Evaluating Bortezomib for Immune Thrombocytopenia

A Phase 2 Study to Investigate Efficacy and Safety Evaluation of Bortezomib in Patients With Relapsed/Refractory Immune Thrombocytopenia

This study is testing if a medication called bortezomib can help people with immune thrombocytopenia who haven't responded to other treatments.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 29 (estimated)
• Ages: 19 Years and up
• Sex: All
• Sponsor: Seoul National University Hospital (Academic / other)
• Drugs / interventions: Rituximab, Alemtuzumab, chemotherapy, cyclophosphamide
• Locations: 4 sites (Guri-si and 3 other locations)
• Trial ID: NCT05599880 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the efficacy and safety of bortezomib in patients suffering from relapsed or refractory immune thrombocytopenia. Participants will receive the treatment on an outpatient basis over three cycles, with follow-up assessments conducted for 12 months. During each visit, vital signs, medical history, physical examinations, and blood tests will be performed to monitor the patient's response and any adverse reactions. Bone marrow tests may also be conducted at the investigator's discretion.

Who should consider this trial

Good fit: Ideal candidates include adults aged 19 and older diagnosed with immune thrombocytopenia who have specific criteria indicating the need for treatment.

Not a fit: Patients with immune thrombocytopenia due to decreased platelet production in the bone marrow may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with difficult-to-treat immune thrombocytopenia.

How similar studies have performed: While this approach is being evaluated in this trial, previous studies have shown varying results with similar treatments for immune thrombocytopenia.

Eligibility criteria

Inclusion Criteria:

1. Men and women 19 years of age and older
2. Patients diagnosed with immune thrombocytopenia (primary or secondary)
3. (Patients with at least one of B, C, or D meeting A below.) A.A person who has been confirmed not to be due to a cause of decreased platelet production in the bone marrow in the results of bone marrow examination within the past 5 years. (Bone marrow examination is required if there is no bone marrow examination result within the last 5 years.) B.After being diagnosed with ITP, there has been a case of complete platelet response with plt \>100k in immunoglobulin therapy.

   C.Patients suspected of having primary immune thrombocytopenia with proven anti-platelet antibody., D.Helicobacter IgG, antinuclear antibody (ANA), anticardiolipin antibody (ACA), nonspecific inhibitor (NSI), etc. have been proven, and secondary immune thrombocytopenia caused by autoimmune disease is suspected.
4. platelets \<30 x 109/L
5. Patients in need of clinical treatment.
6. Patients who have relapsed or refractory after receiving treatment with corticosteroids (prednisolone cumulative dose 5.6mg/kg (0.8mg/kg 1 week) or more or dexamethasone cumulative dose 80mg (20mg 4 days) or more)., However, there are exceptions when corticosteroids are contraindicated.
7. Patients who have been relapsed or refractory after receiving at least 2 treatments with a mechanism other than corticosteroids.
8. Patients who underwent two different treatments from among the treatment methods below.

   * Splenectomy
   * eltrombopag or romiplostim
   * Rituximab treatment
   * Azathioprine
   * cyclosporine or mycophenolate mofetil(MMF)
   * vincristine or cyclophosphamide
   * danazol
   * dapsone
   * Alemtuzumab
9. Female patients of childbearing potential should have a negative urine or serum pregnancy test within 28 days before starting administration of the test drug.
10. Patients who voluntarily or legal guardians have given written consent to participate in this clinical trial.

Exclusion Criteria:

1. pregnant, lactating women
2. Patients allergic to bortezomib.
3. Patients with Grade 2 or higher peripheral neuropathy requiring drug treatment.
4. Patients with severe or uncontrolled active infection.
5. Patients with significant immunoglobulin degradation in the immunoglobulin profile.
6. HBV, HCV carrier or HIV patient. (However, if the patient is being stably maintained with antiviral treatment, and the expert opinion determines that it is possible to participate in the clinical trial along with antiviral treatment and prevention, participation is possible.)
7. Patients currently undergoing chemotherapy.
8. Patients whose bone marrow did not recover after cancer treatment, or whose cause other than immune destruction was confirmed by bone marrow examination, such as thrombocytopenia due to intramedullary abnormalities. However, if the presence of anti-platelet antibodies is proven and thrombocytopenia due to immune destruction is strongly suspected clinically, registration is possible even if there are other causes of thrombocytopenia.)
9. Patients who may become pregnant. However, women of childbearing age(If postmenopausal women are not of childbearing age, they must have been menstruating for at least 12 months.) who have not undergone sterilization can participate in the clinical trial only if the pregnancy test is negative. And, effective contraception must be maintained throughout the entire trial period.
10. Patients currently participating in other clinical trials.
11. Patients who are not considered suitable for participation in clinical trials in the opinion of the researcher.
12. Patients with inadequate organ function.

    * total bilirubin \> 3 x ULN
    * creatinine \> 1.5 x ULN
    * liver function test AST(SGOT) \& ALT(SGPT) \> 3 x ULN

Where this trial is running

Guri-si and 3 other locations

• Seoul National University Bundang Hospital — Guri-si, South Korea (Recruiting)
• Seoul National University Hospital — Seoul, South Korea (Recruiting)
• SMG-SNU Boramae Medical Center — Seoul, South Korea (Recruiting)
• Seoul National University Hospital — Seoul, South Korea (Not_yet_recruiting)

Study contacts

• Study coordinator: Dong-Yeop Shin, MD, PhD

Conditions Relapsed/Refractory Immune Thrombocytopenia