Immune ThrombocytopeniaPhase 2NCT06478537
TPO-RA Treatment on Immune Tolerance Induction of ITP Patients With Sustained Platelet Recovery After Treatment Termination
A Phase 2 study testing Hetrombopag Olamine and Aspirin in people with Immune Thrombocytopenia, run by Peking Union Medical College Hospital at 1 site in China.
Trial summary
The aim of this study was to observe whether maintaining a high level of platelet count after TPO-RA in patients with primary immune thrombocytopenia (ITP) can induce immune tolerance, develop immune balance in ITP patients, and enable patients to achieve a sustained response (SRoT) after TPO-RA discontinuation. Read more about this study
- Who is it for?
- Ages 18 to 50 years
- Conditions the study is for
- Immune Thrombocytopenia
- What is being tested?
- Hetrombopag Olamine and Aspirin
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 56 participants
- Where
- 1 siteChina
- Study dates
- 20 Jun 2024to 20 Jun 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 18 to 50 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Age ≥18 years old, regardless of gender;
- Patients with newly diagnosed or persistent primary ITP who have shown inadequate response or relapse following first-line corticosteroid treatment with or without IVIg;
- Complete response (PLT > 100 × 10^9/L) achieved after hetrombopag treatment at doses of 2.5mg-7.5mg per day;
- Volunteer to participate in clinical research and sign an informed consent form, willing to follow and capable of completing all trial procedures.
Who cannot join
- Age>50 years old;
- Those who are contraindicated to taking aspirin;
- Previous arterial or venous thrombosis history (including coronary atherosclerotic heart disease, ischemic stroke, deep vein thrombosis or pulmonary embolism, etc.) or clinical symptoms and medical history indicate thrombophilia;
- Risk factors of cardiovascular diseases such as hypertension, diabetes and hyperlipidemia;
- Heart disease occurring within the first 3 months of screening, including congestive heart failure classified as III/IV by the New York Heart Association (NHYA), arrhythmias or myocardial infarction requiring medication, or arrhythmias known to increase the risk of thrombotic events (such as atrial fibrillation), or prolonged QT interval (QTc) after subject correction (QTc>450 milliseconds, or QTc>480 milliseconds in subjects with bundle branch block)
- Patients currently undergoing anticoagulant therapy or antiplatelet therapy;
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- Female patients receiving estrogen replacement therapy or oral contraceptives;
- Patients with past or current malignant tumors;
- Secondary thrombocytopenia, such as myelodysplastic syndrome, immune disorders such as systemic lupus erythematosus, early aplastic anemia, atypical aplastic anemia, antiphospholipid syndrome, thrombotic thrombocytopenic purpura, and other causes of thrombocytopenia;
- The results of bone marrow biopsy during the screening period indicate that the bone marrow fibrosis MF is ≥ 2 (Thieleja 2005, the European expert consensus bone marrow fibrosis scoring standard), or that bone marrow biopsy suggests the presence of other primary diseases that can cause thrombocytopenia besides ITP;
- Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) are three times higher than the upper limit of normal values, total bilirubin is three times higher than the upper limit of normal values, and blood creatinine is 1.5 times higher than the upper limit of normal values;
- Have a history of liver cirrhosis or portal hypertension;
- Uncontrollable infections;
- Hepatitis B surface antigen positive or previous history of hepatitis B, and in the past 3 months, accompanied by HBV-DNA ≥ 2000IU/ML; those with positive hepatitis C antibody, HCV-RNA positive in the past 3 months;
- Individuals who test positive for antibodies against human immunodeficiency virus or specific antibodies against Treponema pallidum;
- Individuals who are known to be allergic to the drug itself or its excipients;
- Breastfeeding or pregnant women or female patients planning to conceive during the study period; 18）Other situations determined by the researcher as unsuitable for participation in this study
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Peking Union Medical College Hospital
Beijing, Beijing Municipality
Tienan Zhu, M.D., 01069155027, zhutn@pumch.cn
About this study
Official title: Thrombopoietin Receptor Agonist (TPO-RA) Treatment on Immune Tolerance Induction of ITP Patients With Sustained Platelet Recovery After Treatment Termination
The aim of this study was to observe whether maintaining a high level of platelet count after TPO-RA in patients with primary immune thrombocytopenia (ITP) can induce immune tolerance, develop immune balance in ITP patients, and enable patients to achieve a sustained response (SRoT) after TPO-RA discontinuation.
Read the detailed description
In this study, ITP patients who has reached complete response (PLT ≥100 x 10^9/L）after thrombopoietin receptor agonist (TPO-RA) therapy do not rush to reduce the drug dose, so that a higher level of platelet count can be maintained for a period of time.
