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

April 11, 2022 updated by: Wuhan Union Hospital, China
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

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

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 Type

Observational

Enrollment (Anticipated)

1150

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • China
    • Hubei
      • Wuhan, Hubei, China, 430022
        • Union Hospital, Tongji Medical College, Huazhong University of Science and Technology

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • ADULT
  • OLDER_ADULT
  • CHILD

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

The Investigators will attempt to consecutively enroll all eligible patients who present for a routine clinical visit or during a routine visit where the patient is started or continued on hetrombopag treatment

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Full-analysis set (FAS)
The FAS includes all enrolled patients. The FAS will be used for all analyses.
Drug: hetrombopag
According to prescription

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Adverse events
Time Frame: 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
Data will be collected for all routine visits completed during the study period no more than 24 months.

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number and proportion of patients achieving hematologic response after 3/6/12/24 months treatment
Time Frame: 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.
Data will be collected for all routine visits completed during the study period which is no more than 24 months.
The maximum continuous duration and total duration of response
Time Frame: 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
Data will be collected for all routine visits completed during the study period which is no more than 24 months.
Number and proportion of patients requiring rescue medication.
Time Frame: 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.
Data will be collected for all routine visits completed during the study period which is no more than 24 months.
Change from enrollment in the 36-Item Short Form Survey (SF-36)
Time Frame: 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.
Data will be collected for all routine visits completed during the study period which is no more than 24 months.
Change from enrollment in the use of concomitant ITP/AA medications throughout the study
Time Frame: 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.
Data will be collected for all routine visits completed during the study period which is no more than 24 months.
Number and proportion of patients achieving a sustained remission off-treatment (SROT) after discontinuation of hetrombopag
Time Frame: 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.
Data will be collected for all routine visits completed during the study period which is no more than 24 months.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Wuhan Union Hospital, China

Collaborators

Jiangsu Hengrui Pharmaceutical Co., Ltd.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ANTICIPATED)

May 1, 2022

Primary Completion (ANTICIPATED)

May 1, 2026

Study Completion (ANTICIPATED)

May 1, 2026

Study Registration Dates

First Submitted

April 11, 2022

First Submitted That Met QC Criteria

April 11, 2022

First Posted (ACTUAL)

April 19, 2022

Study Record Updates

Last Update Posted (ACTUAL)

April 19, 2022

Last Update Submitted That Met QC Criteria

April 11, 2022

Last Verified

April 1, 2022

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • Hetrombopag-ITP/AA

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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