A Prospective, Multicenter, Randomized, Open-Label Phase 2, Parallel, Dose Ranging Multidose Study of Thrombosomes® vs Liquid Stored Platelets (LSP) in Bleeding Thrombocytopenic Patients

Cellphire Therapeutics, Inc.6 sites in 3 countries21 target enrollmentMarch 5, 2021

ConditionsThrombocytopenia Hematologic Malignancy Bone Marrow Aplasia Myeloproliferative Disorders Myelodysplastic Syndromes Platelet Refractoriness

Overview

Phase: Phase 2
Intervention: Not specified
Conditions: Thrombocytopenia
Sponsor: Cellphire Therapeutics, Inc.
Enrollment: 21
Locations: 6
Primary Endpoint: Primary Efficacy Endpoint
Status: Terminated
Last Updated: 2 years ago

Overview Investigators Eligibility Criteria Outcomes Sites Similar Trials

Overview

Brief Summary

This prospective, multicenter, randomized, open-label, Phase 2, parallel, dose ranging, multidose trial will enroll patients into 3 Thrombosomes dose groups and 1 control liquid stored platelets (LSP) group in order to evaluate, in a dose-escalation manner, the safety, and impact on bleeding, and the preliminary effect on coagulation measures of increasing doses of allogeneic Thrombosomes in comparison to standard of care, LSP.

Registry

clinicaltrials.gov

Start Date

March 5, 2021

End Date

January 7, 2022

Last Updated

2 years ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Cellphire Therapeutics, Inc.

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Adults (≥18 years) with TCP as defined by BOTH (a) and (b):
•a platlet count of ≤ 70,000 platelets/μL blood
•ANY ONE OR MORE of (1-3):
•confirmed diagnosis of hematologic malignancy, myeloproliferative disorder, myelodysplastic syndrome, or aplasia
•undergoing chemotherapy, immunotherapy, radiation therapy or hematopoietic stem cell transplantation
•refractory to platelet transfusion defined as two 1-hour CCI of \<5,000 on consecutive transfusions of LSP or as defined by local site policy (Sacher, 2003)
•WHO Bleeding Score of 2 or 3
•Able to provide informed consent directly or through legally authorized representative, and comply with treatment and monitoring
•Negative pregnancy test for women of childbearing potential

Exclusion Criteria

•Any disorder or condition related to any venous thrombosis, embolism, or ischemia within the past 3 months
•Any disorder or condition related to arterial thrombosis including: ischemia, stroke, MI, or stent placement, within past 6 months
•Any valve replacement and/or repair of left atrial appendance occlusion device
•Sinusoidal obstruction syndrom (veno-occlusive disease) or cytopkine release syndrome associated with CAR-T cell therapy
•Refusal to accept blood products
•Liver enzyme blood levels greater than 3× the upper limit of normal (ULN)
•Blood creatinine level greater than 3× ULN
•Received platelet inhibitor drugs, cyclooxygenase-2 (COX-2) inhibitors, or nonsteroidal anti-inflammatory drugs within 5 days prior to infusion
•Currently (at the time of randomization) receiving anticoagulant therapy or antiplatelet therapy. Low dose prophylaxis for line clots is not excluded.
•Receipt of any pro-coagulant agents (e.g., DDAVP, recombinant Factor VIIa or prothrombin complex concentrates (PCC)) other than Tranexamic Acid (TXA) or Epsilon Aminocaproic Acid (EACA, Amicar), within 48 hours of first infusion, or with known hypercoagulable state

Outcomes

Primary Outcomes

Primary Efficacy Endpoint

Time Frame: Evaluated at 24 hours post initial infusion

Cessation or decrease in bleeding at primary bleeding site, based upon the most severe bleeding location at Day 1 baseline taken with in 12 hours prior to infusion, as evidenced by ordinal change in WHO (World Health Organization) Bleeding Score evaluated at 24 hours post initial infusion.

Secondary Outcomes

Secondary Efficacy Endpoint assessed by Number of days alive and without WHO (World Health Organization) Grade 2a or greater bleeding(7 days after first Thrombosomes or LSP infusion)
Secondary Efficacy Endpoint assessed for Number, timing, type and reason for administration of all blood products(7 days after first Thrombosomes or LSP infusion)
Secondary Efficacy Endpoint assessed by 30 day mortality(30 days post first infusion (+/- 2 days))
Secondary Efficacy Endpoint assessed by cessation or decrease in bleeding, as evidenced by ordinal change in WHO (World Health Organization) Bleeding Score(24, 48, 72 hours, Day 4, Day 5, Day 6 and Day 7 post first infusion)
Secondary Efficacy Endpoint assessed by measures of coagulation(From baseline through last study visit (up to 30 days (+/- 2 days)))
Secondary Efficacy Endpoint assessed by changes in markers of endothelial cell injury/repair(From baseline through last study visit (up to 30 days (+/- 2 days)))
Secondary Efficacy Endpoint assessed by platelet count(24, 48, 72 hours and Day 7 post first infusion)
Secondary Efficacy Endpoint assessed by measures of hematology(From baseline through last study visit (up to 30 days (+/- 2 days)))