Efficacy of Daratumumab to Overcome Platelet Transfusion Refractoriness in Patients With Aplastic Anemia
Study Details
Study Description
Brief Summary
This is a phase 1, prospective, single-arm, open-label study. The aim of this study is to evaluate the transfusion responses of platelet increment by using Daratumumab among aplastic anemia patients with platelet transfusion refractoriness.
Condition or Disease | Intervention/Treatment | Phase |
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Phase 1 |
Study Design
Arms and Interventions
Arm | Intervention/Treatment |
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Experimental: Daratumumab
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Drug: Daratumumab
Daratumumab is a monoclonal globulin targeting the CD38 molecule of immune cells, which can eliminate antigen-presenting cells such as dendritic cells and macrophages, and indirectly reduce the production of autoantibodies. In addition, Daratumumab reduces autoantibody levels by targeting plasma cells, B lymphocytes and T lymphocytes.
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Outcome Measures
Primary Outcome Measures
- Percentage of patients with platelet transfusion responsiveness [12 weeks]
To evaluate the safety and efficacy of Daratumumab to increase the platelet increment, defined as Corrected Count Increment (CCI) ≥7500/μL at 60 min or CCI≥4500/μL at 24 hrs post transfusion, or platelet transfusion independence, i.e. PLT>10×10^9/L without any bleeding events.
Secondary Outcome Measures
- Time to the platelet increment [12 weeks]
Eligibility Criteria
Criteria
Inclusion Criteria:
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Diagnosis of aplastic anemia (AA)
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Dependent on platelet transfusions, characterized by either PLT<10×109/L, or PLT<20×109/L with bleeding events.
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Diagnosed with platelet transfusion refractoriness, characterized by Corrected count increment (CCI) <7500/ul at 60 min, or CCI <4500/ul at 24 hrs.
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Male or female age ≥ 12 years
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ECOG performance status ≤2
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Willing and able to comply with the requirements for this study and written informed consent.
Exclusion Criteria:
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The inherited bone marrow failure syndromes
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The presence of hemolytic PNH clone
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Combination of either radiotherapy or chemotherapy for solid tumors in recent 3 years, excluding the local tumor diagnosed 1 year ago and cured by surgical resection.
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Cytopenia caused by other diseases, including liver cirrhosis, active rheumatic connective tissue disease, and persistence of infectious diseases, etc.
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Uncontrolled infection
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HIV, HCV or HBV active infection
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The presence of any of the following bleeding events:
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Gastrointestinal bleeding
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Respiratory tract hemorrhage
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Central nervous system bleeding
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Abnormal liver function: ALT or AST > 2 ULN, or TBil > 2 ULN after treatment.
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Abnormal kidney function: Creatinine clearance < 30 ml/min
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Heart failure (NYHA class III or IV)
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Poorly controlled diabetes, characterized by fasting blood glucose > 8.8mmol/L or post-meal blood glucose > 11.1mmol/L after therapy with insulin or oral hypoglycemic agents
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History of congestive heart failure, unstable angina pectoris, myocardial infarction, arterial or venous thrombosis
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Pregnant or breast-feeding patients
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Had a history of any psychiatric diseases, cerebrovascular disease or cognitive sequelae of head injury.
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Participation in another clinical trial within 4 weeks before the start of this trial
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Have an allergy to Daratumumab or any other part of this medicine.
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Previously treated with Daratumumab
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Previously treated with ATG/ALG within 4 months before the start of this trial
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Previously treated with the anti-CD20 monoclonal antibody within 2 months before the start of this trial
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Currently treated with TPO-RA except for a minimum of 4 weeks for washout before the start of this trial
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Patients considered to be ineligible for the study by the investigator for reasons other than above
Contacts and Locations
Locations
Site | City | State | Country | Postal Code | |
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1 | Regenerative Medicine Center | Tianjin | Tianjin | China |
Sponsors and Collaborators
- Institute of Hematology & Blood Diseases Hospital
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- 2023NCRCA0112