Thrombosomes® in Bleeding Thrombocytopenic Patients

• ClinicalTrials.gov Identifier: NCT03394755
• Recruitment Status: Active, not recruiting
• First Posted: January 9, 2018
• Last Update Posted: July 11, 2019
• Sponsor: Cellphire, Inc.
• Collaborator: Department of Health and Human Services
• Information provided by (Responsible Party): Cellphire, Inc.

Study Description

Brief Summary:

This study evaluates in a dose-escalation manner, the safety, and preliminary impact on bleeding, and the preliminary effect on coagulation measures of increasing doses of allogeneic Thrombosomes in thrombocytopenic patients.

Condition or disease	Intervention/treatment	Phase
Thrombocytopenia; Hematologic Diseases; Bone Marrow Aplasia	Biological: Thrombosomes	Phase 1

Detailed Description:

The objectives of this trial, conducted in thrombocytopenic patients with modified World Health Organization (WHO) Grade 1* or 2 bleeding, assessing the safety of infusing increasing doses of allogeneic Thrombosomes in WHO bleeding and measures of coagulation.

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 24 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Phase I, Multi-Center, Open-Label, Dose Escalation Study of Thrombosomes® in Bleeding Thrombocytopenic Patients in Three Cohorts
• Actual Study Start Date: March 19, 2018
• Estimated Primary Completion Date: August 3, 2019
• Estimated Study Completion Date: October 18, 2019

Arms and Interventions

Arm	Intervention/treatment
Experimental: Thrombosomes	Biological: Thrombosomes; Freeze-dried platelets

Outcome Measures

Primary Outcome Measures :

1. Frequency of adverse events [ Time Frame: 30 days ]

Secondary Outcome Measures :

1. Changes in WHO Bleeding Grade [ Time Frame: 6 days ]
2. Changes in Coagulation Measures [ Time Frame: 2 days ]
   Measures of PT/INR, aPTT, d-dimer, fibrinogen, TAT, PF1+2, TEG, TGA

Eligibility Criteria

• Ages Eligible for Study: 18 Years to 74 Years (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

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 during the immediate study 6 Day follow-up period.

Exclusion Criteria:

• Hx or condition related to thrombosis, embolism or vascular occlusion/ischemia, including but not limited to: TIA, stroke, MI, 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 or aPTT 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, TTP, or HUS
• Receipt of tranexamic acid or other antifibrinolytics within 48 hrs prior to infusion
• Rx with an investigational drug w/in 1 month of infusion, other than for treatment of their underlying disease

Contacts and Locations

Locations

United States, California

City of Hope

Duarte, California, United States, 91010

United States, District of Columbia

Georgetown University Hospital

Washington, District of Columbia, United States, 20016

United States, Illinois

Loyola University Medical Center

Chicago, Illinois, United States, 60130

United States, Michigan

University of Michigan

Ann Arbor, Michigan, United States, 48109

United States, New Hampshire

Dartmouth Hitchcock Medical Center

Lebanon, New Hampshire, United States, 03766

United States, Ohio

Hoxworth Blood Center

Cincinnati, Ohio, United States, 45219

United States, Texas

MD Anderson Cancer Center

Houston, Texas, United States, 77092

Norway

Haukeland University Hospital

Bergen, Norway

Sponsors and Collaborators

Cellphire, Inc.

Department of Health and Human Services

Investigators

• Study Director: Michael Fitzpatrick; Cellphire, Inc.

More Information

• Responsible Party: Cellphire, Inc.
• ClinicalTrials.gov Identifier: NCT03394755
• Other Study ID Numbers: 2017-1
• First Posted: January 9, 2018
• Last Update Posted: July 11, 2019
• Last Verified: January 2019

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Undecided

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Cellphire, Inc.:

thrombocytopenia; bleeding

Additional relevant MeSH terms:

Thrombocytopenia; Hematologic Diseases; Anemia, Aplastic; Hemorrhage; Pathologic Processes; Blood Platelet Disorders; Anemia; Bone Marrow Diseases