Actively Recruiting
A Prospective, One-arm and Open Clinical Study to Assess Safety and Efficacy of Anti-CD38 Antibody in the Treatment of Children With Primary Immune Thrombocytopenia
Led by Institute of Hematology & Blood Diseases Hospital, China · Updated on 2025-08-05
30
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
This trial studies children aged 6 to 18 with primary immune thrombocytopenia (ITP) who did not respond well to first-line treatments like steroids. ITP is an autoimmune disease causing low platelet counts and bleeding, with limited effective treatments especially for recurrent or resistant cases. The study focuses on evaluating the safety and effectiveness of an anti-CD38 antibody called daratumumab, which targets plasma cells believed to contribute to treatment resistance in ITP. Participants receive intravenous daratumumab at a dose of 16 mg/kg once a week for 4 weeks during the core treatment phase. After this, there is a follow-up period lasting until week 24 to monitor ongoing safety and treatment effects. This is a single-arm, open-label study enrolling 30 children who will be observed for responses and side effects after the antibody treatment. During the study, researchers will measure platelet counts and response rates, track bleeding events using pediatric and WHO bleeding scales, and monitor adverse effects up to 24 weeks. They will also evaluate reduction in other medications and time to response. Safety assessments and ongoing efficacy evaluations will occur throughout the follow-up period, with the primary outcome measured at 8 weeks.
CONDITIONS
Brief Title
Anti-CD38 Antibody Treating Children With Primary Immune Thrombocytopenia (ITP)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 6 to 18 years, male or female
- Diagnosed with primary immune thrombocytopenia for at least 3 months
- Failed glucocorticoid therapy or could not maintain response or relapsed
- History of response to first-line therapy (glucocorticoids and/or intravenous immunoglobulin) with platelet count 6 x 10^9/L
- Platelet count below 30 x 10^9/L on average during screening with at least two tests one week apart
- ECOG performance status score of 0, 1, or 2
- Allowed stable maintenance therapy with one concomitant medication like low-dose glucocorticoids or TPO receptor agonists for at least 4 weeks
- Negative pregnancy test for fertile females
- Use of effective contraception during the study and for 4 to 6 months after treatment
- Able to understand and follow study protocol and provide informed consent
You will not qualify if you...
- Allergy to anti-CD38 antibody or excipients, or prior failure or short response to anti-CD38 antibody
- Secondary thrombocytopenia or autoimmune hemolytic anemia
- History of thrombosis, embolism, or severe bleeding within 12 months before treatment
- Participation in other investigational drug studies within 4 weeks or 5 half-lives prior to treatment
- Use of antiplatelet drugs within 3 weeks prior to treatment
- Emergency ITP treatment within 2 weeks prior to treatment
- Use of certain immunosuppressive drugs within 4 to 6 months prior to treatment
- Splenectomy within 6 months prior to treatment
- Receipt or planned receipt of live vaccines during study
- Diagnosis of myelodysplastic syndrome or recent malignancy within 5 years
- Allogeneic stem cell or organ transplantation
- Clinically significant medical history posing safety risks including heart, lung, immune, mental, or metabolic diseases
- Recent severe or recurrent infections within 4 weeks prior to treatment
- Known or suspected immunosuppression or opportunistic infections
- Significant laboratory abnormalities including elevated liver enzymes, low blood counts, or impaired kidney function
- Positive tests for HIV, syphilis, hepatitis B or C without controlled viral loads
- Pregnant or lactating women or those planning pregnancy or breastfeeding
- Any other conditions judged unsuitable by the investigator
AI-Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
2 visits one week apart for platelet count testing
Duration - 4 weeks
Participants receive intravenous Daratumumab once a week for 4 weeks to assess safety and efficacy of the treatment.
4 weekly visits (in-person) for intravenous infusion
Duration - 20 weeks
Participants are observed for safety and continuous efficacy after completing Daratumumab treatment.
Visits from Week 5 to Week 24 for safety and efficacy monitoring
Trial Site Locations
Total: 1 location
1
Chinese Academy of Medical Science and Blood Disease Hospital
Tianjin, Tianjin Municipality, China, 300020
Actively Recruiting
Research Team
T
Ting Sun, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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