Clinical trial NCT05832216: Efficacy of Daratumumab to Overcome Platelet Transfusion Refractoriness in Patients with Aplastic Anemia
Official title: Efficacy of Daratumumab to Overcome Platelet Transfusion Refractoriness in Patients with Aplastic Anemia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Aplastic Anemia
Platelet Transfusion Refractoriness
Keywords: Platelet transfusion refractoriness
Daratumumab
Lead sponsor: Institute of Hematology & Blood Diseases Hospital, China
Sponsor class: OTHER
First posted: 2023-04-27
Enrollment: 10
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Percentage of patients with platelet transfusion responsiveness
timeFrame: 12 weeks
Secondary outcomes:
measure: Time to the platelet increment
timeFrame: 12 weeks
Other outcomes:
Locations:
city: Tianjin
country: China
facility: Regenerative Medicine Center
state: Tianjin
Study officials:
References: