ThrombocytopeniaPhase 1NCT02078284
Phase 1 Safety Study of Dimethyl Sulfoxide Cryopreserved Platelets
A Phase 1 study testing CPP and LSP in people with Thrombocytopenia, run by U.S. Army Medical Research and Development Command at 4 sites in United States.
Trial summary
This study is to evaluate the safety of intravenous (IV) infusion of dimethyl sulfoxide (DMSO) cryopreserved platelets (CPP) in participants with World Health Organization (WHO) Grade 2 bleed in spite of receiving a transfusion of liquid stored platelets (LSP) in the past 48 hours by collecting adverse events (AEs)… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Thrombocytopenia
- What is being tested?
- CPP and LSP
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 28 participants
- Where
- 4 sitesUnited States
- Study dates
- 5 Nov 2014to 17 Jul 2016
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female, at least 18 years of age.
- Ability to comprehend the study procedures and signed informed consent.
- If pre-menopausal female, must have a negative serum pregnancy test, and, if of child bearing potential, must be using an acceptable method of contraception.
- Diagnosed with any the following: acute leukemia (ALL or AML), chronic leukemia (CML, CLL, CMML, or hairy cell leukemia), myelodysplasia, aplasia, hematopoietic or non-hematopoietic solid tumor, or therapy (chemotherapy or radiation) induced bone marrow aplasia or hypoplasia. Either autologous or allogeneic bone marrow transplant or peripheral or cord blood stem cell recipients may be enrolled.
- WHO grade 2 or greater bleeding.
Who cannot join
- Acute or chronic DIC as evidence by D-dimer greater than 8 μg/mL and fibrinogen less than 100 mg (0.1 g)/dL. Both criteria must be met. If data are in the medical record for fibrin degradation products (FDPs), then FDP must be <=40 μg/mL (FDP >40 μg/mL is indicative of DIC).
- PT or aPTT > 1.3 times the upper limit of normal for the laboratory.
- History of major operative procedures that required general anesthesia in the past 2 weeks.
- History of any prior major unprovoked thrombotic events and/or known inherited disorder of coagulation or platelet function (by history) (not to include clots in catheters, etc).
- A history or diagnosis of immune thrombocytopenia, thrombotic thrombocytopenic purpura, or hemolytic uremic syndrome.
- Females who are breastfeeding.
Show all 9Show fewer
- Veno-occlusive disease or possible veno-occlusive disease.
- Receiving active, inpatient treatment with anti-platelet drugs and/or full anticoagulation therapy. Note: a heparin flush may be given daily and before and after blood draws to patients with a central line to keep the line patent.
- Subject previously enrolled in this study and received a study transfusion.
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Hoxworth Blood Center
Cincinnati, Ohio, 45267
Dartmouth-Hitchcock Medical Center
Lebanon, New Hampshire, 03756
Puget Sound Blood Center
Seattle, Washington, 98104
University of Washington, Division of Hematology
Seattle, Washington, 98195
About this study
Official title: Dose Escalation Study Evaluating the Safety of Dimethyl Sulfoxide Cryopreserved Platelets Compared With Liquid Stored Platelets in Patients With Uncontrolled Bleeding
This study is to evaluate the safety of intravenous (IV) infusion of dimethyl sulfoxide (DMSO) cryopreserved platelets (CPP) in participants with World Health Organization (WHO) Grade 2 bleed in spite of receiving a transfusion of liquid stored platelets (LSP) in the past 48 hours by collecting adverse events (AEs) and by evaluating coagulation-related parameters to assess the evidence of any thrombotic events after CPP or LSP transfusion.
Read the detailed description
After determining eligibility for study participation, 4 sequential cohorts of subjects will receive escalating doses of CPP (Test) or 1 unit of LSP (Control) randomized 6:1 within each cohort. Each sequential cohort will receive the following transfusions starting with the first cohort: 0.5, 1, 2, and 3 units of CPP with an additional single subject in each cohort who will receive 1 unit of LSP for a total of 28 subjects. Assignment to Test and Control platelets for subjects in each cohort will be centrally randomized using an interactive web response system (IWRS). Following the study transfusion, subjects are followed for evidence of transfusion reactions, thrombotic events, other AEs, coagulation-related parameters, and platelet efficacy endpoints. CPP or LSP will be transfused into a patient according to the randomized product and dose assignment within the cohort. Following the transfusion, subjects are followed for the remainder of Study Day 1 and on Study Day 2 for AEs and tested for coagulation markers including fibrinogen, D-dimer, prothrombin fragments 1 + 2 (F 1+2), thrombin antithrombin (TAT), anti-thrombin (AT), prothrombin time (PT), activated partial thromboplastin time (aPTT), thrombin generation testing (TGT) results, thromboelastography (TEG) results, and other potential complications of platelet transfusion such as transfusion reactions and thrombotic events including assessment of vital signs (blood pressure, heart rate, respiration rate, and pulse oximetry). A subject is considered to have completed the study for safety evaluation for dose escalation, if he/she receives a study infusion and completed study-related Day 3 procedures.
- Conditions
- Thrombocytopenia
- Being tested
- CPP, LSP
- Keywords
- platelet transfusion
- Medical subject headings
- Thrombocytopenia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CPPBiological
Also called dimethyl sulfoxide (DMSO) cryopreserved platelets (CPP)
One unit of frozen CPP contains approximately 3.4 x 10^11 irradiated platelets and approximately 6% DMSO in sterile 0.9% sodium chloride solution (total volume of 20 mL to 35 mL) stored frozen at ≤ -65°C.
LSPBiological
Also called liquid stored platelets (LSP)
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 with 0.5 units of CPP | Experimental | A single 30 to 60 minute intravenous (IV) infusion of 0.5 unit of dimethyl sulfoxide (DMSO) cryopreserved platelets (CPP) Receives: Biological: CPP |
| Cohort 1 with 1 unit of LSP | Active comparator | A single 30 to 60 minute intravenous (IV) infusion of 1 unit of liquid stored platelets (LSP) Receives: Biological: LSP |
| Cohort 2 with 1 unit of CPP | Experimental | A single 30 to 60 minute intravenous (IV) infusion of 1 unit of dimethyl sulfoxide (DMSO) cryopreserved platelets (CPP) Receives: Biological: CPP |
| Cohort 2 with 1 unit of LSP | Active comparator | A single 30 to 60 minute intravenous (IV) infusion of 1 unit of liquid stored platelets (LSP) Receives: Biological: LSP |
| Cohort 3 with 2 units of CPP | Experimental | A single 30 to 60 minute intravenous (IV) infusion of 2 units of dimethyl sulfoxide (DMSO) cryopreserved platelets (CPP) Receives: Biological: CPP |
| Cohort 3 with 1 unit of LSP | Active comparator | A single 30 to 60 minute intravenous (IV) infusion of 1 unit of liquid stored platelets (LSP) Receives: Biological: LSP |
| Cohort 4 with 3 units of CPP | Experimental | A single 30 to 60 minute intravenous (IV) infusion of 3 units of dimethyl sulfoxide (DMSO) cryopreserved platelets (CPP) Receives: Biological: CPP |
| Cohort 4 with 1 unit of LSP | Active comparator | A single 30 to 60 minute intravenous (IV) infusion of 1 unit of liquid stored platelets (LSP Receives: Biological: LSP |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events (AEs) by Level of Severity
Clinical laboratory [chemistry (serum creatinine, lactate dehydrogenase (LDH), and troponin, and hematology (hemoglobin and hematocrit)] parameters, physical examination findings, electrocardiogram (ECG)] and vital sign AEs summarized by severity.
Measured: day of thru 6 days after transfusion
Number of Subject Who Experienced Adverse Events (AEs) for a Specific Severity
Number of subjects who experienced AEs at specific levels of severity. Clinical laboratory [chemistry (serum creatinine, lactate dehydrogenase (LDH), and troponin, and hematology (hemoglobin and hematocrit)] parameters, physical examination findings, electrocardiogram (ECG)] and vital signs were evaluated.
Measured: day of thru 6 days after transfusion
Number of Subject With Thrombotic Events
Subjects with any signs or symptoms of thrombotic events.
Measured: 6 days after transfusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Mar 2014 (estimated)
- Study start5 Nov 2014
- Primary completionJul 2016
- Study completion17 Jul 2016
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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 28 participants (actual)
Sponsor and investigators
- Lead sponsor
- U.S. Army Medical Research and Development Command
- Sponsor type
- US federal agency
- Responsible party
- Sponsor
- Investigators
- Sherrill J Slichter, MD, Principal investigator, BloodworksTerry B Gernsheimer, MD, Principal investigator, University of Washington, Division of HematologyZbigniew Szczepiorkowski, MD, PhD, Principal investigator, Center for Transfusion Medicine Research, Lebanon, NHJose A Cancelas-Perez, MD, PhD, Principal investigator, Hoxworth Blood Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02078284
- Sponsor's protocol ID
- S-12-04
- Other IDs
- CPP1-05
- Status last verified
- May 2021
- First submitted
- 26 Feb 2014
- Last updated
- 20 May 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Slichter SJ, Dumont LJ, Cancelas JA, Jones M, Gernsheimer TB, Szczepiorkowski ZM, Dunbar NM, Prakash G, Medlin S, Rugg N, Kinne B, Macdonald VW, Housler G, Valiyaveettil M, Hmel P, Ransom JH. Safety and efficacy of cryopreserved platelets in bleeding patients with thrombocytopenia. Transfusion. 2018 Sep;58(9):2129-2138. doi: 10.1111/trf.14780.
