Extension Study of Hetrombopag in Severe Aplastic Anemia

NCT04961710 | Active Not Recruiting Phase 3

• 1 other identifier: HR-TPO-SAA-III-EXT
• Study Type: interventional
• Target: 157
• Locations: 1 country
• Sites: 1

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 All subjects who have completed or withdrawn from the HR-TPO-SAA-III study will voluntarily participate in this extension study.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

Conditions

Treatment-naive Severe Aplastic Anemia

Outcome Measures

Primary Outcomes (4)

• 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 Outcomes (8)

• 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

Study Arms (2)

Hetrombopag Olamine

EXPERIMENTAL

Drug: Hetrombopag Olamine

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

Hetrombopag Olamine DRUG

Hetrombopag Olamine; once daily

Hetrombopag Olamine

Placebo DRUG

Placebo; once daily

Placebo

Eligibility Criteria

• Age: 15 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects who have completed or withdrawn from the HR-TPO-SAA-III study
• Subjects who have signed the informed consent form
• 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
• Subjects who have completed the end-of-treatment evaluation in the original study

You may not qualify if:

• Any unstable situation or situation that will compromise the safety of the subject
• Evidence of clonal cytogenetic abnormalities at the end-of-treatment examination of the HR-TPO-SAA-III study
• Subjects with uncontrollable hemorrhage and/or infection after standard treatment
• Subjects who have experienced deep vein thrombosis, myocardial infarction, stroke, or peripheral arterial embolism within 1 year
• Any situation that may compromise the subject and the safety or compliance thereof during the study

Sponsors & Collaborators

• Jiangsu HengRui Medicine Co., Ltd.: lead

Study Sites (1)

Blood Diseases Hospital, Chinese Academy of Medical Sciences

Tianjin, Tianjin Municipality, 300020, China

Location

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 12, 2021
• First Posted: July 14, 2021
• Study Start: September 19, 2019
• Primary Completion: November 30, 2024
• Study Completion: December 31, 2024
• Last Updated: September 19, 2024

Record last verified: 2024-09

Locations

CN (1)