COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Thrombosomes® in Bleeding Thrombocytopenic Patients Study

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04631211
Recruitment Status : Not yet recruiting
First Posted : November 17, 2020
Last Update Posted : January 20, 2021
Sponsor:
Collaborator:
Department of Health and Human Services
Information provided by (Responsible Party):
Cellphire, Inc.

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.

Condition or disease Intervention/treatment Phase
Thrombocytopenia Hematologic Malignancy Bone Marrow Aplasia Myeloproliferative Disorders Myelodysplastic Syndromes Platelet Refractoriness Biological: Thrombosomes Biological: Liquid Stored Platelets (LSP) Phase 2

Expanded Access : An investigational treatment associated with this study is available outside the clinical trial.   More info ...

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 160 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Dose Ranging Multidose
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Prospective, Multicenter, Randomized, Open-Label Phase 2, Parallel, Dose Ranging Multidose Study of Thrombosomes® vs Liquid Stored Platelets (LSP) in Bleeding Thrombocytopenic Patients
Estimated Study Start Date : February 2021
Estimated Primary Completion Date : February 2022
Estimated Study Completion Date : February 2022


Arm Intervention/treatment
Experimental: Thrombosomes Low Dose Biological: Thrombosomes
Human platelet derived lyophilized hemostatic

Experimental: Thrombosomes Medium Dose Biological: Thrombosomes
Human platelet derived lyophilized hemostatic

Experimental: Thrombosomes High Dose Biological: Thrombosomes
Human platelet derived lyophilized hemostatic

Active Comparator: Liquid Stored Platelets (Control) Biological: Liquid Stored Platelets (LSP)
Leukocyte reduced apheresis platelets or whole blood derived pooled platelet concentrate equivalent (4-6 units)




Primary Outcome Measures :
  1. 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 Outcome Measures :
  1. Secondary Efficacy Endpoint assessed by Number of days alive and without WHO (World Health Organization) Grade 2a or greater bleeding [ Time Frame: 7 days after first Thrombosomes or LSP infusion ]
    Number of days alive and without WHO (World Health Organization) Grade 2a or greater bleeding through initial 7 days after first Thrombosomes or LSP Infusion

  2. Secondary Efficacy Endpoint assessed by 30 day mortality [ Time Frame: 30 days post first infusion (+/- 2 days) ]
    30-day mortality post first infusion of Thrombosomes or post first infusion of LSP as control

  3. Secondary Efficacy Endpoint assessed by cessation or decrease in bleeding, as evidenced by ordinal change in WHO (World Health Organization) Bleeding Score [ Time Frame: 12, 24, 48, 72 hours and Day 7 post first infusion ]
    Cessation or decrease in primary bleeding site, as evidenced by ordinal change in WHO (World Health Organization) Bleeding Score at 12, 24, 48, 72 hours and Day 7 after first infusion of Thrombosomes or LSP infusion as control. Also evaluate at Days 4-6 if patient is hospitalized at that time.

  4. Secondary Efficacy Endpoint assessed by cessation or decrease in bleeding, as evidenced by ordinal change in WHO (World Health Organization) Bleeding Score [ Time Frame: 12, 24, 48, 72 hours and Day 7 post first infusion ]
    Cessation or decrease in each additional bleeding site (other than primary bleeding site), as evidenced by ordinal change in WHO (World Health Organization) Bleeding Score at 12, 24, 48, 72 hours and Day 7 after first infusion of Thrombosomes or LSP infusion as control. Also, evaluate at Days 4-6 if patient is hospitalized at that time.

  5. Secondary Efficacy Endpoint assessed for Number, timing, type and reason for administration of all blood products [ Time Frame: 7 days after first Thrombosomes or LSP infusion ]
    Number, timing, type and reason for administration of all blood products including platelets and Thrombosomes during the initial 7 days after first Thrombosomes or LSP infusion

  6. Secondary Efficacy Endpoint assessed by platelet count [ Time Frame: 12, 24, 48, 72 hours and Day 7 post first infusion ]
    Platelet count measured at 12, 24, 48, 72 hours and Day 7 of first infusion of Thrombosomes or LSP. Also evaluate at Day 4-6 if patient is hospitalized at that time.

  7. Secondary Efficacy Endpoint assessed by measures of hematology [ Time Frame: From baseline through last study visit (up to 30 days (+/- 2 days)) ]
    Measures of hematology including: Prothrombin Fragment 1+2; thrombin generation assay (TGA); Thrombopoietin; activated Protein C, tissue plasminogen activator (TPA), and plasminogen activator inhibitor (PAI) per schedule of assessments

  8. Secondary Efficacy Endpoint assessed by measures of coagulation [ Time Frame: From baseline through last study visit (up to 30 days (+/- 2 days)) ]
    Measures of coagulation including: prothrombin time (PT); international normalized ratio (INR); fibrinogen; D-dimer; activated partial thromboplastin time (aPTT); and thromboelastography (TEG) or rotational thromboelastometry (ROTEM) per schedule of assessments

  9. Secondary Efficacy Endpoint assessed by changes in markers of endothelial cell injury/repair [ Time Frame: From baseline through last study visit (up to 30 days (+/- 2 days)) ]
    Changes in markers of endothelial cell injury/repair from preinfusion baseline through 72 hours after first infusion, including: Syndecan-1, hyaluronan, thrombomodulin, vascular endothelial growth factor (VEGF), interleukin 6, sVE cadherin per schedule of assessments.


