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

Severe Aplastic Anemia Clinical Trial

Official title:

A Multicentre, Open-label, Single-arm, Phase II Study to Evaluate the Safety and Efficacy of Hetrombopag Olamine in Patients With Severe Aplastic Anemia

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03557099
• Other study ID #: HR-TPO-SAA-II
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: June 20, 2018
• Est. completion date: July 23, 2020

Study information

• Verified date: April 2020
• Source: Jiangsu HengRui Medicine Co., Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 55
• Est. completion date: July 23, 2020
• Est. primary completion date: October 31, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Anemia
• Anemia, Aplastic
• Severe Aplastic Anemia

Intervention

Drug:
• Hetrombopag Olamine
once daily

Locations

Country	Name	City	State
China	Blood Diseases Hospital, Chinese Academy of Medical Sciences	Tianjin	Tianjin

Sponsors (1)

Lead Sponsor	Collaborator
Jiangsu HengRui Medicine Co., Ltd.

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Proportion of Subjects With Hematological Response	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.	up to 18 weeks