Extension Study of Hetrombopag in Severe Aplastic Anemia

Extension Study of Hetrombopag Olamine in Patients With Treatment-naive Severe Aplastic Anemia

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 All subjects who have completed or withdrawn from the HR-TPO-SAA-III study will voluntarily participate in this extension study.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

Study Overview

• Status: Recruiting
• Conditions: Treatment-naive Severe Aplastic Anemia
• Intervention / Treatment: Drug: Hetrombopag Olamine; Drug: Placebo

• Study Type: Interventional
• Enrollment (Anticipated): 180
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Yaqi Shen; Phone Number: 021-61053363; Email: yaqi.shen@hengrui.com

Study Locations

• China
  • Tianjin
    • Tianjin, Tianjin, China, 300020
      • Recruiting
      • Blood Diseases Hospital, Chinese Academy of medical Sciences
      • Principal Investigator: Fengkui Zhang, Doctor

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 15 years to 75 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Subjects who have completed or withdrawn from the HR-TPO-SAA-III study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

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Drug: Placebo; Placebo; once daily
Experimental: Hetrombopag Olamine	Drug: Hetrombopag Olamine; Hetrombopag Olamine; once daily

What is the study measuring?

Primary Outcome Measures

Outcome 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 Outcome Measures

Outcome Measure	Time Frame
Red blood cell count	by 1day visit
Hemoglobin	by 1day visit
Platelet 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

Collaborators and Investigators

• Sponsor: Jiangsu HengRui Medicine Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): September 19, 2019
• Primary Completion (Anticipated): November 30, 2024
• Study Completion (Anticipated): December 31, 2024

Study Registration Dates

• First Submitted: July 12, 2021
• First Submitted That Met QC Criteria: July 13, 2021
• First Posted (Actual): July 14, 2021

Study Record Updates

• Last Update Posted (Actual): July 14, 2021
• Last Update Submitted That Met QC Criteria: July 13, 2021
• Last Verified: July 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Bone Marrow Diseases; Hematologic Diseases; Bone Marrow Failure Disorders; Anemia; Anemia, Aplastic
• Other Study ID Numbers: HR-TPO-SAA-III-EXT

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No