Evaluating TPO-RAs for treating immune thrombocytopenia in patients over 14

Efficacy of TPO-RA in the Management of Primary Immune Thrombocytopenia (ITP) in Patients Older Than 14 Years With Poor First-line Response: a Multi-center, Prospective, One-arm Study

This study is testing if a new type of medication can help people over 14 with immune thrombocytopenia who haven't improved with other treatments by looking at how it affects their platelet counts and bleeding.

Quick facts

• Phase: Not applicable
• Study type: Interventional
• Enrollment: 100 (estimated)
• Ages: 14 Years and up
• Sex: All
• Sponsor: Institute of Hematology & Blood Diseases Hospital, China (Academic / other)
• Drugs / interventions: rituximab
• Locations: 1 site (Tianjin, Tianjin Municipality)
• Trial ID: NCT04890041 on ClinicalTrials.gov

What this trial studies

This multi-center study investigates the efficacy of TPO receptor agonists (TPO-RAs) in Chinese patients aged 14 and older diagnosed with primary immune thrombocytopenia (ITP) who have not responded to first-line treatments. The study aims to enroll 100 eligible subjects, adjusting the dosage of TPO-RAs based on platelet counts measured from week 1 to week 6. The primary goal is to assess how well these medications can improve platelet counts and reduce bleeding episodes in this patient population.

Who should consider this trial

Good fit: Ideal candidates are individuals aged 14 and older with primary immune thrombocytopenia who have not responded to previous first-line therapies.

Not a fit: Patients with secondary immune thrombocytopenia or those with a history of severe thrombotic events may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a new effective treatment option for patients with resistant primary immune thrombocytopenia.

How similar studies have performed: While there have been studies on TPO-RAs, this specific approach in the Chinese population with treatment-resistant ITP is relatively novel.

Eligibility criteria

Inclusion Criteria:

* Men and women greater than or equal to 14 years of age.
* Participants diagnosed with primary immune thrombocytopenia with two platelet counts of \< 30x10\^9/L or with bleeding at least 7 days apart，do not have evidence of other causes of thrombocytopenia (e.g.,pseudothrombocytopenia, myeloid fibrosis).
* Previous treatment with poor response to first-line therapy and any of the maximum 4-week doses of eltrombopag, herombopag, avatrombopag, or 300U/kg/ day × 14-day rhTPO with no response to treatment (platelet count \< 30×109/L after treatment, or platelet count increase less than twice the baseline value, or with bleeding)
* Participants willing and able to comply with the requirements of the study protocol, and sign the informed consent.

Exclusion Criteria:

* Patients diagnosed with secondary immune thrombocytopenia.
* A history of arteriovenous thrombosis, disseminated intravascular coagulation, myocardial infarction, cerebral obstruction, thrombotic microangiopaemia, autoimmune diseases, malignant tumors, liver cirrhosis and other diseases that were not eligible for inclusion.
* Liver disease with one of the following indicators: a. total bilirubin ≥ 2 times of the upper limit of normal; b. alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≥ 2 times the upper limit of normal value; Patients with renal disease (serum creatinine ≥ 1.5 times the upper limit of normal);
* Subjects with known allergies to eltrombopag, herombopag, rh-TPO, avatrombopag, or any of excipients;
* Have used rituximab in the past 3 months;
* Splenectomy in recent 3 months;
* Those who are not considered suitable for this study by the researcher;
* Women who are pregnant or who intend to become pregnant in the near future are excluded.

Where this trial is running

Tianjin, Tianjin Municipality

• Institute of Hematology & Blood Diseases Hospital — Tianjin, Tianjin Municipality, China (Recruiting)

Study contacts

• Principal investigator: Lei Zhang — Institute of Hematology & Blood Diseases Hospital, China
• Study coordinator: Wei Liu

Conditions Primary Immune Thrombocytopenic Purpura