Extension Study of Hetrombopag in Severe Aplastic Anemia

Phase 3

Recruiting

• Conditions: Treatment-naive Severe Aplastic Anemia
• Interventions: Drug: Hetrombopag Olamine; Drug: Placebo

• Registration Number: NCT04961710
• Lead Sponsor: Jiangsu HengRui Medicine Co., Ltd.

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 All subjects who have completed or withdrawn from the HR-TPO-SAA-III study will voluntarily participate in this extension study.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

Detailed Description

Not available

Study Timeline

• Study Start: 2019-09-19
• Status Verified: 2021-07
• Study First Submitted: 2021-07-12
• Study First Submitted QC: 2021-07-13
• Last Update Submitted: 2021-07-13
• Study First Posted: 2021-07-14
• Last Update Posted: 2021-07-14
• Primary Completion: 2024-11-30
• Study Completion: 2024-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 180

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 Subjects who have completed or withdrawn from the HR-TPO-SAA-III study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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Hetrombopag Olamine	Hetrombopag Olamine	-
Placebo	Placebo	-

Primary Outcome Measures

Name	Time	Method
The ratio of subjects with clonal evolution at 6 months and 18 months	6 months and 18 months
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
All SIEs, regardless of whether they are related to the investigational product	3 years

Secondary Outcome Measures

Name	Time	Method
Hemoglobin	by 1day visit
Platelet count	by 1day visit
Red blood cell count	by 1day visit
White blood cell count	by 1day visit
Neutrophil count	by 1day visit
Reticulocyte count	by 1day visit
The 6-month and 18-month survival rates of subjects	6 months and 18 months
The recurrence rates at 6 months and 18 months	6 months and 18 months

Trial Locations

Locations (1)

Blood Diseases Hospital, Chinese Academy of Medical Sciences; 🇨🇳 Tianjin, Tianjin, China