Thrombosomes® in Bleeding Thrombocytopenic Patients Study

NCT04631211 | Terminated Phase 2 DMC FDA Drug

• 1 other identifier: #2019-1
• Study Type: interventional
• Target: 21
• Locations: 3 countries
• Sites: 6

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.

Conditions

Thrombocytopenia; Hematologic Malignancy; Bone Marrow Aplasia; Myeloproliferative Disorders; Myelodysplastic Syndromes; Platelet Refractoriness

Outcome Measures

Primary Outcomes (1)

• Primary Efficacy Endpoint

  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.

  Evaluated at 24 hours post initial infusion

Secondary Outcomes (9)

• 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 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 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 for Number, timing, type and reason for administration of all blood products

  7 days after first Thrombosomes or LSP infusion

Other Outcomes (3)

• Safety Endpoint

  From baseline through last study visit (up to 30 days (+/- 2 days))

• Safety Endpoint

  From baseline through last study visit (up to 30 days (+/- 2 days))

• Safety Endpoint

  From baseline through last study visit (up to 30 days (+/- 2 days))

Study Arms (4)

Thrombosomes Low Dose

EXPERIMENTAL

Biological: Thrombosomes

Thrombosomes Medium Dose

EXPERIMENTAL

Biological: Thrombosomes

Thrombosomes High Dose

EXPERIMENTAL

Biological: Thrombosomes

Liquid Stored Platelets (Control)

ACTIVE COMPARATOR

Biological: Liquid Stored Platelets (LSP)

Interventions

Thrombosomes BIOLOGICAL

Human platelet derived lyophilized hemostatic

Thrombosomes High Dose; Thrombosomes Low Dose; Thrombosomes Medium Dose

Liquid Stored Platelets (LSP) BIOLOGICAL

Leukocyte reduced apheresis platelets or whole blood derived pooled platelet concentrate equivalent (4-6 units)

Liquid Stored Platelets (Control)

Eligibility Criteria

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

You may qualify if:

• 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

You may not qualify if:

• 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
• WHO Bleeding Score of 2 solely due to lumbar puncture, retinal bleeding or GI bleeding or WHO Bleeding Score of 3 solely due to lumbar puncture
• Receiving L-asparaginase as part of a current cycle of treatment
• Known inherited or acquired bleeding disorder including, but not limited to: acquired storage pool deficiency or paraproteinemia with platelet inhibition
• Known inherited or acquired prothrombotic disorders, including antiphospholipid syndrome (Those with lupus anticoagulant or positive antiphospholipid serology without thrombosis are NOT excluded.)
• Anuria

Sponsors & Collaborators

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

Study Sites (6)

City of Hope

Duarte, California, 91010, United States

Location

Medstar Georgetown

Washington D.C., District of Columbia, 20007, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Rambam Medical Center

Haifa, Israel

Location

Helse Bergen Haukeland University Hospital

Bergen, Norway

Location

MeSH Terms

Conditions

Thrombocytopenia; Hematologic Neoplasms; Anemia, Aplastic; Myeloproliferative Disorders; Myelodysplastic Syndromes

Condition Hierarchy (Ancestors)

Blood Platelet Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Cytopenia; Neoplasms by Site; Neoplasms; Anemia; Bone Marrow Failure Disorders; Bone Marrow Diseases

Study Officials

• Terry Gernsheimer, MD

  University of Washington

  PRINCIPAL INVESTIGATOR

• Mike Fitzpatrick, PhD

  Cellphire Therapeutics, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Model Details: Dose Ranging Multidose

Study Record Dates

• First Submitted: November 5, 2020
• First Posted: November 17, 2020
• Study Start: March 5, 2021
• Primary Completion: January 7, 2022
• Study Completion: January 7, 2022
• Last Updated: May 25, 2023

Record last verified: 2023-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (4); IL (1); NO (1)