ThrombocytopeniaPhase 1NCT03394755
Thrombosomes® in Bleeding Thrombocytopenic Patients
A Phase 1 study testing Thrombosomes in people with Thrombocytopenia and Hematologic Diseases, run by Cellphire Therapeutics, Inc. at 7 sites in 2 countries.
Trial summary
The study evaluates the safety and potential early signals of efficacy of allogeneic Thrombosomes in bleeding thrombocytopenic patients Read more about this study
- Who is it for?
- Ages 18 to 74 years
- Conditions the study is for
- Thrombocytopenia, Hematologic Diseases, Bone Marrow Aplasia
- What is being tested?
- Thrombosomes
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 7 sitesin 2 countries
- Study dates
- 19 Mar 2018to 25 Sep 2019
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 to 74 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
7 sites in 2 countries.
7 sites in 2 countries
United States6 sites
University of Michigan
Ann Arbor, Michigan, 48109
Hoxworth Blood Center/University of Cincinnati
Cincinnati, Ohio, 45219
City of Hope
Duarte, California, 91010
MD Anderson Cancer Center
Houston, Texas, 77092
Dartmouth Hitchcock Medical Center
Lebanon, New Hampshire, 03766
Georgetown University Hospital
Washington D.C., District of Columbia, 20016
About this study
Official title: A Phase I, Multi-Center, Open-Label, Dose Escalation Study of Thrombosomes® in Bleeding Thrombocytopenic Patients in Three Cohorts
The study evaluates the safety and potential early signals of efficacy of allogeneic Thrombosomes in bleeding thrombocytopenic patients
Read the detailed description
The primary objective of the present study was to assess the safety of increasing dose levels of Thrombosomes in bleeding patients with thrombocytopenia. The secondary objective was to explore early signals of clinical efficacy of Thrombosomes in this population. The secondary objectives included: 1) Evaluation of the impact on WHO (World Health Organization) bleeding scores at various timepoints; 2) number and type of blood products infused through day 6 follow-up period; and 3) post hoc analysis of hematology, coagulation, and chemistry.
- Conditions
- Thrombocytopenia, Hematologic Diseases, Bone Marrow Aplasia
- Being tested
- Thrombosomes
- Keywords
- thrombocytopenia, bleeding
- Medical subject headings
- Thrombocytopenia, Hematologic Diseases, Anemia, Aplastic, Hemorrhage
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ThrombosomesBiological
Freeze-dried platelets
| Study group | Role | What they receive |
|---|---|---|
| 9.45 x 10^7 Thrombosomes/kg | Experimental | Cohort 1 Receives: Biological: Thrombosomes |
| 1.89 x 10^8 Thrombosomes/kg | Experimental | Cohort 2 Receives: Biological: Thrombosomes |
| 3.78 x10^8 Thrombosomes/kg | Experimental | Cohort 3 Receives: Biological: Thrombosomes |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Patients With Treatment-Emergent Adverse Events (TEAE)
Overall frequency of (and number and percentage of patients who experience) TEAEs including serious adverse drug reactions and treatment-related events specifically defining the study's suspension and stopping rules (i.e., thromboembolic events, acute lung injury, anaphylaxis, and death).
Measured: 30 days
Number of Patients With Treatment-Emergent Serious Adverse Events (TESAE)
Overall frequency of (and number and percentage of patients who experience) TESAEs including serious adverse drug reactions and treatment-related events specifically defining the study's suspension and stopping rules (i.e., thromboembolic events, acute lung injury, anaphylaxis, and death).
Measured: 30 days
Secondary outcomes (6)
Number of WHO Bleeding Sites With Status Change From Baseline
Grade-level change in WHO bleeding assessment score at most severe bleeding site from baseline; WHO bleeding assessment score ranges from 1 to 4, with higher numbers indicating worse bleeding.
Measured: 1, 6, 24 hours, and Day 6 post infusion
Number of Patients With Grade-level Change in WHO Bleeding Assessment Score From Baseline - Patients WHO Score at Primary Bleeding Site
Patients WHO score at primary bleeding site. WHO bleeding assessment score ranges from 1 to 4, with higher numbers indicating worse bleeding. The maximum WHO bleeding assessment score for patients on study was 2.
Measured: Baseline, 1, 6, 24 hours, and Day 6 post infusion
Number of Patients With a Shift From Baseline in Hemoglobin
Shift from baseline clinical status of hemoglobin measure at different timepoints post infusion
Measured: 1, 6, 24 hours, Day 3, 4, 5, and 6 post infusion
Number of Patients With a Shift From Baseline in Hematocrit
Shift from baseline clinical status of hematocrit measure at different timepoints post infusion
Measured: 1, 6, 24 hours, Day 3, 4, 5, and 6 post infusion
Number or Patients With a Shift From Baseline in Coagulation Measures 24 Hours Post Infusion
Shift from baseline clinical status of coagulation measure at 24 hours post infusion for each cohort
Measured: 24 hours post infusion
Median Platelet Counts
Median Platelet Counts Per Time Point
Measured: Screening, Baseline, 1, 6, 24 hours, Day 3, 4, 5, and 6 post infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jan 2018
- Study start19 Mar 2018
- Primary completion1 Aug 2019
- Study completion25 Sep 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for ThrombocytopeniaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cellphire Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Department of Health and Human Services
- Responsible party
- Sponsor
- Investigators
- Michael Fitzpatrick, Study director, Cellphire Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03394755
- Sponsor's protocol ID
- 2017-1
- Status last verified
- Apr 2023
- First submitted
- 3 Jan 2018
- Last updated
- 14 Apr 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Ohanian M, Cancelas JA, Davenport R, Pullarkat V, Hervig T, Broome C, Marek K, Kelly M, Gul Z, Rugg N, Nestheide S, Kinne B, Szczepiorkowski Z, Kantarjian H, Pehta J, Biehl R, Yu A, Aung F, Antebi B, Fitzpatrick GM. Freeze-dried platelets are a promising alternative in bleeding thrombocytopenic patients with hematological malignancies. Am J Hematol. 2022 Mar 1;97(3):256-266. doi: 10.1002/ajh.26403. Epub 2021 Dec 23.
