NCT05832216

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
10

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Apr 2023

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

April 3, 2023

Completed
14 days until next milestone

Study Start

First participant enrolled

April 17, 2023

Completed
10 days until next milestone

First Posted

Study publicly available on registry

April 27, 2023

Completed
1.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 12, 2024

Completed
2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2024

Completed
Last Updated

January 3, 2025

Status Verified

January 1, 2025

Enrollment Period

1.5 years

First QC Date

April 3, 2023

Last Update Submit

January 1, 2025

Conditions

Aplastic AnemiaPlatelet Transfusion Refractoriness

Keywords

Platelet transfusion refractorinessDaratumumab

Outcome Measures

Primary Outcomes (1)

  • 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.

    12 weeks

Secondary Outcomes (1)

  • Time to the platelet increment

    12 weeks

Study Arms (1)

Daratumumab

EXPERIMENTAL
Drug: Daratumumab

Interventions

DaratumumabDRUG

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.

Daratumumab

Eligibility Criteria

Age12 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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
  • +9 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Regenerative Medicine Center

Tianjin, Tianjin Municipality, China

Location

Related Publications (1)

  • Gao Z, Pan H, Zhang L, Li W, Li R, Zhao J, Luo Y, Lian Y, Yu X, Kuang Z, Nie N, Li J, Huang J, Zhao X, Li Y, Fang L, Ge M, Zheng Y, Shi J. Targeting CD38 with daratumumab for platelet transfusion refractoriness in aplastic anemia. Blood. 2025 Jun 26;145(26):3189-3193. doi: 10.1182/blood.2025029006.

    PMID: 40331918DERIVED

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

daratumumab

Condition Hierarchy (Ancestors)

AnemiaHematologic DiseasesHemic and Lymphatic DiseasesBone Marrow Failure DisordersBone Marrow Diseases

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 3, 2023

First Posted

April 27, 2023

Study Start

April 17, 2023

Primary Completion

October 12, 2024

Study Completion

December 1, 2024

Last Updated

January 3, 2025

Record last verified: 2025-01

Data Sharing

IPD Sharing
Will not share

Locations

CN(1)