Thrombosomes® in Bleeding Thrombocytopenic Patients

Cellphire Therapeutics, Inc.24 enrolled

Overview

The study evaluates the safety and potential early signals of efficacy of allogeneic Thrombosomes in bleeding thrombocytopenic patients

The primary objective of the present study was to assess the safety of increasing dose levels of Thrombosomes in bleeding patients with thrombocytopenia. The secondary objective was to explore early signals of clinical efficacy of Thrombosomes in this population. The secondary objectives included: 1) Evaluation of the impact on WHO (World Health Organization) bleeding scores at various timepoints; 2) number and type of blood products infused through day 6 follow-up period; and 3) post hoc analysis of hematology, coagulation, and chemistry.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Thrombocytopenia Hematologic Diseases Bone Marrow Aplasia

Interventions

ThrombosomesBIOLOGICAL

Freeze-dried platelets

Eligibility

Sex: ALLMin age: 18 YearsMax age: 74 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Adults up to 74 y/o with any of following: acute leukemia (ALL or AML), myelodysplasia, aplasia, and/or therapy (chemotherapy or radiation) induced bone marrow aplasia or hypoplasia with thrombocytopenia (platelet count ≥ 5,000 and ≤ 70,000/μL) for a minimum of 2 days. May include bone marrow transplant or peripheral or cord blood stem cell recipients, but not subjects with Graft-vs-Host disease. * Hospitalized patients (or willing to be hospitalized for 24 hours after Rx) with Modified WHO Grade 1 (subset) or Grade 2 Bleeding Score or at risk for same within 4 weeks of screening. The Grade 1 subset includes patients who have either epistaxis, hematuria, oral petechiae, or bleeding at invasive or other wound sites. * No platelet inhibitor drugs within 5 days prior to infusion and through Day 6 follow-up period. Exclusion Criteria: * History or condition related to thrombosis, embolism or vascular occlusion/ischemia, including but not limited to: transient ischemic attack, stroke, myocardial infarction, stent placement, valve replacement and/or repair * Currently with an active acute infection, or suspected infection, a single oral temperature of ≥ 101° F or a temperature of ≥ 100.4°F sustained over a 1 h period in past 24 h. Subjects on prophylactic antibiotics are not excluded from study * Coagulopathy or receiving anticoagulants that result in PT (prothrombin time) or aPTT (activated partial thromboplastin time) values greater than 1.3 X upper limit of normal or elevated D-dimer of decreased fibrinogen * History of any inherited coagulation or platelet function, disorder or ITP (idiopathic thrombocytopenic purpura), TTP (thrombotic thrombocytopenic purpura), or HUS (hemolytic-uremic syndrome) * Receipt of tranexamic acid or other antifibrinolytics within 48 hrs prior to infusion * Treatment with an investigational drug within 1 month of infusion, other than for treatment of their underlying disease

Locations (7)

City of Hope

Duarte, California, United States

Georgetown University Hospital

Washington D.C., District of Columbia, United States

University of Michigan

Ann Arbor, Michigan, United States

Dartmouth Hitchcock Medical Center

Lebanon, New Hampshire, United States

Outcomes

Primary Outcomes

Number of Patients With Treatment-Emergent Adverse Events (TEAE)

Overall frequency of (and number and percentage of patients who experience) TEAEs including serious adverse drug reactions and treatment-related events specifically defining the study's suspension and stopping rules (i.e., thromboembolic events, acute lung injury, anaphylaxis, and death).

Time frame: 30 days

Number of Patients With Treatment-Emergent Serious Adverse Events (TESAE)

Overall frequency of (and number and percentage of patients who experience) TESAEs including serious adverse drug reactions and treatment-related events specifically defining the study's suspension and stopping rules (i.e., thromboembolic events, acute lung injury, anaphylaxis, and death).

Time frame: 30 days

Secondary Outcomes

Number of WHO Bleeding Sites With Status Change From Baseline

Grade-level change in WHO bleeding assessment score at most severe bleeding site from baseline; WHO bleeding assessment score ranges from 1 to 4, with higher numbers indicating worse bleeding.

Time frame: 1, 6, 24 hours, and Day 6 post infusion

Number of Patients With Grade-level Change in WHO Bleeding Assessment Score From Baseline - Patients WHO Score at Primary Bleeding Site

Patients WHO score at primary bleeding site. WHO bleeding assessment score ranges from 1 to 4, with higher numbers indicating worse bleeding. The maximum WHO bleeding assessment score for patients on study was 2.

Time frame: Baseline, 1, 6, 24 hours, and Day 6 post infusion

Number of Patients With a Shift From Baseline in Hemoglobin

Shift from baseline clinical status of hemoglobin measure at different timepoints post infusion

Time frame: 1, 6, 24 hours, Day 3, 4, 5, and 6 post infusion

Data from ClinicalTrials.gov

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