Clinical trial NCT03557099: A Study to Evaluate the Safety and Efficacy of Hetrombopag Olamine in Severe Aplastic Anemia （SAA） Patient
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Severe Aplastic Anemia
Keywords: SAA
Lead sponsor: Jiangsu HengRui Medicine Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2018-06-14
Enrollment: 55
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Proportion of Subjects With Hematological Response
timeFrame: up to 18 weeks
Secondary outcomes:
Other outcomes:
Locations:
city: Tianjin
country: China
facility: Blood Diseases Hospital, Chinese Academy of Medical Sciences
state: Tianjin
Study officials:
References:
citation: Peng G, He G, Chang H, Gao S, Liu X, Chen T, Li P, Han B, Miao M, Ge Z, Ge X, Li F, Li Y, Wang S, Wang Y, Shen Y, Zhang T, Zou J, Zhang F. A multicenter phase II study on the efficacy and safety of hetrombopag in patients with severe aplastic anemia refractory to immunosuppressive therapy. Ther Adv Hematol. 2022 Mar 30;13:20406207221085197. doi: 10.1177/20406207221085197. eCollection 2022.