Thrombosomes® in Bleeding Thrombocytopenic Patients

NCT03394755 | Completed Phase 1 Results DMC FDA Drug

• 1 other identifier: 2017-1
• Study Type: interventional
• Target: 24
• Locations: 2 countries
• Sites: 7

Brief Summary

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

Conditions

Thrombocytopenia; Hematologic Diseases; Bone Marrow Aplasia

Keywords

thrombocytopenia; bleeding

Outcome Measures

Primary Outcomes (2)

• 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

• 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 Outcomes (6)

• Number of WHO Bleeding Sites With Status Change From Baseline

  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

  Baseline, 1, 6, 24 hours, and Day 6 post infusion

• Number of Patients With a Shift From Baseline in Hemoglobin

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

• Number of Patients With a Shift From Baseline in Hematocrit

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

• Number or Patients With a Shift From Baseline in Coagulation Measures 24 Hours Post Infusion

  24 hours post infusion

Study Arms (3)

9.45 x 10^7 Thrombosomes/kg

EXPERIMENTAL

Cohort 1

Biological: Thrombosomes

1.89 x 10^8 Thrombosomes/kg

EXPERIMENTAL

Cohort 2

Biological: Thrombosomes

3.78 x10^8 Thrombosomes/kg

EXPERIMENTAL

Cohort 3

Biological: Thrombosomes

Interventions

Thrombosomes BIOLOGICAL

Freeze-dried platelets

1.89 x 10^8 Thrombosomes/kg; 3.78 x10^8 Thrombosomes/kg; 9.45 x 10^7 Thrombosomes/kg

Eligibility Criteria

• Age: 18 Years - 74 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Cellphire Therapeutics, Inc.: lead
• Department of Health and Human Services: collaborator

Study Sites (7)

City of Hope

Duarte, California, 91010, United States

Location

Georgetown University Hospital

Washington D.C., District of Columbia, 20016, United States

Location

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

Dartmouth Hitchcock Medical Center

Lebanon, New Hampshire, 03766, United States

Location

Hoxworth Blood Center/University of Cincinnati

Cincinnati, Ohio, 45219, United States

Location

MD Anderson Cancer Center

Houston, Texas, 77092, United States

Location

Haukeland University Hospital

Bergen, Norway

Location

Related 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.

  PMID: 34748664 BACKGROUND

MeSH Terms

Conditions

Thrombocytopenia; Hematologic Diseases; Anemia, Aplastic; Hemorrhage

Condition Hierarchy (Ancestors)

Blood Platelet Disorders; Hemic and Lymphatic Diseases; Cytopenia; Anemia; Bone Marrow Failure Disorders; Bone Marrow Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Mike Fitzpatrick, PhD
• Organization: Cellphire Therapeutics, Inc.

mfitzpatrick@cellphire.com

240-268-2470

Study Officials

• Michael Fitzpatrick

  Cellphire Therapeutics, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 3, 2018
• First Posted: January 9, 2018
• Study Start: March 19, 2018
• Primary Completion: August 1, 2019
• Study Completion: September 25, 2019
• Last Updated: April 14, 2023
• Results First Posted: April 14, 2023

Record last verified: 2023-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (6); NO (1)