Phase 1 Safety Study of Dimethyl Sulfoxide Cryopreserved Platelets (CPP1-05)

This study is not yet open for participant recruitment. (see Contacts and Locations)
Verified February 2014 by U.S. Army Medical Research and Materiel Command
Sponsor:
Information provided by (Responsible Party):
U.S. Army Medical Research and Materiel Command
ClinicalTrials.gov Identifier:
NCT02078284
First received: February 26, 2014
Last updated: February 28, 2014
Last verified: February 2014
  Purpose

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.

Descriptive statistics will be used to present the study data.


Condition Intervention Phase
Thrombocytopenia
Biological: CPP
Biological: LSP
Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Dose Escalation Study Evaluating the Safety of Dimethyl Sulfoxide Cryopreserved Platelets Compared With Liquid Stored Platelets in Patients With Uncontrolled Bleeding Not Responding to Standard Platelet Therapy

Resource links provided by NLM:


Further study details as provided by U.S. Army Medical Research and Materiel Command:

Primary Outcome Measures:
  • Adverse events [ Time Frame: Up to 6 days after transfusion ] [ Designated as safety issue: Yes ]
    Number and percentage of adverse events

  • Clinical laboratory findings [ Time Frame: Up to 6 days after transfusion ] [ Designated as safety issue: Yes ]
    Number and percentage of participants with clinical laboratory findings. Evaluations to include are chemistry (serum creatinine, lactate dehydrogenase (LDH), and troponin, and hematology (hemoglobin and hematocrit).

  • Physical examination [ Time Frame: Up to 6 days after transfusion ] [ Designated as safety issue: Yes ]
    Number and percentage of participants with physical examination findings.

  • Electrocardiogram (ECG) [ Time Frame: Up to 6 days after transfusion ] [ Designated as safety issue: Yes ]
    Number and percentage of participants with electrocardiogram (ECG) findings

  • Vital signs [ Time Frame: Up to 6 days after transfusion ] [ Designated as safety issue: Yes ]
    Number and percentage of participants with vital sign findings

  • Thrombotic events [ Time Frame: Up to 6 days after transfusion ] [ Designated as safety issue: Yes ]
    Number and percentage of participants with any signs or symptoms of thrombotic events.

  • Pre and post CPP transfusion coagulation [ Time Frame: Up to 6 days after transfusion ] [ Designated as safety issue: Yes ]
    Number and percentage of participants with pre and post CPP transfusion coagulation findings. These include fibrinogen, D-dimer, prothrombin fragments 1 + 2, thrombin-anti-thrombin (TAT), anti-thrombin (AT), prothrombin time (PT), and activated partial thromboplastin time (aPTT) findings


Estimated Enrollment: 42
Study Start Date: April 2014
Estimated Study Completion Date: April 2015
Estimated Primary Completion Date: April 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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)
Biological: 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 Name: dimethyl sulfoxide (DMSO) cryopreserved platelets (CPP)
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)
Biological: LSP
Other Name: liquid stored platelets (LSP)
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)
Biological: 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 Name: dimethyl sulfoxide (DMSO) cryopreserved platelets (CPP)
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)
Biological: LSP
Other Name: liquid stored platelets (LSP)
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)
Biological: 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 Name: dimethyl sulfoxide (DMSO) cryopreserved platelets (CPP)
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)
Biological: LSP
Other Name: liquid stored platelets (LSP)
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)
Biological: 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 Name: dimethyl sulfoxide (DMSO) cryopreserved platelets (CPP)
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
Biological: LSP
Other Name: liquid stored platelets (LSP)

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male or female, at least 18 years of age.
  • Blood type A or O.
  • 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, chronic 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.
  • World Health Organization (WHO) grade 2 or greater bleeding.
  • Liquid stored platelets (LSP) transfusion within the previous 48 hours.

Exclusion Criteria:

  • Splenomegaly (for the purposes of this study, it is defined as a palpable spleen 5 cm below the costal margin or >18 cm in the largest dimension by ultrasound).
  • Blood type other than A or O.
  • Acute or chronic disseminated intravascular coagulation (DIC) as evidence by D-dimers 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).
  • Prothrombin time (PT) or activated partial thromboplastin time (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.
  • Clinically significant chronic renal (creatinine >1.8 mg/dL) or hepatic dysfunction (PT >1.3 x the upper limit of normal for the laboratory).
  • Females who are breastfeeding.
  • Veno-occlusive disease or possible veno-occlusive disease.
  • Receiving active, inpatient treatment with anti-platelet drugs and/or full anti-coagulation 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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02078284

Contacts
Contact: Sherrill J Slichter, MD 206-292-6541 sjslichter@psbc.org
Contact: Terry B Gernsheimer, MD 206-543-3360 bldbuddy@u.washington.edu

Locations
United States, Washington
Puget Sound Blood Center Not yet recruiting
Seattle, Washington, United States, 98104
Contact: Sherrill J Slichter, MD    206-292-6541    sjslichter@psbc.org   
Principal Investigator: Sherrill J Slichter, MD         
University of Washington, Division of Hematology Not yet recruiting
Seattle, Washington, United States, 98195
Contact: Terry B Gernsheimer, MD    206-543-3360    bldbuddy@u.washington.edu   
Principal Investigator: Terry B Gernsheimer, MD         
Sponsors and Collaborators
U.S. Army Medical Research and Materiel Command
Investigators
Principal Investigator: Sherrill J Slichter, MD Puget Sound Blood Center
Principal Investigator: Terry B Gernsheimer, MD University of Washington, Division of Hematology
  More Information

No publications provided

Responsible Party: U.S. Army Medical Research and Materiel Command
ClinicalTrials.gov Identifier: NCT02078284     History of Changes
Other Study ID Numbers: S-12-04
Study First Received: February 26, 2014
Last Updated: February 28, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by U.S. Army Medical Research and Materiel Command:
platelet transfusion

Additional relevant MeSH terms:
Blood Platelet Disorders
Thrombocytopenia
Hematologic Diseases
Dimethyl Sulfoxide
Cryoprotective Agents
Protective Agents
Physiological Effects of Drugs
Pharmacologic Actions
Free Radical Scavengers
Antioxidants
Molecular Mechanisms of Pharmacological Action

ClinicalTrials.gov processed this record on September 16, 2014