The treatment goal is to maintain the patient's platelet count at 300-600 × 10^9/L, and adjust the dosage of hetrombopag (2.5mg/d~7.5mg/d) based on the patient's platelet count. After 24-week TPO-RA treatment, all patients with a platelet count of ≥ 50 × 10^9/L after two consecutive visits will enter an 8-week reduction period. All patients who successfully discontinued the drug and maintained their platelet count at ≥30×109/L entered the efficacy and safety follow-up period.
The aim is to investigate whether this strategy could lead to the development or achievement of immune tolerance, achieving sustained response off treatment (SROT) (PLT≥50×109/L, no other ITP-specific medications, no bleeding) after TPO-RA discontinuation.
- Conditions
- Immune Thrombocytopenia
- Being tested
- Hetrombopag Olamine, Aspirin
- Keywords
- immune thrombocytopenia, thrombopoietin receptor agonist (TPO-RA), hetrombopag
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Hetrombopag OlamineDrug
Also called Hetrombopag
Treatment period: 24-week Hetrombopag (2.5mg/d~7.5mg/d) treatment.
- Two consecutive visits with PLT>600 × 10^9/L: daily dose reduction of 2.5mg; If the lowest dose has been used, extend the dosing interval.
- Two consecutive visits with PLT<300 × 10^9/L: increase the daily dose by 2.5mg until the maximum dose is reached.
- Two consecutive visits with PLT<50 × 10^9/L: increase the daily dose by 2.5mg until the maximum dose is reached; If PLT is still<50 × 10^9/L with7.5mg/d × 28d, the patient will be withdrawn.
Drug discontinuation period：8-week Hetrombopag (2.5mg/d~7.5mg/d) reduction. Hetrombopag reduces by 2.5mg every 2 weeks, and after reducing to the minimum dose of 2.5mg/d x 2 weeks, it is changed to 2.5mg once every other day (Qod) treatment, with a maximum reduction time of 8 weeks. If the PLT during two consecutive visits is less than 30 × 10^9/L, the patient will be withdrawn.
AspirinDrug
Aspirin 100mg, qd
| Study group | Role | What they receive |
|---|---|---|
| hetrombopag treatment | Experimental | Not described Receives: Drug: Hetrombopag Olamine; Drug: Aspirin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Sustained response off treatment (SROT)
The percentage of participants with successful discontinuation of TPO-RAs after tapering and discontinuation
Measured: 3 year
Secondary outcomes (5)
Quality of life (QoL)
The degree to which an individual is healthy, comfortable, and able to participate in or enjoy life events according to the MOS 36-item short-form health survey (SF-36) at week1, week24 and week56 (±5 d).
Measured: 3 year
Duration of Complete response (DCR)
The time from the first assessment of complete resonse (platelet count ≥ 100 × 10^9/L, without bleeding) until the date of the first occurrence of progression, or until the date of death.t
Measured: 3 year
Duration of Response (DoR)
The time from the first assessment of response (platelet count ≥ 50 × 10^9/L) until the date of the first occurrence of progression, or until the date of death.
Measured: 3 year
Adverse events
The incidence and severity of thromboembolism, as well as other commonly used indicators such as abnormal clinical symptoms and vital signs, abnormalities observed in laboratory tests, are recorded.Toxicity will be graded according to NCI-CTC AE 5.0.
Measured: 3 years
Bleeding events
Bleeding events occurring in patients due to ITP
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jun 2024
- Study start20 Jun 2024 (estimated)
- Primary completion20 Jun 2027 (estimated)
- Study completion20 Jun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Immune ThrombocytopeniaStudies of the same condition, open and under way.
- More trials run by Peking Union Medical College HospitalThe sponsor's other registered studies.
- 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 56 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking Union Medical College Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Tienan Zhu, Peking Union Medical College Hospital
- Investigators
- Tienan Zhu, M.D., Principal investigator, Peking Union Medical College Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06478537
- Sponsor's protocol ID
- I-24PJ1123
- Status last verified
- Jun 2024
- First submitted
- 13 Jun 2024
- Last updated
- 27 Jun 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Thrombopoietin Receptor Agonist (TPO-RA) Treatment on Immune Tolerance Induction of ITP Patients With Sustained Platelet Recovery After Treatment Termination
- ClinicalTrials.gov ID
- NCT06478537
- Acronym / sponsor ID
- I-24PJ1123
- Conditions
- Immune Thrombocytopenia
- Keywords
- immune thrombocytopenia, thrombopoietin receptor agonist (TPO-RA), hetrombopag
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 56 participants (estimated)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Study start
- 20 Jun 2024 (estimated)
- Primary completion
- 20 Jun 2027 (estimated)
- Study completion
- 20 Jun 2027 (estimated)
- First posted
- 27 Jun 2024 (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.
