Clinical trial NCT03394755: Thrombosomes® in Bleeding Thrombocytopenic Patients
Official title: A Phase I, Multi-Center, Open-Label, Dose Escalation Study of Thrombosomes® in Bleeding Thrombocytopenic Patients in Three Cohorts
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Thrombocytopenia
Hematologic Diseases
Bone Marrow Aplasia
Keywords: thrombocytopenia
bleeding
Lead sponsor: Cellphire Therapeutics, Inc.
Sponsor class: INDUSTRY
Collaborators: name: Department of Health and Human Services
First posted: 2018-01-09
Enrollment: 24
Sex: ALL
Minimum age (years): 18
Maximum age (years): 74
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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

Detailed 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.

Eligibility 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 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

Primary outcomes:
measure: Number of Patients With Treatment-Emergent Adverse Events (TEAE)
timeFrame: 30 days
measure: Number of Patients With Treatment-Emergent Serious Adverse Events (TESAE)
timeFrame: 30 days
Secondary outcomes:
measure: Number of WHO Bleeding Sites With Status Change From Baseline
timeFrame: 1, 6, 24 hours, and Day 6 post infusion
measure: Number of Patients With Grade-level Change in WHO Bleeding Assessment Score From Baseline - Patients WHO Score at Primary Bleeding Site
timeFrame: Baseline, 1, 6, 24 hours, and Day 6 post infusion
measure: Number of Patients With a Shift From Baseline in Hemoglobin
timeFrame: 1, 6, 24 hours, Day 3, 4, 5, and 6 post infusion
measure: Number of Patients With a Shift From Baseline in Hematocrit
timeFrame: 1, 6, 24 hours, Day 3, 4, 5, and 6 post infusion
measure: Number or Patients With a Shift From Baseline in Coagulation Measures 24 Hours Post Infusion
timeFrame: 24 hours post infusion
measure: Median Platelet Counts
timeFrame: Screening, Baseline, 1, 6, 24 hours, Day 3, 4, 5, and 6 post infusion
Other outcomes:
Locations:
city: Duarte
country: United States
facility: City of Hope
state: California
city: Washington
country: United States
facility: Georgetown University Hospital
state: District of Columbia
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Lebanon
country: United States
facility: Dartmouth Hitchcock Medical Center
state: New Hampshire
city: Cincinnati
country: United States
facility: Hoxworth Blood Center/University of Cincinnati
state: Ohio
city: Houston
country: United States
facility: MD Anderson Cancer Center
state: Texas
city: Bergen
country: Norway
facility: Haukeland University Hospital
Study officials:
affiliation: Cellphire Therapeutics, Inc.
name: Michael Fitzpatrick
role: STUDY_DIRECTOR
References:
citation: 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.