Thrombocytopenia
Conditions
Keywords
Mirasol, Thrombocytopenic, Pathogen reduction, Platelets, Thromboelastography, CCI, Corrected Count Increment
Brief summary
The PRESS trial is a single-center, two-part, randomized cross-over pilot study in Copenhagen that will evaluate 1) the safety and performance of Mirasol-treated and untreated platelet products stored for 2-4 days and for 7-days in Platelet Additive Solution (PAS) and 2) the correlation between the TEG® parameters and platelet count increments after platelet transfusions in thrombocytopenic subjects.
Detailed description
1. To evaluate in thrombocytopenic subjects the efficacy and safety of platelets treated with Mirasol Pathogen Reduction Technology (PRT) in Platelet Additive Solution. The following safety and efficacy measures will be compared between Mirasol treated and untreated platelets stored for 2-4 days and stored for 7 days: * Thrombelastography (TEG®) parameters: reaction time R, alpha angle α and maximal amplitude (MA) * Platelet Count Increment and Corrected Count Increment * Time to next platelet transfusion * Incidence of transfusion related (serious) adverse events * Incidence and severity of bleedings 2. To evaluate the use of the TEG® parameters as a measure for platelet transfusion efficacy and to evaluate the correlation between the TEG® parameters and the platelet Corrected Count Increments after platelet transfusions in thrombocytopenic subjects.
Interventions
BIOLOGICAL2-4-day-old Mirasol-treated Platelets Transfusion
2-4-day-old Mirasol-treated Platelets Units with: * Platelet yield between 2.4x10e11 and 4.5x10e11 * Plasma carryover of \>32% * Cell count \> 800x103/µL
BIOLOGICAL2-4-day-old Untreated Platelets Transfusion
2-4-day-old Untreated Platelets Units with: * Platelet yield between 2.4x10e11 and 4.5x10e11 * Plasma carryover of \>32% * Cell count \> 800x103/µL
Sponsors
Terumo BCTbio
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
BASIC_SCIENCE
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Male or female of age of 18 years or older. * Have thrombocytopenia or expected to develop thrombocytopenia requiring at least 3 platelet transfusions. * Woman of child bearing potential must have a negative serum or urine pregnancy test within 72 hours before randomization. * Subjects must have signed and dated the Informed Consent Form before performing any protocol related procedure.
Exclusion criteria
* History of any hypersensitivity reaction to riboflavin or metabolites. * History of refractoriness to platelet transfusions (defined as 2 successive CCI1hr\<5000) or presence of human leukocyte antigen (HLA) antibodies or positive lymphocytotoxicity or previously documented alloimmunization. * Previous exposure to PRT-treated platelet concentrates. * Active bleeding requiring one or more red cells concentrate transfusions (i.e. grade 3 or 4 bleeding according to the WHO bleeding assessment scale in Appendix 1). * Exposure to an investigational product, within 30 days before randomization. * Splenomegaly (presence of a palpable spleen whose border could be felt more than 4 cm below the costal margin) or splenectomy. * History or diagnosis of Immune/Idiopathic Thrombocytopenic Purpura (ITP), Thrombotic Thrombocytopenia Purpura (TTP), or Haemolytic Uremic Syndrome (HUS). * Use of prohibited medication (see section 5.5). * Pregnant or lactating females. * Any medical condition or treatment that would be expected to compromise the effectiveness of a platelet transfusion or that would interfere with an expected platelet count increment. * Any other medical condition that would compromise the participation of the subject in the study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Maximum Amplitude at 1-hour Post-transfusion | pre-transfusion, 1-hour post transfusion | Thromboelastography (TEG) Parameter: Pre- to post-transfusional modification of Maximum Amplitude at 1-hour post-transfusion |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Maximum Amplitude at 24-hours Post-transfusion | pre-transfusion, 24-hour post transfusion | Thromboelastography parameter: Pre- to post-transfusional modification in Maximum Amplitude at 24-hours post-transfusion |
Countries
Denmark
Participant flow
Pre-assignment details
16 patients enrolled versus 15 patients started: One patient was enrolled by error (exclusion criteria) and excluded after randomization.
Participants by arm
| Arm | Count |
|---|---|
| Mirasol First, Then Reference This study arm will receive first a Mirasol treated 2-4-day-old platelet transfusion and then a reference 2-4-day-old platelet transfusion (Mirasol-Reference sequence).
Mirasol-treated Platelets: Mirasol-treated platelet units with:
* Platelet yield between 2.4x10e11 and 4.5x10e11
* Plasma carryover of \>32%
* Cell count \> 800x103/µL | 8 |
| Reference First, Then Mirasol This study arm will receive first a reference untreated 2-4-day-old platelet transfusion and then a Mirasol treated 2-4-day-old platelet transfusion (Reference-Mirasol sequence).
Mirasol-treated Platelets: Mirasol-treated platelet units with:
* Platelet yield between 2.4x10e11 and 4.5x10e11
* Plasma carryover of \>32%
* Cell count \> 800x103/µL | 7 |
| Total | 15 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Second Platelets Transfusion | No need for additional transfusion | 1 | 2 |
Baseline characteristics
| Characteristic | Mirasol First, Then Reference | Reference First, Then Mirasol | Total |
|---|---|---|---|
| Age, Continuous | 59.13 Years STANDARD_DEVIATION 12.76 | 55.29 Years STANDARD_DEVIATION 19.95 | 57.33 Years STANDARD_DEVIATION 15.99 |
| Region of Enrollment Denmark | 8 participants | 7 participants | 15 participants |
| Sex: Female, Male Female | 0 Participants | 1 Participants | 1 Participants |
| Sex: Female, Male Male | 8 Participants | 6 Participants | 14 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 0 / 13 | 0 / 14 |
| serious Total, serious adverse events | 0 / 13 | 0 / 14 |
Outcome results
Primary
Change in Maximum Amplitude at 1-hour Post-transfusion
Thromboelastography (TEG) Parameter: Pre- to post-transfusional modification of Maximum Amplitude at 1-hour post-transfusion
Time frame: pre-transfusion, 1-hour post transfusion
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Mirasol | Change in Maximum Amplitude at 1-hour Post-transfusion | 9.4 mm | Standard Deviation 7 |
| Untreated Reference | Change in Maximum Amplitude at 1-hour Post-transfusion | 15.4 mm | Standard Deviation 6.1 |
Secondary
Change in Maximum Amplitude at 24-hours Post-transfusion
Thromboelastography parameter: Pre- to post-transfusional modification in Maximum Amplitude at 24-hours post-transfusion
Time frame: pre-transfusion, 24-hour post transfusion
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Mirasol | Change in Maximum Amplitude at 24-hours Post-transfusion | 10.4 mm | Standard Deviation 8 |
| Untreated Reference | Change in Maximum Amplitude at 24-hours Post-transfusion | 8.4 mm | Standard Deviation 4.7 |