Phase 1
N=24
Thrombosomes® in Bleeding Thrombocytopenic Patients
Thrombocytopenia · Hematologic Diseases · Bone Marrow Aplasia
Bottom Line
View on ClinicalTrials.gov: NCT03394755 ↗Enrolled (actual)
24
Serious AEs
41.7%
Results posted
Apr 2023
Primary outcome: Primary: Number of Patients With Treatment-Emergent Adverse Events (TEAE) — 7; 6; 5 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 1
- Interventions
- Thrombosomes (Biological)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Cellphire Therapeutics, Inc.
- Primary completion
- Aug 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Patients With Treatment-Emergent Adverse Events (TEAE) |
7; 6; 5 | — |
| PRIMARY Number of Patients With Treatment-Emergent Serious Adverse Events (TESAE) |
1; 5; 4 | — |
| SECONDARY Number of WHO Bleeding Sites With Status Change From Baseline |
0; 24; 3; 0; 21; 6 | — |
| SECONDARY Number of Patients With Grade-level Change in WHO Bleeding Assessment Score From Baseline - Patients WHO Score at Primary Bleeding Site |
0; 7; 15; 1; 7; 14 | — |
| SECONDARY Number of Patients With a Shift From Baseline in Hemoglobin |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Number of Patients With a Shift From Baseline in Hematocrit |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Number or Patients With a Shift From Baseline in Coagulation Measures 24 Hours Post Infusion |
6; 5; 5; 0; 1; 0 | — |
| SECONDARY Median Platelet Counts |
16; 18.5; 13.5; 14; 20.5; 22.5 | — |
Summary
The study evaluates the safety and potential early signals of efficacy of allogeneic Thrombosomes in bleeding thrombocytopenic patients
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
Data sourced from ClinicalTrials.gov (NCT03394755). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.