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Trial record 8 of 203 for:    MIRASOL

Efficacy of Mirasol-treated Apheresis Platelets in Patients With Hypoproliferative Thrombocytopenia (MIPLATE)

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ClinicalTrials.gov Identifier: NCT02964325
Recruitment Status : Terminated (Based interim analysis results, Data Monitoring Committee did not believe the primary efficacy endpoint would be met. No patient safety concerns.)
First Posted : November 16, 2016
Results First Posted : July 16, 2021
Last Update Posted : August 19, 2021
Sponsor:
Collaborator:
Biomedical Advanced Research and Development Authority
Information provided by (Responsible Party):
Terumo BCTbio

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Investigator, Outcomes Assessor);   Primary Purpose: Supportive Care
Conditions Hematologic Malignancies
Hypoproliferative Thrombocytopenia
Interventions Device: Mirasol platelets (MIR PLTs)
Device: Reference platelets (REF PLTs)
Enrollment 422
Recruitment Details Recruitment occurred at 11 hospital sites within the US. Enrollment occurred between 05 MAY 2017 and 07 APR 2020.
Pre-assignment Details Full Analysis Set (FAS) - all randomized subjects. Safety Set (SS) - randomized subjects who received at least 1 PLT transfusion post-randomization, independent of the outcome or successful completions of the procedure. Modified Intent-to-Treat (mITT) - all randomized subjects who had at least 1 study transfusion according to randomized study group. 422 subjects consented, 92 screen failed, 330 FAS, 28 received no transfusion, 302 SS, 5 received no transfusion per assigned group, 297 mITT.
Arm/Group Title MIRASOL CONTROL
Hide Arm/Group Description

Randomized to leukoreduced, Trima Accel® apheresis platelets stored in 100% plasma, pathogen reduced with the Mirasol® Pathogen Reduction Technology (PRT) System

Mirasol platelets (MIR PLTs): The final product to be transfused to the subject will be leukoreduced (LR), apheresis (Aph) single-donor platelets (PLTs) at the standard therapeutic dose of 1 unit of Aph PLTs containing ≥ 3.0 × 1.0E11 PLTs. MIR PLTs will be treated with the Mirasol pathogen reduction technology system.

Randomized to leukoreduced, apheresis platelets stored in 100% plasma

Reference platelets (REF PLTs): The final product to be transfused to the subject will be LR-Aph single-donor PLTs at the standard therapeutic dose of 1 unit of Aph PLTs containing ≥ 3.0 × 1.0E11 PLTs.

Period Title: Overall Study
Started 164 166
Full Analysis Set [1] 164 166
Modified Intent-to-Treat [2] 145 152
Safety Set [3] 141 161
Completed 143 143
Not Completed 21 23
Reason Not Completed
Lost to Follow-up             3             3
Physician Decision             5             4
Death             1             3
Withdrawal by Subject             2             1
Did not require PLT transfusion, had HLA positive             10             12
[1]
The Full Analysis Set (FAS) included all randomized subjects. Subjects were analyzed according to the treatment group to which they were assigned at randomization.
[2]
The Modified Intent-to-Treat (mITT) Set included all randomized subjects who underwent at least 1 study transfusion post randomization according to the treatment group (MIRASOL or CONTROL) to which they were randomized.
[3]
The Safety Set (SS) included all randomized subjects who underwent at least 1 PLT transfusion post randomization, independent of the outcome or successful completion of the procedure. Subjects were analyzed according to the majority treatment (MIR PLTs or REF PLTs) received.
Arm/Group Title MIRASOL CONTROL Total
Hide Arm/Group Description

Randomized to leukoreduced, Trima Accel® apheresis platelets stored in 100% plasma, pathogen reduced with the Mirasol® Pathogen Reduction Technology (PRT) System

Mirasol platelets (MIR PLTs): The final product to be transfused to the subject will be leukoreduced (LR), apheresis (Aph) single-donor platelets (PLTs) at the standard therapeutic dose of 1 unit of Aph PLTs containing ≥ 3.0 × 1.0E11 PLTs. MIR PLTs will be treated with the Mirasol pathogen reduction technology system.

Randomized to leukoreduced, apheresis platelets stored in 100% plasma

Reference platelets (REF PLTs): The final product to be transfused to the subject will be LR-Aph single-donor PLTs at the standard therapeutic dose of 1 unit of Aph PLTs containing ≥ 3.0 × 1.0E11 PLTs.

