A Study to Evaluate the Safety and Efficacy of Hetrombopag Olamine in Severe Aplastic Anemia （SAA） Patient

NCT03557099 | Completed Phase 1 DMC

• 1 other identifier: HR-TPO-SAA-II
• Study Type: interventional
• Target: 55
• Locations: 1 country
• Sites: 1

Brief Summary

This is a Multicentre, Open-label, single-arm, Phase II Study to Evaluate the Safety and Efficacy of Hetrombopag Olamine in Patients With Severe Aplastic Anemia. 55 adult patients with SAA will be enrolled in the study. Treatment with Hetrombopag will be started at 7.5 mg/day and uptitrated according to the platelet count. The primary objective of the study is to assess the safety and efficacy of Hetrombopag in patients with SAA.

Conditions

Severe Aplastic Anemia

Keywords

SAA

Outcome Measures

Primary Outcomes (1)

• Proportion of Subjects With Hematological Response

  Hematological Response is defined by changes in the platelet count or platelet transfusion requirements, hemoglobin levels or number of red blood cell transfusions, neutrophil counts or G-CSF usage requirements.

  up to 18 weeks

Study Arms (1)

Hetrombopag Olamine

EXPERIMENTAL

Hetrombopag will be started at 7.5 mg/day and uptitrated according to the platelet count.

Drug: Hetrombopag Olamine

Interventions

Hetrombopag Olamine DRUG

once daily

Hetrombopag Olamine

Eligibility Criteria

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

You may qualify if:

• Previous diagnosis of refractory severe aplastic anemia, not meeting partial response criteria after at least one treatment course of immunosuppressive therapy, not suitable or unwilling to do hematopoietic stem cell transplantation.
• Platelet count ≤ 30×109/L.
• Signed informed consent.

You may not qualify if:

• Bleeding and/or Infection not adequately responding to appropriate therapy.
• Patients with a PNH clone size in neutrophils of ≥50%.
• Treatment with immunosuppressive therapy within 6 months prior to study entry.
• Any laboratory or clinical evidence for HIV infection. Any clinical history for hepatitis C infection; chronic hepatitis B infection; or any evidence for active hepatitis at the time of subjects screening.
• ALT\> 2.5 x upper limit of normal (ULN), AST\> 2.5 x upper limit of normal (ULN) DBLI\> 1.5 x upper limit of normal (ULN), Scr\> upper limit of normal (ULN).
• Subjects diagnosed with cirrhosis or portal hypertension.
• Subjects diagnosed with tumor.
• Patients with any prior history of congestive heart failure, arrhythmia, and peripheral arteriovenous thrombosis within 1 year. With a history of myocardial infarction or cerebral infarction within 3 months.
• ECOG Performance Status of 3 or greater.
• Female subjects who are nursing or pregnant within 6 months.
• Subjects cannot take effective contraception.
• Subjects have participated in other clinical trial within the 3 months prior to study entry.

Sponsors & Collaborators

• Jiangsu HengRui Medicine Co., Ltd.: lead

Study Sites (1)

Blood Diseases Hospital, Chinese Academy of Medical Sciences

Tianjin, Tianjin Municipality, China

Location

Related Publications (1)

• Peng G, He G, Chang H, Gao S, Liu X, Chen T, Li P, Han B, Miao M, Ge Z, Ge X, Li F, Li Y, Wang S, Wang Y, Shen Y, Zhang T, Zou J, Zhang F. A multicenter phase II study on the efficacy and safety of hetrombopag in patients with severe aplastic anemia refractory to immunosuppressive therapy. Ther Adv Hematol. 2022 Mar 30;13:20406207221085197. doi: 10.1177/20406207221085197. eCollection 2022.

  PMID: 35371427 DERIVED

MeSH Terms

Conditions

Anemia, Aplastic

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 3, 2018
• First Posted: June 14, 2018
• Study Start: June 20, 2018
• Primary Completion: October 31, 2019
• Study Completion: July 23, 2020
• Last Updated: July 29, 2022

Record last verified: 2020-04

Locations

CN (1)