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

Primary Purpose

Aplastic Anemia, Platelet Transfusion Refractoriness

Status
Recruiting
Phase
Phase 1
Locations
China
Study Type
Interventional
Intervention
Daratumumab
Sponsored by
Institute of Hematology & Blood Diseases Hospital, China
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Aplastic Anemia focused on measuring Platelet transfusion refractoriness, Daratumumab

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept 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

Sites / Locations

  • Regenerative Medicine CenterRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Daratumumab

Arm Description

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Time to the platelet increment

Full Information

First Posted
April 3, 2023
Last Updated
April 25, 2023
Sponsor
Institute of Hematology & Blood Diseases Hospital, China
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1. Study Identification

Unique Protocol Identification Number
NCT05832216
Brief Title
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

2. Study Status

Record Verification Date
March 2023
Overall Recruitment Status
Recruiting
Study Start Date
April 17, 2023 (Actual)
Primary Completion Date
March 31, 2026 (Anticipated)
Study Completion Date
December 31, 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Institute of Hematology & Blood Diseases Hospital, China

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Aplastic Anemia, Platelet Transfusion Refractoriness
Keywords
Platelet transfusion refractoriness, Daratumumab

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Daratumumab
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Daratumumab
Intervention Description
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 Outcome Measure Information:
Title
Percentage of patients with platelet transfusion responsiveness
Description
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.
Time Frame
12 weeks
Secondary Outcome Measure Information:
Title
Time to the platelet increment
Time Frame
12 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Zhen Gao
Phone
‭15522360862‬
Email
gaozhen@ihcams.ac.cn
Facility Information:
Facility Name
Regenerative Medicine Center
City
Tianjin
State/Province
Tianjin
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Zhen Gao, MD
Phone
15522360862
Email
gaozhen@ihcams.ac.cn

12. IPD Sharing Statement

Plan to Share IPD
No

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

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