Treatment-naive Severe Aplastic Anemia
Conditions
Brief summary
This is a multicenter, placebo-control, phase 3 study of hetrombopag in patients with treatment-naive severe aplastic anemia. All subjects who have completed or withdrawn from the HR-TPO-SAA-III study will voluntarily participate in this extension study. Subjects will receive the same study drug (hetrombopag or placebo) as in study HR-TPO-SAA-III, with the same doses and administration schedule or with modifications based on the actual conditions. The primary objective of this extension study is to give the subjects participating in the HR-TPO-SAA-III study the continued access to the study drug (hetrombopag or placebo) after the completion of the HR-TPO-SAA-III study.
Interventions
DRUGPlacebo
Placebo; once daily
Hetrombopag Olamine; once daily
Sponsors
Jiangsu HengRui Medicine Co., Ltd.
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
15 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
1. Subjects who have completed or withdrawn from the HR-TPO-SAA-III study 2. Subjects who have signed the informed consent form 3. Female and male subjects of childbearing age who agree to take adequate contraceptive measures during the extension study period and within 28 days after the last dose 4. Subjects who have completed the end-of-treatment evaluation in the original study
Exclusion criteria
1. Any unstable situation or situation that will compromise the safety of the subject 2. Evidence of clonal cytogenetic abnormalities at the end-of-treatment examination of the HR-TPO-SAA-III study 3. Subjects with uncontrollable hemorrhage and/or infection after standard treatment 4. Subjects who have experienced deep vein thrombosis, myocardial infarction, stroke, or peripheral arterial embolism within 1 year 5. Any situation that may compromise the subject and the safety or compliance thereof during the study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The ratio of subjects with clonal evolution at 6 months and 18 months | 6 months and 18 months |
| All SIEs, regardless of whether they are related to the investigational product | 3 years |
| All SAEs, regardless of whether they are related to the investigational product | 3 years |
| All AEs resulting in discontinuation and withdrawal from study | 3 years |
Secondary
| Measure | Time frame |
|---|---|
| Neutrophil count | by 1day visit |
| Reticulocyte count | by 1day visit |
| Red blood cell count | by 1day visit |
| The recurrence rates at 6 months and 18 months | 6 months and 18 months |
| The 6-month and 18-month survival rates of subjects | 6 months and 18 months |
| Hemoglobin | by 1day visit |
| Platelet count | by 1day visit |
| White blood cell count | by 1day visit |
Countries
China
Outcome results
None listed