Phase 1 Safety Study of Dimethyl Sulfoxide Cryopreserved Platelets
Dose Escalation Study Evaluating the Safety of Dimethyl Sulfoxide Cryopreserved Platelets Compared With Liquid Stored Platelets in Patients With Uncontrolled Bleeding
2 other identifiers
interventional
28
1 country
4
Brief 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) and by evaluating coagulation-related parameters to assess the evidence of any thrombotic events after CPP or LSP transfusion.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_1
Started Nov 2014
4 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
February 26, 2014
CompletedFirst Posted
Study publicly available on registry
March 5, 2014
CompletedStudy Start
First participant enrolled
November 5, 2014
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 1, 2016
CompletedStudy Completion
Last participant's last visit for all outcomes
July 17, 2016
CompletedResults Posted
Study results publicly available
May 20, 2021
CompletedMay 20, 2021
May 1, 2021
1.7 years
February 26, 2014
September 25, 2018
May 19, 2021
Conditions
Keywords
Outcome Measures
Primary Outcomes (3)
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.
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.
day of thru 6 days after transfusion
Number of Subject With Thrombotic Events
Subjects with any signs or symptoms of thrombotic events.
6 days after transfusion
Other Outcomes (2)
Corrected Count Increments (CCI)
Day 1 up to 6 hrs after transfusion and Day 2 approx 24 hrs after transfusion
Count Increment
On Day 1 up to 6 hours after transfusion and on Day 2 approximately 24 hours after transfusion
Study Arms (8)
Cohort 1 with 0.5 units of CPP
EXPERIMENTALA 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 COMPARATORA single 30 to 60 minute intravenous (IV) infusion of 1 unit of liquid stored platelets (LSP)
Cohort 2 with 1 unit of CPP
EXPERIMENTALA 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 COMPARATORA single 30 to 60 minute intravenous (IV) infusion of 1 unit of liquid stored platelets (LSP)
Cohort 3 with 2 units of CPP
EXPERIMENTALA 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 COMPARATORA single 30 to 60 minute intravenous (IV) infusion of 1 unit of liquid stored platelets (LSP)
Cohort 4 with 3 units of CPP
EXPERIMENTALA 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 COMPARATORA single 30 to 60 minute intravenous (IV) infusion of 1 unit of liquid stored platelets (LSP
Interventions
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.
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (4)
Dartmouth-Hitchcock Medical Center
Lebanon, New Hampshire, 03756, United States
Hoxworth Blood Center
Cincinnati, Ohio, 45267, United States
Puget Sound Blood Center
Seattle, Washington, 98104, United States
University of Washington, Division of Hematology
Seattle, Washington, 98195, United States
Related Publications (1)
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.
PMID: 30204953DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Sherrill Slichter
- Organization
- Pugent Sound Blood Center
Study Officials
- 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
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- No
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- FED
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 26, 2014
First Posted
March 5, 2014
Study Start
November 5, 2014
Primary Completion
July 1, 2016
Study Completion
July 17, 2016
Last Updated
May 20, 2021
Results First Posted
May 20, 2021
Record last verified: 2021-05