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

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.

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Key information

Conditions

Aplastic Anemia Anemia Anemia, Aplastic Bone Marrow Diseases Bone Marrow Failure Disorders Hematologic Diseases Hemic and Lymphatic Diseases Platelet Transfusion Refractoriness

Age range

12 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Regenerative Medicine Center

Tianjin, Tianjin Municipality, China

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Daratumumab

Drug

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.

Primary outcomes

Percentage of patients with platelet transfusion responsiveness

Time frame: 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 outcomes

Time to the platelet increment

Time frame: 12 weeks

Sponsors and collaborators

Lead sponsor

Institute of Hematology & Blood Diseases Hospital, China

Other

Registry information

Important dates

Study start: 2023
Primary completion: 2024
Study completion: 2024
First posted: Apr 27, 2023
Registry last updated: Jan 3, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed