Clinical trial NCT05333861: An Observational, Multicenter Study to Evaluate the Safety and Effectiveness of Hetrombopag in Patients With ITP or AA
Official title: An Observational, Multicenter Study to Evaluate the Safety and Effectiveness of Hetrombopag in Patients With ITP or AA
Study type: OBSERVATIONAL
Overall status: NOT_YET_RECRUITING
Conditions: Primary Immune Thrombocytopenia
Aplastic Anemia
Keywords: Hetrombopag
Lead sponsor: Wuhan Union Hospital, China
Sponsor class: OTHER
Collaborators: name: Jiangsu Hengrui Pharmaceutical Co., Ltd.
First posted: 2022-04-19
Enrollment: 1150
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
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

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.

Eligibility criteria: 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

Primary outcomes:
measure: Adverse events
timeFrame: Data will be collected for all routine visits completed during the study period no more than 24 months.
Secondary outcomes:
measure: Number and proportion of patients achieving hematologic response after 3/6/12/24 months treatment
timeFrame: Data will be collected for all routine visits completed during the study period which is no more than 24 months.
measure: The maximum continuous duration and total duration of response
timeFrame: Data will be collected for all routine visits completed during the study period which is no more than 24 months.
measure: Number and proportion of patients requiring rescue medication.
timeFrame: Data will be collected for all routine visits completed during the study period which is no more than 24 months.
measure: Change from enrollment in the 36-Item Short Form Survey (SF-36)
timeFrame: Data will be collected for all routine visits completed during the study period which is no more than 24 months.
measure: Change from enrollment in the use of concomitant ITP/AA medications throughout the study
timeFrame: Data will be collected for all routine visits completed during the study period which is no more than 24 months.
measure: Number and proportion of patients achieving a sustained remission off-treatment (SROT) after discontinuation of hetrombopag
timeFrame: Data will be collected for all routine visits completed during the study period which is no more than 24 months.
Other outcomes:
Locations:
city: Wuhan
country: China
facility: Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
state: Hubei
Study officials:
References: