An Observational, Multicenter Study to Evaluate the Safety and Effectiveness of Hetrombopag in Patients With ITP or AA

Not yet recruiting

• Conditions: Primary Immune Thrombocytopenia; Aplastic Anemia
• Interventions: Drug: hetrombopag

• Registration Number: NCT05333861
• Lead Sponsor: Wuhan Union Hospital, China

Brief Summary

This is a multi-center, observational study in patients with Immune Thrombocytopenia (ITP) or aplastic anemia(AA) designed to describe the real-world safety and effectiveness of hetrombopag and assess the patterns of drug utilization to add to the knowledge base regarding the use of hetrombopag in routine medical practice. Patients eligible for participation will, as part of their routine medical care, be receiving hetrombopag for the treatment of ITP/AA

Detailed Description

This is a multi-center, observational study in patients with ITP/AA designed to describe the real-world safety and effectiveness of hetrombopag and assess the patterns of drug utilization to add to the knowledge base regarding the use of hetrombopag in routine medical practice.

Patients eligible for participation will, as part of their routine medical care, be receiving hetrombopag for the treatment of ITP/AA

The scope of the study is to collect both retrospective and prospective data. The main part of the study will be prospective collecting data on usage, effectiveness, safety and patient-reported outcomes whereas the retrospective part will consist of collection of information on previous treatments, reason for treatment switch and, if applicable, hetrombopag treatment prior to enrollment. The retrospective data collection will be based on the information available in the patient's medical records. Data will be collected for up to 12 months prior to hetrombopag treatment start.

Prospective data will be collected at routine clinical visits throughout the study. Patients will be followed for 24 months and will be enrolled until their first scheduled visit after 24 months of enrollment, or until early termination, whichever occurs first.

Study Timeline

• Status Verified: 2022-04
• Study First Submitted: 2022-04-11
• Study First Submitted QC: 2022-04-11
• Last Update Submitted: 2022-04-11
• Study First Posted: 2022-04-19
• Last Update Posted: 2022-04-19
• Study Start: 2022-05-01
• Primary Completion: 2026-05-01
• Study Completion: 2026-05-01

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 1150

Inclusion Criteria

• 1.Established and well documented ITP diagnosis
• 2.Patient is treated with, or at enrollment prescribed hetrombopag for ITP. Decision to initiate treatment shall be made by the treating physician and independently from the decision to include the patient in the study.
• 3.Signed and dated informed consent provided by the patient before any study-related activities are undertaken
• 4.Willing and able to comply with protocol requirements

Exclusion Criteria

• Enrollment in a concurrent clinical interventional study

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Full-analysis set (FAS)	hetrombopag	The FAS includes all enrolled patients. The FAS will be used for all analyses.

Primary Outcome Measures

Name	Time	Method
Adverse events	Data will be collected for all routine visits completed during the study period no more than 24 months.	Information will be collected via reports from the Investigators based on the patient's medical records and classified according to the National Cancer Institute Common Terminology Criteria for Adverse Events, version 5.0

Secondary Outcome Measures

Name	Time	Method
Number and proportion of patients achieving hematologic response after 3/6/12/24 months treatment	Data will be collected for all routine visits completed during the study period which is no more than 24 months.	Laboratory measures of CBC will be collected if performed according to routine clinical practice and available in the patient's medical records. All analyses of platelet counts and other hematologic assessments will be based on local laboratory results.
The maximum continuous duration and total duration of response	Data will be collected for all routine visits completed during the study period which is no more than 24 months.	Hematologic assessments will be based on local laboratory results
Number and proportion of patients requiring rescue medication.	Data will be collected for all routine visits completed during the study period which is no more than 24 months.	Information will be collected via the patient's medical records.
Change from enrollment in the 36-Item Short Form Survey (SF-36)	Data will be collected for all routine visits completed during the study period which is no more than 24 months.	SF-36 is a set of generic, coherent, and easily administered quality-of-life measures. These measures rely upon patient self-reporting.
Change from enrollment in the use of concomitant ITP/AA medications throughout the study	Data will be collected for all routine visits completed during the study period which is no more than 24 months.	Information will be collected via the patient's medical records.
Number and proportion of patients achieving a sustained remission off-treatment (SROT) after discontinuation of hetrombopag	Data will be collected for all routine visits completed during the study period which is no more than 24 months.	The proportion of responders that were able to taper and discontinue hetrombopag maintaining the response during a period of observation of at least six months.

Trial Locations

Locations (1)

Union Hospital, Tongji Medical College, Huazhong University of Science and Technology; 🇨🇳 Wuhan, Hubei, China