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ThrombocytopeniaPhase 2NCT04631211

Thrombosomes® in Bleeding Thrombocytopenic Patients Study

A Phase 2 study testing Thrombosomes and Liquid Stored Platelets (LSP) in people with Thrombocytopenia and Hematologic Malignancy, run by Cellphire Therapeutics, Inc. at 6 sites in 3 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial 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… Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Thrombosomes and Liquid Stored Platelets (LSP)
Could I receive a placebo?
No, everyone receives an active treatment
How many people
21 participants
Where
6 sitesin 3 countries
Study dates
5 Mar 2021to 7 Jan 2022
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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)
Show all 9Show fewer
  • 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

Who cannot join

  • 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)
Show all 21Show fewer
  • 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
  • On dialysis
  • Receipt of an investigational drug within 1 month before first infusion, other than for treatment of their underlying disease
  • 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
  • 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
  • Prior participation in this study with successful infusion of the investigational or control product
  • Currently enrolled in other trials not related to their primary disease process or involving platelet transfusions, platelet growth factors, or other pro-coagulant agents

Trial locations and contacts

6 sites in 3 countries.

6 sites in 3 countries

United States4 sites

  • City of Hope

    Duarte, California, 91010

  • MD Anderson Cancer Center

    Houston, Texas, 77030

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232

  • Medstar Georgetown

    Washington D.C., District of Columbia, 20007

Other trials in United States

Israel1 site

  • Rambam Medical Center

    Haifa

Other trials in Israel

Norway1 site

  • Helse Bergen Haukeland University Hospital

    Bergen

Other trials in Norway

About this study

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

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.

Being tested
Thrombosomes, Liquid Stored Platelets (LSP)
Medical subject headings
Thrombocytopenia, Hematologic Neoplasms, Anemia, Aplastic, Myeloproliferative Disorders, Myelodysplastic Syndromes

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ThrombosomesBiological

    Human platelet derived lyophilized hemostatic

  • Liquid Stored Platelets (LSP)Biological

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

Study groups in NCT04631211
Study groupRoleWhat they receive
Thrombosomes Low DoseExperimentalNot described
Receives: Biological: Thrombosomes
Thrombosomes Medium DoseExperimentalNot described
Receives: Biological: Thrombosomes
Thrombosomes High DoseExperimentalNot described
Receives: Biological: Thrombosomes
Liquid Stored Platelets (Control)Active comparatorNot described
Receives: Biological: Liquid Stored Platelets (LSP)

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

    Measured: Evaluated at 24 hours post initial infusion

Secondary outcomes (9)

  1. Secondary Efficacy Endpoint assessed by Number of days alive and without WHO (World Health Organization) Grade 2a or greater bleeding

    Number of days alive and without WHO (World Health Organization) Grade 2 or greater bleeding through initial 7 days after first Thrombosomes or LSP Infusion

    Measured: 7 days after first Thrombosomes or LSP infusion

  2. Secondary Efficacy Endpoint assessed by 30 day mortality

    30-day mortality post first infusion of Thrombosomes or post first infusion of LSP as control

    Measured: 30 days post first infusion (+/- 2 days)

  3. Secondary Efficacy Endpoint assessed by cessation or decrease in bleeding, as evidenced by ordinal change in WHO (World Health Organization) Bleeding Score

    Cessation or decrease in primary bleeding site, as evidenced by ordinal change in WHO (World Health Organization) Bleeding Score at 24, 48, 72 hours, Day 4, Day 5, Day 6 and Day 7 after first infusion of Thrombosomes or LSP infusion as control.

    Measured: 24, 48, 72 hours, Day 4, Day 5, Day 6 and Day 7 post first infusion

  4. Secondary Efficacy Endpoint assessed by cessation or decrease in bleeding, as evidenced by ordinal change in WHO (World Health Organization) Bleeding Score

    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 24, 48, 72 hours, Day 4, Day 5, Day 6 and Day 7 after first infusion of Thrombosomes or LSP infusion as control.

    Measured: 24, 48, 72 hours, Day 4, Day 5, Day 6 and Day 7 post first infusion

  5. Secondary Efficacy Endpoint assessed for Number, timing, type and reason for administration of all blood products

    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

    Measured: 7 days after first Thrombosomes or LSP infusion

  6. Secondary Efficacy Endpoint assessed by platelet count

    Platelet count measured at 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.

    Measured: 24, 48, 72 hours and Day 7 post first infusion

  7. Secondary Efficacy Endpoint assessed by measures of hematology

    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

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

  8. Secondary Efficacy Endpoint assessed by measures of coagulation

    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

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

  9. Secondary Efficacy Endpoint assessed by changes in markers of endothelial cell injury/repair

    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.

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

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted17 Nov 2020
  2. Study start5 Mar 2021
  3. Primary completion7 Jan 2022
  4. Study completion7 Jan 2022

Why it stopped: This study was terminated due to loss of funding for this indication from the Biomedical Advanced Research and Development Authority (BARDA) in November 2022.

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
Dose Ranging Multidose
Masking
None (open label)
Enrolment
21 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04631211
Sponsor's protocol ID
#2019-1
Status last verified
May 2023
First submitted
5 Nov 2020
Last updated
25 May 2023
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No
Expanded access
Available

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Thrombosomes and Liquid Stored Platelets (LSP) in people with Thrombocytopenia and Hematologic Malignancy, run by Cellphire Therapeutics, Inc. at 6 sites in 3 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 9 inclusion and 21 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 25 May 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 6 sites in United States, Israel, Norway. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early (reason given: This study was terminated due to loss of funding for this indication from the Biomedical Advanced Research and Development Authority (BARDA) in November 2022.). Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Primary Efficacy Endpoint (time frame: Evaluated at 24 hours post initial infusion). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04631211.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 May 2023.

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