Hetrombopag or Placebo in Treatment-Naive Severe Aplastic Anemia
Primary Purpose
Severe Aplastic Anemia
Status
Recruiting
Phase
Phase 3
Locations
China
Study Type
Interventional
Intervention
Hetrombopag Olamine+Standard Therapy
Placebo+Standard Therapy
Sponsored by
About this trial
This is an interventional treatment trial for Severe Aplastic Anemia focused on measuring SAA
Eligibility Criteria
Inclusion Criteria:
1.15 Years to 75 Years (weight greater than 50 kg if Age < 18 years old). 2.Severe aplastic anemia characterized by Bone marrow cell proliferation less than 25 percent (If ≥25% but <50%, the remaining hematopoietic cells should be <30%) AND At least two of the following: Absolute neutrophil count <0.5×109/L; Platelet count <20×109/L; Absolute reticulocyte count <20×109/L.
3.Unsuitable or unwilling to perform hematopoietic stem cell transplantation (HSCT).
4.Signed informed consent.
Exclusion Criteria:
- Diagnosis of whole blood cell reduction due to other causes or bone marrow hypoproliferative diseases.
- Subjects who have previously received immunosuppressive therapy with mycophenolate mofetil, sirolimus, high dose cyclophosphamide (≥45mg/kg/d), alemtuzumab, etc;or have treated with thrombopoietin receptor agonist (eg, eltrombopag, romiplostim, rhTPO, etc.) prior to randomization.
- Previous history of hematopoietic stem cell transplantation.
- Subjects who is known or suspected of contraindications or hypersensitivity to Hetrombopag's API (Active Pharmaceutical Ingredient).
- Evidence of clonal cytogenetic abnormalities at the time of screening.
- Bleeding and/or Infection not adequately responding to appropriate therapy.
- 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 with uncontrolled hypertension (>180/100mmHg), severe arrhythmia (such as complete left bundle branch block, QT interval prolongation (Bazetts formula), torsade ventricular tachycardia, etc.), unstable angina, pulmonary hypertension at the time of screening.
- Subjects diagnosed with cirrhosis or portal hypertension.
- Subjects with malignant solid tumors of any organ system within 5 years prior to screening, with or without treatment, metastasis or recurrence, except for local cutaneous basal cell carcinoma; subjects with hematological tumors found previously or during screening.
- Subjects with deep vein thrombosis, myocardial infarction, stroke or peripheral arterial embolization within 12 months prior to randomization.
- Female subjects who are nursing or pregnant.
- Subjects cannot comply with 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 SciencesRecruiting
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Hetrombopag Olamine+Standard Therapy
Placebo+Standard Therapy
Arm Description
Outcomes
Primary Outcome Measures
Rate of complete hematologic response at six months.
Secondary Outcome Measures
Full Information
NCT ID
NCT03825744
First Posted
January 30, 2019
Last Updated
July 28, 2022
Sponsor
Jiangsu HengRui Medicine Co., Ltd.
1. Study Identification
Unique Protocol Identification Number
NCT03825744
Brief Title
Hetrombopag or Placebo in Treatment-Naive Severe Aplastic Anemia
Official Title
A Phase III Multicenter Randomized Study of Hetrombopag or Placebo in Treatment-Naive Severe Aplastic Anemia
Study Type
Interventional
2. Study Status
Record Verification Date
July 2022
Overall Recruitment Status
Recruiting
Study Start Date
March 5, 2019 (Actual)
Primary Completion Date
December 2022 (Anticipated)
Study Completion Date
December 2022 (Anticipated)
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
No
5. Study Description
Brief Summary
This is a multicenter, randomized, placebo-control, phase III study to investigate hetrombopag in subjects with severe AA who are treated naive.
180 treated naive patients with SAA will be enrolled in the study. The primary objective of the study will be the rate of complete hematologic response at six months.
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 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
180 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Hetrombopag Olamine+Standard Therapy
Arm Type
Experimental
Arm Title
Placebo+Standard Therapy
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
Hetrombopag Olamine+Standard Therapy
Intervention Description
once daily
Intervention Type
Drug
Intervention Name(s)
Placebo+Standard Therapy
Intervention Description
once daily
Primary Outcome Measure Information:
Title
Rate of complete hematologic response at six months.
Time Frame
6 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
15 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
1.15 Years to 75 Years (weight greater than 50 kg if Age < 18 years old). 2.Severe aplastic anemia characterized by Bone marrow cell proliferation less than 25 percent (If ≥25% but <50%, the remaining hematopoietic cells should be <30%) AND At least two of the following: Absolute neutrophil count <0.5×109/L; Platelet count <20×109/L; Absolute reticulocyte count <20×109/L.
3.Unsuitable or unwilling to perform hematopoietic stem cell transplantation (HSCT).
4.Signed informed consent.
Exclusion Criteria:
Diagnosis of whole blood cell reduction due to other causes or bone marrow hypoproliferative diseases.
Subjects who have previously received immunosuppressive therapy with mycophenolate mofetil, sirolimus, high dose cyclophosphamide (≥45mg/kg/d), alemtuzumab, etc;or have treated with thrombopoietin receptor agonist (eg, eltrombopag, romiplostim, rhTPO, etc.) prior to randomization.
Previous history of hematopoietic stem cell transplantation.
Subjects who is known or suspected of contraindications or hypersensitivity to Hetrombopag's API (Active Pharmaceutical Ingredient).
Evidence of clonal cytogenetic abnormalities at the time of screening.
Bleeding and/or Infection not adequately responding to appropriate therapy.
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 with uncontrolled hypertension (>180/100mmHg), severe arrhythmia (such as complete left bundle branch block, QT interval prolongation (Bazetts formula), torsade ventricular tachycardia, etc.), unstable angina, pulmonary hypertension at the time of screening.
Subjects diagnosed with cirrhosis or portal hypertension.
Subjects with malignant solid tumors of any organ system within 5 years prior to screening, with or without treatment, metastasis or recurrence, except for local cutaneous basal cell carcinoma; subjects with hematological tumors found previously or during screening.
Subjects with deep vein thrombosis, myocardial infarction, stroke or peripheral arterial embolization within 12 months prior to randomization.
Female subjects who are nursing or pregnant.
Subjects cannot comply with effective contraception.
Subjects have participated in other clinical trial within the 3 months prior to study entry.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Ya-Qi Shen
Phone
+86-18036617093
Email
shenyaqi@hrglobe.cn
Facility Information:
Facility Name
Blood Diseases Hospital, Chinese Academy of Medical Sciences
City
Tianjin
State/Province
Tianjin
ZIP/Postal Code
300041
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Fengkui Zhang
Email
zhfk@hotmail.com
12. IPD Sharing Statement
Learn more about this trial
Hetrombopag or Placebo in Treatment-Naive Severe Aplastic Anemia
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