PubMed 30204953
Study at a glance
From the registry record, last updated by the sponsor on 20 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Dose Escalation Study Evaluating the Safety of Dimethyl Sulfoxide Cryopreserved Platelets Compared With Liquid Stored Platelets in Patients With Uncontrolled Bleeding
- ClinicalTrials.gov ID
- NCT02078284
- Acronym / sponsor ID
- S-12-04
- Conditions
- Thrombocytopenia
- Keywords
- platelet transfusion
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 28 participants (actual)
- Lead sponsor
- U.S. Army Medical Research and Development CommandUS federal agency
- Study start
- 5 Nov 2014 (actual)
- Primary completion
- Jul 2016 (actual)
- Study completion
- 17 Jul 2016 (actual)
- First posted
- 5 Mar 2014 (estimated)
- Results
- Posted 20 May 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is to evaluate the safety of intravenous (IV) infusion of dimethyl sulfoxide (DMSO) cryopreserved platelets (CPP) in participants with World Health Organization (WHO) Grade 2 bleed in spite of receiving a transfusion of liquid stored platelets (LSP) in the past 48 hours by collecting adverse events (AEs) and by evaluating coagulation-related parameters to assess the evidence of any thrombotic events after CPP or LSP transfusion.
Detailed description
After determining eligibility for study participation, 4 sequential cohorts of subjects will receive escalating doses of CPP (Test) or 1 unit of LSP (Control) randomized 6:1 within each cohort. Each sequential cohort will receive the following transfusions starting with the first cohort: 0.5, 1, 2, and 3 units of CPP with an additional single subject in each cohort who will receive 1 unit of LSP for a total of 28 subjects. Assignment to Test and Control platelets for subjects in each cohort will be centrally randomized using an interactive web response system (IWRS). Following the study transfusion, subjects are followed for evidence of transfusion reactions, thrombotic events, other AEs, coagulation-related parameters, and platelet efficacy endpoints. CPP or LSP will be transfused into a patient according to the randomized product and dose assignment within the cohort. Following the transfusion, subjects are followed for the remainder of Study Day 1 and on Study Day 2 for AEs and tested for coagulation markers including fibrinogen, D-dimer, prothrombin fragments 1 + 2 (F 1+2), thrombin antithrombin (TAT), anti-thrombin (AT), prothrombin time (PT), activated partial thromboplastin time (aPTT), thrombin generation testing (TGT) results, thromboelastography (TEG) results, and other potential complications of platelet transfusion such as transfusion reactions and thrombotic events including assessment of vital signs (blood pressure, heart rate, respiration rate, and pulse oximetry). A subject is considered to have completed the study for safety evaluation for dose escalation, if he/she receives a study infusion and completed study-related Day 3 procedures.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 8
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 with 0.5 units of CPP | Experimental | Biological: CPPA single 30 to 60 minute intravenous (IV) infusion of 0.5 unit of dimethyl sulfoxide (DMSO) cryopreserved platelets (CPP) |
| Cohort 1 with 1 unit of LSP | Active comparator | Biological: LSPA single 30 to 60 minute intravenous (IV) infusion of 1 unit of liquid stored platelets (LSP) |
| Cohort 2 with 1 unit of CPP | Experimental | Biological: CPPA single 30 to 60 minute intravenous (IV) infusion of 1 unit of dimethyl sulfoxide (DMSO) cryopreserved platelets (CPP) |
| Cohort 2 with 1 unit of LSP | Active comparator | Biological: LSPA single 30 to 60 minute intravenous (IV) infusion of 1 unit of liquid stored platelets (LSP) |
| Cohort 3 with 2 units of CPP | Experimental | Biological: CPPA single 30 to 60 minute intravenous (IV) infusion of 2 units of dimethyl sulfoxide (DMSO) cryopreserved platelets (CPP) |
| Cohort 3 with 1 unit of LSP | Active comparator | Biological: LSPA single 30 to 60 minute intravenous (IV) infusion of 1 unit of liquid stored platelets (LSP) |
| Cohort 4 with 3 units of CPP | Experimental | Biological: CPPA single 30 to 60 minute intravenous (IV) infusion of 3 units of dimethyl sulfoxide (DMSO) cryopreserved platelets (CPP) |
| Cohort 4 with 1 unit of LSP | Active comparator | Biological: LSPA single 30 to 60 minute intravenous (IV) infusion of 1 unit of liquid stored platelets (LSP |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Male or female, at least 18 years of age.
