Thrombosomes® in Bleeding Thrombocytopenic Patients

Thrombocytopenia Clinical Trial

Official title:

A Phase I, Multi-Center, Open-Label, Dose Escalation Study of Thrombosomes® in Bleeding Thrombocytopenic Patients in Three Cohorts

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03394755
• Other study ID #: 2017-1
• Status: Completed
• Phase: Phase 1
• Start date: March 19, 2018
• Est. completion date: September 25, 2019

Study information

• Verified date: April 2023
• Source: Cellphire Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Description:

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 24
• Est. completion date: September 25, 2019
• Est. primary completion date: August 1, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 74 Years

Eligibility

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

Related Conditions & MeSH terms

• Anemia, Aplastic
• Bone Marrow Aplasia
• Hematologic Diseases
• Hemorrhage
• Thrombocytopenia

Intervention

Biological:
• Thrombosomes
Freeze-dried platelets

Locations

Country	Name	City	State
Norway	Haukeland University Hospital	Bergen
United States	University of Michigan	Ann Arbor	Michigan
United States	Hoxworth Blood Center/University of Cincinnati	Cincinnati	Ohio
United States	City of Hope	Duarte	California
United States	MD Anderson Cancer Center	Houston	Texas
United States	Dartmouth Hitchcock Medical Center	Lebanon	New Hampshire
United States	Georgetown University Hospital	Washington	District of Columbia

Sponsors (2)

Lead Sponsor	Collaborator
Cellphire Therapeutics, Inc.	Department of Health and Human Services

Countries where clinical trial is conducted

United States,  Norway, 

References & Publications (1)

Ohanian M, Cancelas JA, Davenport R, Pullarkat V, Hervig T, Broome C, Marek K, Kelly M, Gul Z, Rugg N, Nestheide S, Kinne B, Szczepiorkowski Z, Kantarjian H, Pehta J, Biehl R, Yu A, Aung F, Antebi B, Fitzpatrick GM. Freeze-dried platelets are a promising alternative in bleeding thrombocytopenic patients with hematological malignancies. Am J Hematol. 2022 Mar 1;97(3):256-266. doi: 10.1002/ajh.26403. Epub 2021 Dec 23. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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).	30 days
Primary	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).	30 days
Secondary	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.	1, 6, 24 hours, and Day 6 post infusion
Secondary	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.	Baseline, 1, 6, 24 hours, and Day 6 post infusion
Secondary	Number of Patients With a Shift From Baseline in Hemoglobin	Shift from baseline clinical status of hemoglobin measure at different timepoints post infusion	1, 6, 24 hours, Day 3, 4, 5, and 6 post infusion
Secondary	Number of Patients With a Shift From Baseline in Hematocrit	Shift from baseline clinical status of hematocrit measure at different timepoints post infusion	1, 6, 24 hours, Day 3, 4, 5, and 6 post infusion
Secondary	Number or Patients With a Shift From Baseline in Coagulation Measures 24 Hours Post Infusion	Shift from baseline clinical status of coagulation measure at 24 hours post infusion for each cohort	24 hours post infusion
Secondary	Median Platelet Counts	Median Platelet Counts Per Time Point	Screening, Baseline, 1, 6, 24 hours, Day 3, 4, 5, and 6 post infusion