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

Sponsor

Institute of Hematology & Blood Diseases Hospital (Other)

Overall Status

Recruiting

CT.gov ID

NCT05832216

Collaborator

(none)

Enrollment

Location

Arm

44.5

Anticipated Duration (Months)

0.2

Patients Per Site Per Month

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
Aplastic Anemia Platelet Transfusion Refractoriness	Drug: Daratumumab	Phase 1

Study Design

Study Type:

Interventional

Anticipated Enrollment :

10 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Actual Study Start Date :

Apr 17, 2023

Anticipated Primary Completion Date :

Mar 31, 2026

Anticipated Study Completion Date :

Dec 31, 2026

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Daratumumab	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.

Arm

Intervention/Treatment

Experimental: Daratumumab

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.

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

Ages Eligible for Study:

12 Years and Older

Sexes Eligible for Study:

All

Accepts 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
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.

Responsible Party:

Institute of Hematology & Blood Diseases Hospital

ClinicalTrials.gov Identifier:

NCT05832216

Other Study ID Numbers:

2023NCRCA0112

First Posted:

Apr 27, 2023

Last Update Posted:

Apr 27, 2023

Last Verified:

Mar 1, 2023

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Keywords provided by Institute of Hematology & Blood Diseases Hospital

Platelet transfusion refractoriness

Daratumumab

Additional relevant MeSH terms:

Anemia

Anemia, Aplastic

Daratumumab

Study Results

No Results Posted as of Apr 27, 2023