Primary Immune ThrombocytopeniaNCT05333861
An Observational, Multicenter Study to Evaluate the Safety and Effectiveness of Hetrombopag in Patients With ITP or AA
An observational study of Primary Immune Thrombocytopenia and Aplastic Anemia, run by Union Hospital, Tongji Medical College, Huazhong University of Science and Technology at 1 site in China.
Trial 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… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Primary Immune Thrombocytopenia, Aplastic Anemia
- What is being tested?
- hetrombopag
- How many people
- 1,150 participants
- Where
- 1 siteChina
- Study dates
- 1 May 2022to 1 May 2026
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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
Who may be able to join
- 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
Who cannot join
- Enrollment in a concurrent clinical interventional study
Trial locations and contacts
1 site in 1 country.
China1 site
Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
Wuhan, Hubei, 430022
About this study
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
Read the 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.
- Conditions
- Primary Immune Thrombocytopenia, Aplastic Anemia
- Being tested
- hetrombopag
- Keywords
- Hetrombopag
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic, Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
hetrombopagDrug
According to prescription
| Study group | Role | What they receive |
|---|---|---|
| Full-analysis set (FAS) | The FAS includes all enrolled patients. The FAS will be used for all analyses. Receives: Drug: hetrombopag |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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
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.
Measured: 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
Hematologic assessments will be based on local laboratory results
Measured: 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.
Information will be collected via the patient's medical records.
Measured: 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)
SF-36 is a set of generic, coherent, and easily administered quality-of-life measures. These measures rely upon patient self-reporting.
Measured: 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
Information will be collected via the patient's medical records.
Measured: 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
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.
Measured: Data will be collected for all routine visits completed during the study period which is no more than 24 months.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Apr 2022
- Study start1 May 2022 (estimated)
- Primary completion1 May 2026 (estimated)
- Study completion1 May 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Immune ThrombocytopeniaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 1,150 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
- Sponsor type
- University, hospital or other
- Collaborators
- Jiangsu Hengrui Pharmaceutical Co., Ltd.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05333861
- Sponsor's protocol ID
- Hetrombopag-ITP/AA
- Status last verified
- Apr 2022
- First submitted
- 11 Apr 2022
- Last updated
- 19 Apr 2022
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Apr 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Observational, Multicenter Study to Evaluate the Safety and Effectiveness of Hetrombopag in Patients With ITP or AA
- ClinicalTrials.gov ID
- NCT05333861
- Acronym / sponsor ID
- Hetrombopag-ITP/AA
- Conditions
- Primary Immune Thrombocytopenia, Aplastic Anemia
- Keywords
- Hetrombopag
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 1,150 participants (estimated)
- Lead sponsor
- Union Hospital, Tongji Medical College, Huazhong University of Science and TechnologyUniversity, hospital or other
- Collaborators
- Jiangsu Hengrui Pharmaceutical Co., Ltd.
- Study start
- 1 May 2022 (estimated)
- Primary completion
- 1 May 2026 (estimated)
- Study completion
- 1 May 2026 (estimated)
- First posted
- 19 Apr 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Full-analysis set (FAS) | – | Drug: hetrombopagThe FAS includes all enrolled patients. The FAS will be used for all analyses. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (1 listed)
- Enrollment in a concurrent clinical interventional study
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| hetrombopagAccording to prescription | Drug | Full-analysis set (FAS) |
Primary outcome
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
6 secondary outcomes
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.
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.
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.
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.
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.
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.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Apr 2022.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Primary Immune Thrombocytopenia and Aplastic Anemia, run by Union Hospital, Tongji Medical College, Huazhong University of Science and Technology at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 19 Apr 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse events (time frame: Data will be collected for all routine visits completed during the study period no more than 24 months.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Apr 2022.