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

Phase 1

Completed

• Conditions: Severe Aplastic Anemia
• Interventions: Drug: Hetrombopag Olamine

• Registration Number: NCT03557099
• Lead Sponsor: Jiangsu HengRui Medicine Co., Ltd.

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2018-06-03
• Study First Submitted QC: 2018-06-03
• Study First Posted: 2018-06-14
• Study Start: 2018-06-20
• Primary Completion: 2019-10-31
• Status Verified: 2020-04
• Study Completion: 2020-07-23
• Last Update Submitted: 2022-07-28
• Last Update Posted: 2022-07-29

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 55

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Hetrombopag Olamine	Hetrombopag Olamine	Hetrombopag will be started at 7.5 mg/day and uptitrated according to the platelet count.

Primary Outcome Measures

Name	Time	Method
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.

Trial Locations

Locations (1)

Blood Diseases Hospital, Chinese Academy of Medical Sciences; 🇨🇳 Tianjin, Tianjin, China