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

NCT05333861 | Not Yet Recruiting

• 1 other identifier: Hetrombopag-ITP/AA
• Study Type: observational
• Target: 1,150
• Locations: 1 country
• Sites: 1

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

Conditions

Primary Immune Thrombocytopenia; Aplastic Anemia

Keywords

Hetrombopag

Outcome Measures

Primary Outcomes (1)

• Adverse events

  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 Outcomes (6)

• 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.

• 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.

• 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.

• 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.

• 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.

Study Arms (1)

Full-analysis set (FAS)

The FAS includes all enrolled patients. The FAS will be used for all analyses.

Drug: hetrombopag

Interventions

hetrombopag DRUG

According to prescription

Full-analysis set (FAS)

Eligibility Criteria

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

You may qualify if:

• Established and well documented ITP diagnosis
• 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.
• Signed and dated informed consent provided by the patient before any study-related activities are undertaken
• Willing and able to comply with protocol requirements

You may not qualify if:

• Enrollment in a concurrent clinical interventional study

Sponsors & Collaborators

• Union Hospital, Tongji Medical College, Huazhong University of Science and Technology: lead
• Jiangsu Hengrui Pharmaceutical Co., Ltd.: collaborator

Study Sites (1)

Union Hospital, Tongji Medical College, Huazhong University of Science and Technology

Wuhan, Hubei, 430022, China

Location

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic; Anemia, Aplastic

Interventions

hetrombopag

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms; Anemia; Bone Marrow Failure Disorders; Bone Marrow Diseases

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 11, 2022
• First Posted: April 19, 2022
• Study Start: May 1, 2022
• Primary Completion: May 1, 2026
• Study Completion: May 1, 2026
• Last Updated: April 19, 2022

Record last verified: 2022-04

Locations

CN (1)