Back to resultsA Study to Evaluate the Safety and Efficacy of Hetrombopag Olamine in Severe Aplastic Anemia （SAA） Patient
Primary Purpose
Severe Aplastic Anemia
Status
Completed
Phase
Phase 1
Locations
China
Study Type
Interventional
Intervention
Hetrombopag Olamine
Sponsored by
About this trial
This is an interventional treatment trial for Severe Aplastic Anemia focused on measuring SAA
Eligibility Criteria
Inclusion Criteria:
- 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.
- Platelet count ≤ 30×109/L.
- Signed informed consent.
Exclusion Criteria:
- Bleeding and/or Infection not adequately responding to appropriate therapy.
- Patients with a PNH clone size in neutrophils of ≥50%.
- Treatment with immunosuppressive therapy within 6 months prior to study entry.
- 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.
- 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).
- Subjects diagnosed with cirrhosis or portal hypertension.
- Subjects diagnosed with tumor.
- 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.
- ECOG Performance Status of 3 or greater.
- Female subjects who are nursing or pregnant within 6 months.
- Subjects cannot take effective contraception.
- Subjects have participated in other clinical trial within the 3 months prior to study entry.
Sites / Locations
- Blood Diseases Hospital, Chinese Academy of Medical Sciences
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Hetrombopag Olamine
Arm Description
Hetrombopag will be started at 7.5 mg/day and uptitrated according to the platelet count.
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
Full Information
NCT ID
NCT03557099
First Posted
June 3, 2018
Last Updated
July 28, 2022
Sponsor
Jiangsu HengRui Medicine Co., Ltd.
1. Study Identification
Unique Protocol Identification Number
NCT03557099
Brief Title
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
2. Study Status
Record Verification Date
April 2020
Overall Recruitment Status
Completed
Study Start Date
June 20, 2018 (Actual)
Primary Completion Date
October 31, 2019 (Actual)
Study Completion Date
July 23, 2020 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Jiangsu HengRui Medicine Co., Ltd.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Severe Aplastic Anemia
Keywords
SAA
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
55 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Hetrombopag Olamine
Arm Type
Experimental
Arm Description
Hetrombopag will be started at 7.5 mg/day and uptitrated according to the platelet count.
Intervention Type
Drug
Intervention Name(s)
Hetrombopag Olamine
Intervention Description
once daily
Primary Outcome Measure Information:
Title
Proportion of Subjects With Hematological Response
Description
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.
Time Frame
up to 18 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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.
Platelet count ≤ 30×109/L.
Signed informed consent.
Exclusion Criteria:
Bleeding and/or Infection not adequately responding to appropriate therapy.
Patients with a PNH clone size in neutrophils of ≥50%.
Treatment with immunosuppressive therapy within 6 months prior to study entry.
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.
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).
Subjects diagnosed with cirrhosis or portal hypertension.
Subjects diagnosed with tumor.
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.
ECOG Performance Status of 3 or greater.
Female subjects who are nursing or pregnant within 6 months.
Subjects cannot take effective contraception.
Subjects have participated in other clinical trial within the 3 months prior to study entry.
Facility Information:
Facility Name
Blood Diseases Hospital, Chinese Academy of Medical Sciences
City
Tianjin
State/Province
Tianjin
Country
China
12. IPD Sharing Statement
Citations:
PubMed Identifier
35371427
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.
Results Reference
derived
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A Study to Evaluate the Safety and Efficacy of Hetrombopag Olamine in Severe Aplastic Anemia （SAA） Patient
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