A Prospective, One-arm and Open Clinical Study of Obinutuzumab in the Treatment of Pediatric Primary Immune Thrombocytopenia (ITP)

NCT06094881 | Recruiting Phase 2 DMC

• 1 other identifier: IIT2023047
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 1

Brief Summary

To evaluate the safety and efficacy of Obinutuzumab in the treatment of pediatric primary immune thrombocytopenia in patients who have not responded adequately or relapsed after first-line glucocorticoid treatment.

Conditions

Immune Thrombocytopenia; Treatment

Outcome Measures

Primary Outcomes (1)

• Evaluation of overall efficacy response after Obinutuzumab treatment within 12 weeks

  Overall response rate defined as proportion of subjects with a platelet count ≥ 30 × 10\^9/L and at least 2-fold from baseline at the meanwhile within 12 weeks after initial administration in absence of rescue therapy, and without having had dose increment of TPO-RA or corticosteroids during the study period

  12 weeks

Secondary Outcomes (17)

• Evaluation of overall efficacy response after Obinutuzumab treatment within 8 weeks

  8 weeks

• Evaluation of overall efficacy response after Obinutuzumab treatment within 6 months

  6 months

• Evaluation of overall efficacy response after Obinutuzumab treatment within 12 months

  12 months

• Evaluation of sustained response rate after Obinutuzumab treatment within 6 months

  6 months

• Evaluation of sustained response rate after Obinutuzumab treatment within 12 months

  12 months

Other Outcomes (1)

• Prognostic model establishment using multi-omics data such as transcriptome, proteome, metabolome, microbiome, et al.

  12 months

Study Arms (1)

Intervention (Obinutuzumab)

EXPERIMENTAL

60 enrolled subjects: one infusion

Drug: Obinutuzumab Injection [Gazyva]

Interventions

Obinutuzumab Injection [Gazyva] DRUG

intravenous Obinutuzumab administration This study adopts a prospective, single arm, open design method. 60 subjects were enrolled in the study and were treated with CD20 monoclonal antibody (Obinutuzumab: 1000mg) for once. The first stage is the main research stage (d1-w12), which is the core treatment period. The subjects will receive intravenous infusion of 1000mg Obinutuzumab for once to observe the safety and efficacy during treatment. The second stage (w12-w48) is the stage of withdrawal from the visit, mainly to observe the safety and continuous efficacy of Obinutuzumab after treatment.

Intervention (Obinutuzumab)

Eligibility Criteria

• Age: 12 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead

Study Sites (1)

Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College

Tianjin, China

RECRUITING

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

obinutuzumab

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

Study Officials

• Lei Zhang, MD

  Chinese Academy of Medical Science and Blood Disease Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Ting Sun, MD

CONTACT

+8615822339131; sunting@ihcams.ac.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 13, 2023
• First Posted: October 23, 2023
• Study Start: December 1, 2023
• Primary Completion: November 1, 2025
• Study Completion (Estimated): November 1, 2026
• Last Updated: February 24, 2025

Record last verified: 2024-12

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: 12 months to 36 months after study completion
• Access Criteria: Upon request to PI

Locations

CN (1)