- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02078284
Phase 1 Safety Study of Dimethyl Sulfoxide Cryopreserved Platelets
May 19, 2021 updated by: U.S. Army Medical Research and Development Command
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.
Study Overview
Status
Completed
Conditions
Intervention / Treatment
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 Type
Interventional
Enrollment (Actual)
28
Phase
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
-
New Hampshire
-
Lebanon, New Hampshire, United States, 03756
- Dartmouth-Hitchcock Medical Center
-
-
Ohio
-
Cincinnati, Ohio, United States, 45267
- Hoxworth Blood Center
-
-
Washington
-
Seattle, Washington, United States, 98104
- Puget Sound Blood Center
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Seattle, Washington, United States, 98195
- University of Washington, Division of Hematology
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years and older (Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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.
- 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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Cohort 1 with 0.5 units of CPP
A single 30 to 60 minute intravenous (IV) infusion of 0.5 unit of 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.
Other Names:
|
Active Comparator: Cohort 1 with 1 unit of LSP
A single 30 to 60 minute intravenous (IV) infusion of 1 unit of liquid stored platelets (LSP)
|
Other Names:
|
Experimental: Cohort 2 with 1 unit of CPP
A single 30 to 60 minute intravenous (IV) infusion of 1 unit of 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.
Other Names:
|
Active Comparator: Cohort 2 with 1 unit of LSP
A single 30 to 60 minute intravenous (IV) infusion of 1 unit of liquid stored platelets (LSP)
|
Other Names:
|
Experimental: Cohort 3 with 2 units of CPP
A single 30 to 60 minute intravenous (IV) infusion of 2 units of 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.
Other Names:
|
Active Comparator: Cohort 3 with 1 unit of LSP
A single 30 to 60 minute intravenous (IV) infusion of 1 unit of liquid stored platelets (LSP)
|
Other Names:
|
Experimental: Cohort 4 with 3 units of CPP
A single 30 to 60 minute intravenous (IV) infusion of 3 units of 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.
Other Names:
|
Active Comparator: Cohort 4 with 1 unit of LSP
A single 30 to 60 minute intravenous (IV) infusion of 1 unit of liquid stored platelets (LSP
|
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
|
day of thru 6 days after transfusion
|
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.
|
day of thru 6 days after transfusion
|
Number of Subject With Thrombotic Events
Time Frame: 6 days after transfusion
|
Subjects with any signs or symptoms of thrombotic events.
|
6 days after transfusion
|
Other Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Corrected Count Increments (CCI)
Time Frame: Day 1 up to 6 hrs after transfusion and Day 2 approx 24 hrs after transfusion
|
Corrected Count Increments Expressed in units of mm^2/(µL*10¹¹ platelets) (CCI) in the 6 hour period after the study platelet transfusion and on Day 2 (approximately 24 hours after the study platelet transfusion)
|
Day 1 up to 6 hrs after transfusion and Day 2 approx 24 hrs after transfusion
|
Count Increment
Time Frame: On Day 1 up to 6 hours after transfusion and on Day 2 approximately 24 hours after transfusion
|
Count Increment expressed in units of platelets (x10^3 µL) (CI)
|
On Day 1 up to 6 hours after transfusion and on Day 2 approximately 24 hours after transfusion
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Investigators
- Principal Investigator: Sherrill J Slichter, MD, Bloodworks
- Principal Investigator: Terry B Gernsheimer, MD, University of Washington, Division of Hematology
- Principal Investigator: Zbigniew Szczepiorkowski, MD, PhD, Center for Transfusion Medicine Research, Lebanon, NH
- Principal Investigator: Jose A Cancelas-Perez, MD, PhD, Hoxworth Blood Center
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
November 5, 2014
Primary Completion (Actual)
July 1, 2016
Study Completion (Actual)
July 17, 2016
Study Registration Dates
First Submitted
February 26, 2014
First Submitted That Met QC Criteria
February 28, 2014
First Posted (Estimate)
March 5, 2014
Study Record Updates
Last Update Posted (Actual)
May 20, 2021
Last Update Submitted That Met QC Criteria
May 19, 2021
Last Verified
May 1, 2021
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- S-12-04
- CPP1-05 (Other Identifier: Army)
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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