Platelet count ≤ 30×109/L. 3. Signed informed consent.

Exclusion criteria

1. Bleeding and/or Infection not adequately responding to appropriate therapy. 2. Patients with a PNH clone size in neutrophils of ≥50%. 3. Treatment with immunosuppressive therapy within 6 months prior to study entry. 4. Any laboratory or clinical evidence for HIV infection. Any clinical history for hepatitis C infection; chronic hepatitis B infection; or any evidence for active hepatitis at the time of subjects screening. 5. ALT\> 2.5 x upper limit of normal (ULN), AST\> 2.5 x upper limit of normal (ULN) DBLI\> 1.5 x upper limit of normal (ULN), Scr\> upper limit of normal (ULN). 6. Subjects diagnosed with cirrhosis or portal hypertension. 7. Subjects diagnosed with tumor. 8. Patients with any prior history of congestive heart failure, arrhythmia, and peripheral arteriovenous thrombosis within 1 year. With a history of myocardial infarction or cerebral infarction within 3 months. 9. ECOG Performance Status of 3 or greater. 10. Female subjects who are nursing or pregnant within 6 months. 11. Subjects cannot take effective contraception. 12. Subjects have participated in other clinical trial within the 3 months prior to study entry.

Design outcomes

Primary

Measure	Time frame	Description
Proportion of Subjects With Hematological Response	up to 18 weeks	Hematological Response is defined by changes in the platelet count or platelet transfusion requirements, hemoglobin levels or number of red blood cell transfusions, neutrophil counts or G-CSF usage requirements.

Countries

China

Outcome results

None listed