TPO-RA Treatment on Immune Tolerance Induction of ITP Patients With Sustained Platelet Recovery After Treatment Termination

NCT06478537 | Recruiting Phase 2

• 1 other identifier: I-24PJ1123
• Study Type: interventional
• Target: 56
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Immune Thrombocytopenia

Keywords

immune thrombocytopenia; thrombopoietin receptor agonist (TPO-RA); hetrombopag

Outcome Measures

Primary Outcomes (1)

• Sustained response off treatment (SROT)

  The percentage of participants with successful discontinuation of TPO-RAs after tapering and discontinuation

  3 year

Secondary Outcomes (5)

• Quality of life (QoL)

  3 year

• Duration of Complete response (DCR)

  3 year

• Duration of Response (DoR)

  3 year

• Adverse events

  3 years

• Bleeding events

  3 years

Study Arms (1)

hetrombopag treatment

EXPERIMENTAL

Drug: Hetrombopag Olamine; Drug: Aspirin

Interventions

Hetrombopag Olamine DRUG

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50 × 10\^9/L with7.5mg/d × 28d, the patient will be withdrawn.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 If the PLT during two consecutive visits is less than 30 × 10\^9/L, the patient will be withdrawn.

Also known as: Hetrombopag

hetrombopag treatment

Aspirin DRUG

Aspirin 100mg, qd

hetrombopag treatment

Eligibility Criteria

• Age: 18 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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;
• 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;

Sponsors & Collaborators

• Peking Union Medical College Hospital: lead

Study Sites (1)

Peking Union Medical College Hospital

Beijing, Beijing Municipality, China

RECRUITING

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

hetrombopag; Aspirin

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Intervention Hierarchy (Ancestors)

Salicylates; Hydroxybenzoates; Phenols; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Organic Chemicals

Study Officials

• Tienan Zhu, M.D.

  Peking Union Medical College Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Tienan Zhu, M.D.

CONTACT

01069155027; zhutn@pumch.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director

Study Record Dates

• First Submitted: June 13, 2024
• First Posted: June 27, 2024
• Study Start: June 20, 2024
• Primary Completion (Estimated): June 20, 2027
• Study Completion (Estimated): June 20, 2027
• Last Updated: June 27, 2024

Record last verified: 2024-06

Locations

CN (1)