Efficacy of Daratumumab to Overcome Platelet Transfusion Refractoriness in Patients With Aplastic Anemia

Aplastic Anemia Clinical Trial

Official title:

Efficacy of Daratumumab to Overcome Platelet Transfusion Refractoriness in Patients With Aplastic Anemia

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05832216
• Other study ID #: 2023NCRCA0112
• Status: Recruiting
• Phase: Phase 1
• Start date: April 17, 2023
• Est. completion date: December 31, 2026

Study information

• Verified date: March 2023
• Source: Institute of Hematology & Blood Diseases Hospital
• Contact: Zhen Gao
• Phone: ?15522360862?
• Email: gaozhen[@]ihcams.ac.cn
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 10
• Est. completion date: December 31, 2026
• Est. primary completion date: March 31, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

Inclusion Criteria:

• Diagnosis of aplastic anemia (AA)
• Dependent on platelet transfusions, characterized by either PLT<10×10^9/L, or PLT<20×10^9/L with bleeding events.
• Diagnosed with platelet transfusion refractoriness, characterized by Corrected count increment (CCI) <7500/ul at 60 min, or CCI <4500/ul at 24 hrs.
• Male or female age = 12 years
• ECOG performance status =2
• Willing and able to comply with the requirements for this study and written informed consent.

Exclusion Criteria:

• The inherited bone marrow failure syndromes
• The presence of hemolytic PNH clone
• 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.
• Cytopenia caused by other diseases, including liver cirrhosis, active rheumatic connective tissue disease, and persistence of infectious diseases, etc.
• Uncontrolled infection
• HIV, HCV or HBV active infection
• The presence of any of the following bleeding events:
• Gastrointestinal bleeding
• Respiratory tract hemorrhage
• Central nervous system bleeding
• Abnormal liver function: ALT or AST > 2 ULN, or TBil > 2 ULN after treatment.
• Abnormal kidney function: Creatinine clearance < 30 ml/min
• Heart failure (NYHA class III or IV)
• 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
• History of congestive heart failure, unstable angina pectoris, myocardial infarction, arterial or venous thrombosis
• Pregnant or breast-feeding patients
• Had a history of any psychiatric diseases, cerebrovascular disease or cognitive sequelae of head injury.
• Participation in another clinical trial within 4 weeks before the start of this trial
• Have an allergy to Daratumumab or any other part of this medicine.
• Previously treated with Daratumumab
• Previously treated with ATG/ALG within 4 months before the start of this trial
• Previously treated with the anti-CD20 monoclonal antibody within 2 months before the start of this trial
• Currently treated with TPO-RA except for a minimum of 4 weeks for washout before the start of this trial
• Patients considered to be ineligible for the study by the investigator for reasons other than above

Related Conditions & MeSH terms

• Anemia
• Anemia, Aplastic
• Aplastic Anemia
• Platelet Transfusion Refractoriness

Intervention

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.

Locations

Country	Name	City	State
China	Regenerative Medicine Center	Tianjin	Tianjin

Sponsors (1)

Lead Sponsor	Collaborator
Institute of Hematology & Blood Diseases Hospital

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percentage of patients with platelet transfusion responsiveness	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.	12 weeks
Secondary	Time to the platelet increment		12 weeks