PubMed 34748664
Study at a glance
From the registry record, last updated by the sponsor on 14 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I, Multi-Center, Open-Label, Dose Escalation Study of Thrombosomes® in Bleeding Thrombocytopenic Patients in Three Cohorts
- ClinicalTrials.gov ID
- NCT03394755
- Acronym / sponsor ID
- 2017-1
- Conditions
- Thrombocytopenia, Hematologic Diseases, Bone Marrow Aplasia
- Keywords
- thrombocytopenia, bleeding
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Cellphire Therapeutics, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Department of Health and Human Services
- Study start
- 19 Mar 2018 (actual)
- Primary completion
- 1 Aug 2019 (actual)
- Study completion
- 25 Sep 2019 (actual)
- First posted
- 9 Jan 2018 (actual)
- Results
- Posted 14 Apr 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study evaluates the safety and potential early signals of efficacy of allogeneic Thrombosomes in bleeding thrombocytopenic patients
Detailed description
The primary objective of the present study was to assess the safety of increasing dose levels of Thrombosomes in bleeding patients with thrombocytopenia. The secondary objective was to explore early signals of clinical efficacy of Thrombosomes in this population. The secondary objectives included: 1) Evaluation of the impact on WHO (World Health Organization) bleeding scores at various timepoints; 2) number and type of blood products infused through day 6 follow-up period; and 3) post hoc analysis of hematology, coagulation, and chemistry.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| 9.45 x 10^7 Thrombosomes/kg | Experimental | Biological: ThrombosomesCohort 1 |
| 1.89 x 10^8 Thrombosomes/kg | Experimental | Biological: ThrombosomesCohort 2 |
| 3.78 x10^8 Thrombosomes/kg | Experimental | Biological: ThrombosomesCohort 3 |
Who may qualify
- Ages
- 18 to 74 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ThrombosomesFreeze-dried platelets | Biological | 1.89 x 10^8 Thrombosomes/kg, 3.78 x10^8 Thrombosomes/kg, 9.45 x 10^7 Thrombosomes/kg |
Primary outcome
Number of Patients With Treatment-Emergent Adverse Events (TEAE)
Time frame: 30 days
Overall frequency of (and number and percentage of patients who experience) TEAEs including serious adverse drug reactions and treatment-related events specifically defining the study's suspension and stopping rules (i.e., thromboembolic events, acute lung injury, anaphylaxis, and death).
Number of Patients With Treatment-Emergent Serious Adverse Events (TESAE)
Time frame: 30 days
Overall frequency of (and number and percentage of patients who experience) TESAEs including serious adverse drug reactions and treatment-related events specifically defining the study's suspension and stopping rules (i.e., thromboembolic events, acute lung injury, anaphylaxis, and death).
6 secondary outcomes
Number of WHO Bleeding Sites With Status Change From Baseline
Time frame: 1, 6, 24 hours, and Day 6 post infusion
Number of Patients With Grade-level Change in WHO Bleeding Assessment Score From Baseline - Patients WHO Score at Primary Bleeding Site
Time frame: Baseline, 1, 6, 24 hours, and Day 6 post infusion
Number of Patients With a Shift From Baseline in Hemoglobin
Time frame: 1, 6, 24 hours, Day 3, 4, 5, and 6 post infusion
Number of Patients With a Shift From Baseline in Hematocrit
Time frame: 1, 6, 24 hours, Day 3, 4, 5, and 6 post infusion
Number or Patients With a Shift From Baseline in Coagulation Measures 24 Hours Post Infusion
Time frame: 24 hours post infusion
Median Platelet Counts
Time frame: Screening, Baseline, 1, 6, 24 hours, Day 3, 4, 5, and 6 post infusion
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Apr 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 34748664Ohanian M, Cancelas JA, Davenport R, Pullarkat V, Hervig T, Broome C, Marek K, Kelly M, Gul Z, Rugg N, Nestheide S, Kinne B, Szczepiorkowski Z, Kantarjian H, Pehta J, Biehl R, Yu A, Aung F, Antebi B, Fitzpatrick GM. Freeze-dried platelets are a promising alternative in bleeding thrombocytopenic patients with hematological malignancies. Am J Hematol. 2022 Mar 1;97(3):256-266. doi: 10.1002/ajh.26403. Epub 2021 Dec 23.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Michael Fitzpatrick study director
Cellphire Therapeutics, Inc.
7 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Thrombosomes in people with Thrombocytopenia and Hematologic Diseases, run by Cellphire Therapeutics, Inc. at 7 sites in 2 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 3 inclusion and 6 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: finished, as of the sponsor's last update on 14 Apr 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States, 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 14 Apr 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Number of Patients With Treatment-Emergent Adverse Events (TEAE) (time frame: 30 days). The study lists 2 primary outcomes in all. 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 NCT03394755, which also lists 1 linked publication.
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 14 Apr 2023.