Total of all reporting groups
Overall Number of Baseline Participants 141 161 302
Hide Baseline Analysis Population Description
The Safety Analysis Set was used for this analysis. Safety Set (SS) = randomized subjects who received at least 1 PLT transfusion post-randomization, independent of the outcome or successful completions of the procedure. Subjects were analyzed according to the majority treatment received.
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 141 participants 161 participants 302 participants
<=18 years
6
   4.3%
10
   6.2%
16
   5.3%
Between 18 and 65 years
94
  66.7%
100
  62.1%
194
  64.2%
>=65 years
41
  29.1%
51
  31.7%
92
  30.5%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 141 participants 161 participants 302 participants
54.8  (16.32) 54.1  (18.24) 54.4  (17.34)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 141 participants 161 participants 302 participants
Female
53
  37.6%
56
  34.8%
109
  36.1%
Male
88
  62.4%
105
  65.2%
193
  63.9%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 141 participants 161 participants 302 participants
Hispanic or Latino
8
   5.7%
8
   5.0%
16
   5.3%
Not Hispanic or Latino
130
  92.2%
147
  91.3%
277
  91.7%
Unknown or Not Reported
3
   2.1%
6
   3.7%
9
   3.0%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 141 participants 161 participants 302 participants
American Indian or Alaska Native
1
   0.7%
0
   0.0%
1
   0.3%
Black or African American
16
  11.3%
10
   6.2%
26
   8.6%
Native Hawaiian or Other Pacific Islander
0
   0.0%
1
   0.6%
1
   0.3%
White
120
  85.1%
137
  85.1%
257
  85.1%
Missing
0
   0.0%
1
   0.6%
1
   0.3%
Asian
2
   1.4%
10
   6.2%
12
   4.0%
Other
2
   1.4%
2
   1.2%
4
   1.3%
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
United States Number Analyzed 141 participants 161 participants 302 participants
141
 100.0%
161
 100.0%
302
 100.0%
Height (cm)  
Mean (Standard Deviation)
Unit of measure:  Centimeters
Number Analyzed 141 participants 161 participants 302 participants
170.29  (15.039) 167.88  (18.472) 169.01  (16.972)
Weight (kg)  
Mean (Standard Deviation)
Unit of measure:  Kilograms
Number Analyzed 141 participants 161 participants 302 participants
86.96  (23.876) 84.95  (25.400) 85.89  (24.680)
Body Surface Area (BSA)   [1] 
Mean (Standard Deviation)
Unit of measure:  Square meters
Number Analyzed 141 participants 161 participants 302 participants
1.975  (0.3157) 1.934  (0.3613) 1.953  (0.3408)
[1]
Measure Description: The Dubois formula was used to calculate body surface area (BSA). BSA (m2) = 0.007184 × Height (cm)0.725 × Weight (kg)0.425
1.Primary Outcome
Title Days of ≥ Grade 2 Bleeding
Hide Description Number of days of Grade 2 or higher bleeding recorded from treatment start date through 28 days following the first transfusion, until transfusion independence (10 days without PLT transfusion) prior to Day 28, or study termination, whichever occurred first. Subjects who obtained transfusion independence prior to Day 28 were assumed to have zero bleeding events between the date of transfusion independence and Day 28. Observed and simulated data for off-protocol transfusion intervals were included.
Time Frame From the first post-randomization platelet transfusion through 28 days following the first transfusion.
Hide Outcome Measure Data
Hide Analysis Population Description
The Modified Intent-to-Treat Analysis Set was used for this analysis. Modified Intent-to-Treat Analysis Set = all randomized subjects who had at least 1 study transfusion according to randomized study group.
Arm/Group Title MIRASOL CONTROL
Hide Arm/Group Description:

Randomized to leukoreduced, Trima Accel® apheresis platelets stored in 100% plasma, pathogen reduced with the Mirasol® Pathogen Reduction Technology (PRT) System

Mirasol platelets (MIR PLTs): The final product to be transfused to the subject will be leukoreduced (LR), apheresis (Aph) single-donor platelets (PLTs) at the standard therapeutic dose of 1 unit of Aph PLTs containing ≥ 3.0 × 1.0E11 PLTs. MIR PLTs will be treated with the Mirasol pathogen reduction technology system.

Randomized to leukoreduced, apheresis platelets stored in 100% plasma

Reference platelets (REF PLTs): The final product to be transfused to the subject will be LR-Aph single-donor PLTs at the standard therapeutic dose of 1 unit of Aph PLTs containing ≥ 3.0 × 1.0E11 PLTs.

Overall Number of Participants Analyzed 145 152
Mean (Standard Deviation)
Unit of Measure: Days
1.7  (4.05) 0.6  (1.51)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection MIRASOL, CONTROL
Comments The MIRASOL and CONTROL groups were compared with respect to the number of days of WHO ≥ Grade 2 bleeding. This was carried out by fitting a negative binomial regression model with an offset defined as the natural logarithm (LN) of the number of days that bleeding was assessed in order to account for the fact that subjects had different numbers of bleeding assessment days.
Type of Statistical Test Non-Inferiority
Comments An NI analysis was carried out to assess the primary efficacy endpoint with the null hypothesis being the MIRASOL group is inferior to the CONTROL group and the alternative hypothesis being the MIRASOL group is non-inferior to the CONTROL group. In this study, the NI margin was 1.6.
Method of Estimation Estimation Parameter Relative Rate
Estimated Value 2.79
Confidence Interval (2-Sided) 95%
1.67 to 4.67
Estimation Comments The MIRASOL group represents the numerator and the CONTROL group represents the denominator for the relative rate. For days during off-protocol intervals, bleeding data were simulated using an estimate of the individual-specific bleeding rate.
2.Secondary Outcome
Title Number and Percentage of Subjects With Human Leukocyte Antigen (HLA) Alloimmunization
Hide Description The outcome was the development of a new HLA Class I antibodies among subjects negative at baseline within each treatment group. Positivity for Class I HLA antibodies was determined by the 5 SD normalized background ratio cutoffs assay threshold (>59.2, LABScreen Mixed LSM12, One Lambda).
Time Frame HLA antibodies were measured at Baseline and Days 14, 28, and 56.
Hide Outcome Measure Data
Hide Analysis Population Description
The Modified Intent-to-Treat Analysis Set was used for this analysis. Modified Intent-to-Treat Analysis Set = all randomized subjects who had at least 1 study transfusion according to randomized study group. Subjects who tested positive at the high assay threshold (5 SD normalized background ratio cutoffs >59.2, LABScreen Mixed LSM12, One Lambda) at Baseline were excluded from this analysis.
Arm/Group Title MIRASOL CONTROL
Hide Arm/Group Description:

Randomized to leukoreduced, Trima Accel® apheresis platelets stored in 100% plasma, pathogen reduced with the Mirasol® