Clinical trial NCT04961710: Extension Study of Hetrombopag in Severe Aplastic Anemia
Official title: Extension Study of Hetrombopag Olamine in Patients With Treatment-naive Severe Aplastic Anemia
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Treatment-naive Severe Aplastic Anemia
Lead sponsor: Jiangsu HengRui Medicine Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2021-07-14
Enrollment: 157
Sex: ALL
Minimum age (years): 15
Maximum age (years): 75
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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 All subjects who have completed or withdrawn from the HR-TPO-SAA-III study will voluntarily participate in this extension study.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.


Eligibility criteria: 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 Subjects who have completed or withdrawn from the HR-TPO-SAA-III study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

Primary outcomes:
measure: The ratio of subjects with clonal evolution at 6 months and 18 months
timeFrame: 6 months and 18 months
measure: All SIEs, regardless of whether they are related to the investigational product
timeFrame: 3 years
measure: All SAEs, regardless of whether they are related to the investigational product
timeFrame: 3 years
measure: All AEs resulting in discontinuation and withdrawal from study
timeFrame: 3 years
Secondary outcomes:
measure: Red blood cell count
timeFrame: by 1day visit
measure: Hemoglobin
timeFrame: by 1day visit
measure: Platelet count
timeFrame: by 1day visit
measure: White blood cell count
timeFrame: by 1day visit
measure: Neutrophil count
timeFrame: by 1day visit
measure: Reticulocyte count
timeFrame: by 1day visit
measure: The 6-month and 18-month survival rates of subjects
timeFrame: 6 months and 18 months
measure: The recurrence rates at 6 months and 18 months
timeFrame: 6 months and 18 months
Other outcomes:
Locations:
city: Tianjin
country: China
facility: Blood Diseases Hospital, Chinese Academy of Medical Sciences
state: Tianjin
Study officials:
References: