Actively Recruiting
A Prospective, Open-Label Study to Assess Safety and Efficacy of Anti-CD38 Antibody in Treating Pediatric Primary Immune Thrombocytopenia
Led by Institute of Hematology & Blood Diseases Hospital, China · Updated on 2025-11-28
60
Participants Needed
1
Research Sites
260 weeks
Total Duration
On this page
Sponsors
I
Institute of Hematology & Blood Diseases Hospital, China
Lead Sponsor
B
Beijing Children's Hospital
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effectiveness of an Anti-CD38 antibody treatment for children aged 6 to 17 years with primary immune thrombocytopenia (ITP) who have not responded well or have relapsed after initial and at least one second-line therapy, including Anti-CD20 antibody or TPO receptor agonists. ITP is an autoimmune disease causing low platelet counts and bleeding, with current treatments often insufficient for some patients, especially those with recurrent or resistant forms. This study aims to explore a new approach targeting long-lived plasma cells that may contribute to treatment resistance. Participants will receive intravenous Anti-CD38 antibody at a dose of 16 mg/kg once weekly for 8 weeks during the main treatment phase. After this, there is a follow-up period from weeks 9 to 24 to monitor safety and ongoing effects, followed by an extension phase lasting up to 5 years to observe long-term safety and sustained efficacy. The study is designed as a single-arm, open-label trial involving 60 children. During the study, participants will have their platelet counts and immune responses regularly evaluated, along with monitoring for any bleeding events using standard scales. Researchers will measure various immune cell subsets and antibody levels over the course of up to 5 years. Safety assessments will continue throughout the entire study duration, which includes treatment, follow-up, and long-term observation to understand the antibody's impact on pediatric ITP.
CONDITIONS
Brief Title
Anti-CD38 Antibody Treating Pediatric Primary Immune Thrombocytopenia (ITP)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 6 years and above, male or female
- Diagnosed with immune thrombocytopenia (ITP) for at least 3 months
- Platelet count less than 30 x 10^9/L measured within 2 days before inclusion
- Did not respond or relapsed after corticosteroid therapy and at least one second-line treatment including rituximab or TPO receptor agonists
- Completed any emergency ITP treatments at least 2 weeks before starting study drug
- Signed informed consent
- Normal liver and kidney function
- ECOG physical state score of 2 or less
- New York Heart Association cardiac function class 2 or less
- Stable maintenance treatment doses for at least 4 weeks before study start; certain immunosuppressants stopped at least 4 weeks before study start
- More than 3 months since rituximab treatment
- More than 6 months since splenectomy
You will not qualify if you...
- Allergy to daratumumab or its components, or prior daratumumab treatment with poor response lasting less than 6 months
- Uncontrolled serious diseases such as malignant tumors, liver failure, heart failure, or kidney failure
- HIV positive
- Active, uncontrollable infections including hepatitis B, hepatitis C, cytomegalovirus, EB virus, or syphilis
- Severe bleeding such as hemoptysis, gastrointestinal bleeding, or intracranial hemorrhage
- Current heart diseases, arrhythmias needing treatment, or poorly controlled hypertension
- History of thrombotic diseases like pulmonary embolism or thrombosis
- Previous allogeneic stem cell or organ transplantation
- Mental disorders preventing informed consent or trial participation
- Ongoing toxic symptoms from prior treatments
- Other serious illnesses limiting participation (e.g., diabetes, severe cardiac insufficiency, unstable angina)
- Septicemia or severe irregular bleeding
- Current use of antiplatelet drugs
- Pregnant, suspected pregnancy, or breastfeeding
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - 8 weeks
Participants receive intravenous Anti-CD38 antibody once a week for 8 weeks to assess safety and efficacy.
8 weekly visits (in-person)
Duration - 16 weeks
Participants are observed to monitor safety and continued efficacy after treatment ends.
Regular visits during weeks 9 to 24
Duration - Up to 5 years
Participants are monitored for long-term safety and sustained efficacy of the treatment.
Periodic visits up to 5 years
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
Y
Yunfei Chen, 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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