The aim of this study was to observe whether maintaining a high level of platelet count after TPO-RA in patients with primary immune thrombocytopenia (ITP) can induce immune tolerance, develop immune balance in ITP patients, and enable patients to achieve a sustained response (SRoT) after TPO-RA discontinuation.
Detailed description
In this study, ITP patients who has reached complete response (PLT ≥100 x 10^9/L）after thrombopoietin receptor agonist (TPO-RA) therapy do not rush to reduce the drug dose, so that a higher level of platelet count can be maintained for a period of time.
The treatment goal is to maintain the patient's platelet count at 300-600 × 10^9/L, and adjust the dosage of hetrombopag (2.5mg/d~7.5mg/d) based on the patient's platelet count. After 24-week TPO-RA treatment, all patients with a platelet count of ≥ 50 × 10^9/L after two consecutive visits will enter an 8-week reduction period. All patients who successfully discontinued the drug and maintained their platelet count at ≥30×109/L entered the efficacy and safety follow-up period.
The aim is to investigate whether this strategy could lead to the development or achievement of immune tolerance, achieving sustained response off treatment (SROT) (PLT≥50×109/L, no other ITP-specific medications, no bleeding) after TPO-RA discontinuation.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| hetrombopag treatment | Experimental | Drug: Hetrombopag Olamine, Drug: Aspirin |
Who may qualify
- Ages
- 18 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (4 listed)
- Age ≥18 years old, regardless of gender;
- Patients with newly diagnosed or persistent primary ITP who have shown inadequate response or relapse following first-line corticosteroid treatment with or without IVIg;
- Complete response (PLT > 100 × 10^9/L) achieved after hetrombopag treatment at doses of 2.5mg-7.5mg per day;
- Volunteer to participate in clinical research and sign an informed consent form, willing to follow and capable of completing all trial procedures.
Key exclusion criteria (17 listed)
- Age>50 years old;
- Those who are contraindicated to taking aspirin;
- Previous arterial or venous thrombosis history (including coronary atherosclerotic heart disease, ischemic stroke, deep vein thrombosis or pulmonary embolism, etc.) or clinical symptoms and medical history indicate thrombophilia;
- Risk factors of cardiovascular diseases such as hypertension, diabetes and hyperlipidemia;
- Heart disease occurring within the first 3 months of screening, including congestive heart failure classified as III/IV by the New York Heart Association (NHYA), arrhythmias or myocardial infarction requiring medication, or arrhythmias known to increase the risk of thrombotic events (such as atrial fibrillation), or prolonged QT interval (QTc) after subject correction (QTc>450 milliseconds, or QTc>480 milliseconds in subjects with bundle branch block)
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 |
|---|---|---|
| Hetrombopag OlamineAlso: HetrombopagTreatment period: 24-week Hetrombopag (2.5mg/d~7.5mg/d) treatment. * Two consecutive visits with PLT>600 × 10^9/L: daily dose reduction of 2.5mg; If the lowest dose has been used, extend the dosing interval. * Two consecutive visits with PLT<300 × 10^9/L: increase the daily dose by 2.5mg until the maximum dose is reached. * Two consecutive visits with PLT<50 × 10^9/L: increase the daily dose by 2.5mg until the maximum dose is reached; If PLT is still<50 × 10^9/L with7.5mg/d × 28d, the patient will be withdrawn. Drug discontinuation period：8-week Hetrombopag (2.5mg/d~7.5mg/d) reduction. Hetrombopag reduces by 2.5mg every 2 weeks, and after reducing to the minimum dose of 2.5mg/d x 2 weeks, it is changed to 2.5mg once every other day (Qod) treatment, with a maximum reduction time of 8 weeks. If the PLT during two consecutive visits is less than 30 × 10^9/L, the patient will be withdrawn. | Drug | hetrombopag treatment |
| AspirinAspirin 100mg, qd | Drug | hetrombopag treatment |
Primary outcome
Sustained response off treatment (SROT)
Time frame: 3 year
The percentage of participants with successful discontinuation of TPO-RAs after tapering and discontinuation
5 secondary outcomes
Quality of life (QoL)
Time frame: 3 year
Duration of Complete response (DCR)
Time frame: 3 year
Duration of Response (DoR)
Time frame: 3 year
Adverse events
Time frame: 3 years
Bleeding events
Time frame: 3 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jun 2024.
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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.
Tienan Zhu, M.D. principal investigator
Peking Union Medical College Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Hetrombopag Olamine and Aspirin in people with Immune Thrombocytopenia, run by Peking Union Medical College Hospital 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 17 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: looking for participants now, as of the sponsor's last update on 27 Jun 2024. 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Tienan Zhu, M.D. (01069155027, zhutn@pumch.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Sustained response off treatment (SROT) (time frame: 3 year). 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 27 Jun 2024.