Clinical trial NCT04890041: TPO-RA in Primary Immune Thrombocytopenia (ITP) in Patients Older Than 14 Years
Official title: 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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Primary Immune Thrombocytopenic Purpura
Lead sponsor: Institute of Hematology & Blood Diseases Hospital, China
Sponsor class: OTHER
First posted: 2021-05-18
Enrollment: 100
Sex: ALL
Minimum age (years): 14
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This multi-center study aims to study the efficacy of TPO-RAs' transformation in Chinese ITP patients older than 14 years. This study will be conducted in ITP patients who had not responded to first-line in the previous treatment .

Detailed description: The primary objective of this study was to evaluate the efficacy of TPO-RAs' transformation in Chinese ITP patients older than 14 years who had not responded to first-line in the previous treatment.

100 eligible subjects will be enroll ed in this study.The dose will be adjusted according to the subject platelet count during the period from week 1 to week 6.

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.

Primary outcomes:
measure: Treatment response
timeFrame: From the start of study treatment (Day 1) up to the end of week 6
Secondary outcomes:
measure: Treatment response
timeFrame: From the start of study treatment (Day 1) up to the end of week 1,2, 3, 4, 5 and 6.
measure: Treatment response
timeFrame: From the start of study treatment (Day 1) up to the end of week 1,2, 3, 4, 5 and 6.
measure: Duration of response
timeFrame: From the start of study treatment (Day 1) up to the end of week 6
measure: Concomitant medication
timeFrame: From the start of study treatment (Day 1) up to the end of week 6
measure: Adverse events
timeFrame: From the start of study treatment (Day 1) up to the end of week 6
Other outcomes:
Locations:
city: Tianjin
country: China
facility: Institute of Hematology & Blood Diseases Hospital
state: Tianjin
Study officials:
affiliation: Institute of Hematology & Blood Diseases Hospital, China
name: Lei Zhang
role: PRINCIPAL_INVESTIGATOR
References: