Recruiting
Phase 2
Anti-CD38 Antibody Treating Pediatric Primary Immune Thrombocytopenia (ITP)
Condition(s) studied
Immune ThrombocytopeniaTreatment
Investigational drug(s) / intervention(s)
Anti-CD38 antibody Injection
Anti-CD38 antibody Injection: intravenous Anti-CD38 antibody administration
This study adopts a prospective, single arm, open design method. Twenty subjects were enrolled in the study and were treated with Anti-CD38 antibody (16mg/kg/w) for 8 weeks.
The first stage is the main research stage (d1-w8), which is the core treatment period. The subjects will receive intravenous infusion of 16mg/kg Anti-CD38 antibody once a week for 8 weeks to observe the safety and efficacy during treatment.
The second stage (w9-w24) is the stage of withdrawal from the visit, mainly to observe the safety and continuous efficacy of Anti-CD38 antibody after treatment.
The third stage (w 25 to y 5): Extension visit phase, primarily to observe the long-term safety and sustained efficacy following CD38 monoclonal antibody therapy.
Study summary
To evaluate the safety and efficacy of Anti-CD38 Antibody in the treatment of pediatric primary immune thrombocytopenia in patients who have not responded adequately or relapsed after first-line treatment and at least one second-line therapy including Anti-CD20 Antibody and/or TPO-RA, or those in whom no other second-line treatment options are suitable.
Eligibility
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.
Primary outcome measure(s)
- Evaluation of overall efficacy response after Anti-CD38 antibody treatment within 8 weeks — 8 weeks
Proportion of subjects with a platelet count ≥ 50 × 10\^9/L within 8 weeks after initial administration in absence of rescue therapy, and without having had dose increment of TPO-RA or corticosteroids during the study period
- Safety of Anti-CD38 antibody treatment — 5 years
Incidence, severity, and relationship of treatment emergent adverse events after Anti-CD38 antibody treatment
Trial sites (1)
| Facility | City | Region | Status |
|---|
| Chinese Academy of Medical Science and Blood Disease Hospital |
Tianjin |
Tianjin Municipality |
Recruiting |
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