Treatment to induce immune tolerance in ITP patients after stopping TPO-RA

Thrombopoietin Receptor Agonist (TPO-RA) Treatment on Immune Tolerance Induction of ITP Patients With Sustained Platelet Recovery After Treatment Termination

This study is testing if keeping platelet counts high in ITP patients after stopping a specific treatment can help them maintain those levels without needing more treatment.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 56 (estimated)
• Ages: 18 Years to 50 Years
• Sex: All
• Sponsor: Peking Union Medical College Hospital (Academic / other)
• Locations: 1 site (Beijing, Beijing Municipality)
• Trial ID: NCT06478537 on ClinicalTrials.gov

What this trial studies

This study investigates whether maintaining a high platelet count in patients with primary immune thrombocytopenia (ITP) after treatment with thrombopoietin receptor agonists (TPO-RA) can lead to immune tolerance. Patients who achieve a complete response with a platelet count of at least 100 x 10^9/L will continue treatment to maintain platelet levels between 300-600 x 10^9/L. After 24 weeks, those with stable platelet counts will enter a reduction phase, aiming to sustain platelet levels without ongoing treatment. The goal is to determine if this approach can achieve a sustained response off treatment.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 and older with newly diagnosed or persistent primary ITP who have not responded adequately to first-line treatments.

Not a fit: Patients over 50 years old or those with a history of thrombosis or cardiovascular risk factors may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could enable ITP patients to maintain healthy platelet levels without the need for ongoing medication.

How similar studies have performed: While similar approaches have been explored, this specific strategy of maintaining high platelet counts post-TPO-RA is novel and has not been extensively tested.

Eligibility criteria

Inclusion Criteria:

1. Age ≥18 years old, regardless of gender;
2. Patients with newly diagnosed or persistent primary ITP who have shown inadequate response or relapse following first-line corticosteroid treatment with or without IVIg;
3. Complete response (PLT \> 100 × 10\^9/L) achieved after hetrombopag treatment at doses of 2.5mg-7.5mg per day;
4. Volunteer to participate in clinical research and sign an informed consent form, willing to follow and capable of completing all trial procedures.

Exclusion Criteria:

1. Age\>50 years old;
2. Those who are contraindicated to taking aspirin;
3. 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;
4. Risk factors of cardiovascular diseases such as hypertension, diabetes and hyperlipidemia;
5. 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)
6. Patients currently undergoing anticoagulant therapy or antiplatelet therapy;
7. Female patients receiving estrogen replacement therapy or oral contraceptives;
8. Patients with past or current malignant tumors;
9. 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;
10. 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;
11. 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;
12. Have a history of liver cirrhosis or portal hypertension;
13. Uncontrollable infections;
14. 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;
15. Individuals who test positive for antibodies against human immunodeficiency virus or specific antibodies against Treponema pallidum;
16. Individuals who are known to be allergic to the drug itself or its excipients;
17. 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

Where this trial is running

Beijing, Beijing Municipality

• Peking Union Medical College Hospital — Beijing, Beijing Municipality, China (Recruiting)

Study contacts

• Principal investigator: Tienan Zhu, M.D. — Peking Union Medical College Hospital
• Study coordinator: Tienan Zhu, M.D.

Conditions Immune Thrombocytopenia; immune thrombocytopenia; thrombopoietin receptor agonist; hetrombopag