Using Obinutuzumab to treat pediatric immune thrombocytopenia

A Prospective, One-arm and Open Clinical Study to Assess Safety and Efficacy of Anti-Human CD20 Monoclonal Antibody Obinutuzumab in the Treatment of Pediatric Primary Immune Thrombocytopenia

This study is testing if Obinutuzumab can help teenagers with immune thrombocytopenia who haven't responded well to other treatments.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 60 (estimated)
• Ages: 12 Years to 18 Years
• Sex: All
• Sponsor: Institute of Hematology & Blood Diseases Hospital, China (Academic / other)
• Drugs / interventions: rituximab, prednisone, Obinutuzumab
• Locations: 1 site (Tianjin)
• Trial ID: NCT06094881 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety and efficacy of Obinutuzumab, a monoclonal antibody, in treating pediatric patients aged 12-18 with primary immune thrombocytopenia (ITP) who have not adequately responded to or have relapsed after first-line glucocorticoid treatment. The study aims to address the challenges of ITP, which is characterized by low platelet counts and increased bleeding risk. Participants will receive Obinutuzumab injections, and their responses will be monitored to determine the treatment's effectiveness and safety profile.

Who should consider this trial

Good fit: Ideal candidates are pediatric patients aged 12-18 with persistent or chronic immune thrombocytopenia and a platelet count of less than 30 X 10^9/L who have failed previous hormonal therapies.

Not a fit: Patients who have not been diagnosed with immune thrombocytopenia or those who have not failed previous treatments may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a more effective option for managing pediatric ITP, potentially improving patient outcomes and quality of life.

How similar studies have performed: While the use of Obinutuzumab in ITP is a novel approach, similar monoclonal antibody therapies have shown promise in treating other autoimmune conditions.

Eligibility criteria

Inclusion Criteria:

* Age 12-18 years old, male or female
* Conform to the diagnostic criteria of persistent or chronic immune Thrombocytopenia (ITP)
* With a platelet count of \<30 X 10\^9/L measured within 2 days prior to administration（Platelet counts were measured at least 2 times during screening (at least 1 week apart) with platelets\<30 X 10\^9/L）
* Failure or recurrence of previous hormonal therapy or hormone dependence
* The previous emergency treatment of ITP (e.g. methylprednisolone, platelet transfusion, IVIG transfusion) must be completed at least 2 weeks before the first administration
* Signed and dated written informed consent
* With Liver and kidney function\<1.5×upper limit of normal， such as ALT、AST，BUN，Cre，etc.
* ECOG physical state score ≤ 2 points
* Cardiac function of the New York Society of Cardiac Function ≤ 2
* Patients receiving maintenance treatment (including corticosteroids (less than or equal to 0.5mg/kg prednisone), TPO receptor agonists, etc.) must have a stable dose at least 4 weeks before the first administration, and azathioprine, danazol, cyclosporin A, tacrolimus, sirolimus, etc. must be stopped at least 4 weeks before the first administration; The end of rituximab treatment was\>3 months；More than 6 months after splenectomy.

Exclusion Criteria:

* Subjects with primary disease of important organs (liver, kidney, heart, etc.), or with immune system diseases;
* Secondary thrombocytopenia caused by various reasons, such as connective tissue disorders, bone marrow hematopoietic failure disease, myelodysplastic syndrome, malignancy, drugs, inherited thrombocytopenia, common variable immune deficiency, lymphoma, etc.;
* Subjects infected with human immunodeficiency virus (HIV);
* Uncontrollable or active infections during the screening period, including hepatitis B, hepatitis C, cytomegalovirus, EB virus, or positive syphilis antigen;
* Subjects with extensive and severe bleeding, such as hemoptysis, upper gastrointestinal hemorrhage, intracranial hemorrhage;
* Subjects with heart disease that requires treatment or hypertension that has been judged by researchers to be poorly controlled currently;
* Subjects with any venous or arterial thrombosis, atherosclerosis, and other diseases;
* Subjects with a history of malignant solid tumor or have received allogeneic stem cell transplantation or organ transplantation;
* Subjects with mental disorders who are unable to sign normal informed consent and conduct trials and follow-up;
* Subjects whose toxic symptoms caused by pre-trial treatment have not disappeared;
* Subjects with other serious diseases that may limit their participation in this trial (diabetes; severe cardiac insufficiency; myocardial obstruction or unstable arrhythmia or unstable angina pectoris in the last 6 months; gastric ulcer; active autoimmune disease, etc.);
* Subjects with septicemia or other irregular bleeding;
* Patients taking antiplatelet drugs at the same time;
* Any medical history or condition that the investigator deems unsuitable for participation in the study.

Where this trial is running

Tianjin

• Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College — Tianjin, China (Recruiting)

Study contacts

• Principal investigator: Lei Zhang, MD — Chinese Academy of Medical Science and Blood Disease Hospital
• Study coordinator: Ting Sun, MD

Conditions Immune Thrombocytopenia; Treatment