- Ability to comprehend the study procedures and signed informed consent.
- If pre-menopausal female, must have a negative serum pregnancy test, and, if of child bearing potential, must be using an acceptable method of contraception.
- Diagnosed with any the following: acute leukemia (ALL or AML), chronic leukemia (CML, CLL, CMML, or hairy cell leukemia), myelodysplasia, aplasia, hematopoietic or non-hematopoietic solid tumor, or therapy (chemotherapy or radiation) induced bone marrow aplasia or hypoplasia. Either autologous or allogeneic bone marrow transplant or peripheral or cord blood stem cell recipients may be enrolled.
- WHO grade 2 or greater bleeding.
Key exclusion criteria (9 listed)
- Acute or chronic DIC as evidence by D-dimer greater than 8 μg/mL and fibrinogen less than 100 mg (0.1 g)/dL. Both criteria must be met. If data are in the medical record for fibrin degradation products (FDPs), then FDP must be <=40 μg/mL (FDP >40 μg/mL is indicative of DIC).
- PT or aPTT > 1.3 times the upper limit of normal for the laboratory.
- History of major operative procedures that required general anesthesia in the past 2 weeks.
- History of any prior major unprovoked thrombotic events and/or known inherited disorder of coagulation or platelet function (by history) (not to include clots in catheters, etc).
- A history or diagnosis of immune thrombocytopenia, thrombotic thrombocytopenic purpura, or hemolytic uremic syndrome.
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 |
|---|---|---|
| CPPAlso: dimethyl sulfoxide (DMSO) cryopreserved platelets (CPP)One unit of frozen CPP contains approximately 3.4 x 10^11 irradiated platelets and approximately 6% DMSO in sterile 0.9% sodium chloride solution (total volume of 20 mL to 35 mL) stored frozen at ≤ -65°C. | Biological | Cohort 1 with 0.5 units of CPP, Cohort 2 with 1 unit of CPP, Cohort 3 with 2 units of CPP, Cohort 4 with 3 units of CPP |
| LSPAlso: liquid stored platelets (LSP) | Biological | Cohort 1 with 1 unit of LSP, Cohort 2 with 1 unit of LSP, Cohort 3 with 1 unit of LSP, Cohort 4 with 1 unit of LSP |
Primary outcome
Adverse Events (AEs) by Level of Severity
Time frame: day of thru 6 days after transfusion
Clinical laboratory [chemistry (serum creatinine, lactate dehydrogenase (LDH), and troponin, and hematology (hemoglobin and hematocrit)] parameters, physical examination findings, electrocardiogram (ECG)] and vital sign AEs summarized by severity.
Number of Subject Who Experienced Adverse Events (AEs) for a Specific Severity
Time frame: day of thru 6 days after transfusion
Number of subjects who experienced AEs at specific levels of severity. Clinical laboratory [chemistry (serum creatinine, lactate dehydrogenase (LDH), and troponin, and hematology (hemoglobin and hematocrit)] parameters, physical examination findings, electrocardiogram (ECG)] and vital signs were evaluated.
Number of Subject With Thrombotic Events
Time frame: 6 days after transfusion
Subjects with any signs or symptoms of thrombotic events.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 May 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 30204953Slichter SJ, Dumont LJ, Cancelas JA, Jones M, Gernsheimer TB, Szczepiorkowski ZM, Dunbar NM, Prakash G, Medlin S, Rugg N, Kinne B, Macdonald VW, Housler G, Valiyaveettil M, Hmel P, Ransom JH. Safety and efficacy of cryopreserved platelets in bleeding patients with thrombocytopenia. Transfusion. 2018 Sep;58(9):2129-2138. doi: 10.1111/trf.14780.
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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.
Sherrill J Slichter, MD principal investigator
Bloodworks
Terry B Gernsheimer, MD principal investigator
University of Washington, Division of Hematology
Zbigniew Szczepiorkowski, MD, PhD principal investigator
Center for Transfusion Medicine Research, Lebanon, NH
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing CPP and LSP in people with Thrombocytopenia, run by U.S. Army Medical Research and Development Command at 4 sites in United States.
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 5 inclusion and 9 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 20 May 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. 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 20 May 2021). 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?
Adverse Events (AEs) by Level of Severity (time frame: day of thru 6 days after transfusion). The study lists 3 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 NCT02078284, 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 20 May 2021.