Treatment for pediatric immune thrombocytopenia using Anti-CD38 antibody

A Prospective, One-arm and Open Clinical Study to Assess Safety and Efficacy of Anti-CD38 Antibody in the Treatment of Pediatric Primary Immune Thrombocytopenia

This study is testing a new antibody treatment for children with immune thrombocytopenia who haven't had success with other therapies to see if it can help improve their condition.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 60 (estimated)
• Ages: 6 Years to 17 Years
• Sex: All
• Sponsor: Institute of Hematology & Blood Diseases Hospital, China (Academic / other)
• Drugs / interventions: rituximab, daratumumab, prednisone
• Locations: 1 site (Tianjin, Tianjin Municipality)
• Trial ID: NCT06168851 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety and efficacy of an Anti-CD38 antibody for treating pediatric patients with primary immune thrombocytopenia (ITP) who have not responded adequately to first-line treatments and at least one second-line therapy. The study focuses on children aged 6 years and older with a platelet count below 30 X 10^9/L and a diagnosis of ITP for at least three months. Participants must have experienced treatment failure or relapse after prior therapies, including corticosteroids and other immunosuppressive treatments. The trial aims to address the urgent need for effective treatments in recurrent and refractory cases of ITP, which significantly impact patients' quality of life.

Who should consider this trial

Good fit: Ideal candidates for this study are pediatric patients aged 6 years and older diagnosed with primary immune thrombocytopenia who have not responded to previous treatments.

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

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for children with difficult-to-treat immune thrombocytopenia, potentially improving their platelet counts and overall quality of life.

How similar studies have performed: While there have been studies on treatments for immune thrombocytopenia, the use of Anti-CD38 antibodies in this specific pediatric population is a novel approach.

Eligibility criteria

Inclusion Criteria:

* Age 6 years and above, male or female
* Conform to the diagnostic criteria of immune Thrombocytopenia (ITP)
* Diagnosis of ITP ≥3 months, and with a platelet count of \<30 X 10\^9/L measured within 2 days prior to inclusion
* Failure to achieve response or relapse after corticosteroid therapy, and at least one second-line therapy including rituximab or TPORAs.
* 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 normal hepatic and renal functions
* 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.

March 26,2024 After approval by the Ethics Committee，age of subjects has been modified to 6 years and above upon enrollment. Approval Number: IIT2023072-EC-2.

Exclusion Criteria:

* Allergy to daratumumab or its excipients, or prior treatment with daratumumab that was refractory or achieved a response lasting \<6 months；
* Uncontrollable primary diseases of important organs, such as malignant tumors, liver failure, heart failure, renal failure and other diseases;
* HIV positive;
* Accompanied by uncontrollable active infection, including hepatitis B, hepatitis C, cytomegalovirus, EB virus and syphilis positive;
* Accompanied by extensive and severe bleeding, such as hemoptysis, upper gastrointestinal hemorrhage, intracranial hemorrhage, etc.;
* At present, there are heart diseases, arrhythmias that need treatment or hypertension that researchers judge is poorly controlled;
* Patients with thrombotic diseases such as pulmonary embolism, thrombosis and atherosclerosis;
* Those who have received allogeneic stem cell transplantation or organ transplantation in the past;
* Patients with mental disorders who cannot normally obtain informed consent and conduct trials and follow-up;
* Patients whose toxic symptoms caused by pre-trial treatment have not disappeared;
* Other serious diseases that may limit the subject's participation in this test (such as diabetes; Severe cardiac insufficiency; Myocardial obstruction or unstable arrhythmia or unstable angina pectoris in recent 6 months; Gastric ulcer, etc.);
* Patients with septicemia or other irregular severe bleeding;
* Patients taking antiplatelet drugs at the same time;
* Pregnant women, suspected pregnancies (positive pregnancy test for human chorionic gonadotropin in urine at screening) and lactating patients.

Where this trial is running

Tianjin, Tianjin Municipality

• Chinese Academy of Medical Science and Blood Disease Hospital — Tianjin, Tianjin Municipality, 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