Other Outcome Measures:
  1. Safety Endpoint [ Time Frame: From baseline through last study visit (up to 30 days (+/- 2 days)) ]
    Serious Adverse Events (SAEs)

  2. Safety Endpoint [ Time Frame: From baseline through last study visit (up to 30 days (+/- 2 days)) ]
    Adverse Events (AEs)

  3. Safety Endpoint [ Time Frame: From baseline through last study visit (up to 30 days (+/- 2 days)) ]
    Unanticipated problems involving risk to human subjects



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Adults (≥18 years) with TCP as defined by BOTH (a) and (b):

    1. a count of between 10,000 and 70,000 platelets/μL blood, inclusive
    2. ANY ONE OR MORE of (1-3):

      1. confirmed diagnosis of hematologic malignancy, myeloproliferative disorder, myelodysplastic syndrome, or aplasia
      2. undergoing chemotherapy, immunotherapy, radiation therapy or hematopoietic stem cell t transplantation
      3. refractory to platelet transfusion defined as two 1-hour CCI of <5000 on consecutive transfusions of LSP (Sacher, 2003)
  2. WHO Bleeding Score of 2 excluding cutaneous bleeding (designated in this study as 2a)
  3. Able to provide informed consent directly or through legally authorized representative, and comply with treatment and monitoring
  4. Negative pregnancy test for women of childbearing potential

Exclusion Criteria:

  1. Any disorder or condition related to thrombosis, embolism, vascular occlusion, or ischemia (except when a prior history of central line thrombosis has resolved), including but not limited to: past history or current diagnosis of arterial or venous thromboembolic disease including acute coronary syndrome, peripheral vascular disease, and retinal arterial or venous thrombosis, MI, stent placement, valve replacement and/or repair, sinusoidal obstruction syndrome (veno-occlusive disease) or cytokine storm syndrome associated with CAR-T cell therapy
  2. Refusal to accept blood products
  3. Liver enzyme blood levels greater than 3× the upper limit of normal (ULN)
  4. Blood creatinine level greater than 3× ULN
  5. Received platelet inhibitor drugs, cyclooxygenase-2 (COX-2) inhibitors, or nonsteroidal anti-inflammatory drugs within 5 days prior to infusion
  6. Currently receiving anticoagulant therapy or antiplatelet therapy
  7. Receipt of any pro-coagulant agents other than TXA [e.g., DDAVP, recombinant Factor VIIa or prothrombin complex concentrates (PCC)] within 48 hours of first infusion, or with known hypercoagulable state
  8. WHO Bleeding Score of 2 solely due to cutaneous bleeding such as petechia, ecchymosis, or bruising
  9. Receiving L-asparaginase as part of a current cycle of treatment
  10. Known inherited or acquired bleeding disorder including, but not limited to: acquired storage pool deficiency or paraproteinemia with platelet inhibition
  11. Known inherited or acquired prothrombotic disorders, including antiphospholipid syndrome (Those with lupus anticoagulant or positive antiphospholipid serology without thrombosis are NOT excluded.)
  12. Anuria
  13. On dialysis
  14. Receipt of an investigational drug within 1 month before first infusion, other than for treatment of their underlying disease
  15. Females pregnant or nursing or unwilling to use contraception during and for 30 days after taking the study product (females). Evidence of effective birth control may be used, at the discretion of the physician
  16. Acute or chronic medical disorder that, in the opinion of the Investigator, would impair the ability of the patient to receive or respond to study treatment
  17. Prior participation in this study with successful infusion of the investigational or control product
  18. Currently enrolled in other trials not related to their primary disease process or involving platelet transfusions, platelet growth factors, or other pro-coagulant agents

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04631211


Contacts
Layout table for location contacts
Contact: Meaghan Dunn, MS 508-479-2769 meaghan.dunn@avaniaclinical.com

Sponsors and Collaborators
Cellphire, Inc.
Department of Health and Human Services
Investigators
Layout table for investigator information
Principal Investigator: Terry Gernsheimer, MD University of Washington
Study Director: Mike Fitzpatrick, PhD Cellphire, Inc.
  Study Documents (Full-Text)

Documents provided by Cellphire, Inc.:
Informed Consent Form  [PDF] August 20, 2020

Layout table for additonal information
Responsible Party: Cellphire, Inc.
ClinicalTrials.gov Identifier: NCT04631211    
Other Study ID Numbers: #2019-1
First Posted: November 17, 2020    Key Record Dates
Last Update Posted: January 20, 2021
Last Verified: January 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
Layout table for MeSH terms
Hematologic Neoplasms
Myelodysplastic Syndromes
Thrombocytopenia
Myeloproliferative Disorders
Anemia, Aplastic
Neoplasms
Bone Marrow Diseases
Hematologic Diseases
Blood Platelet Disorders
Neoplasms by Site
Anemia