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

Institute of Hematology & Blood Diseases Hospital, China

Status and phase

Completed

Phase 1

Conditions

Platelet Transfusion Refractoriness

Aplastic Anemia

Treatments

Drug: Daratumumab

Study type

Interventional

Funder types

Other

Identifiers

NCT05832216

IIT2023015

Details and patient eligibility

About

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.

Enrollment

10 patients

Sex

All

Ages

12+ years old

Volunteers

No Healthy Volunteers

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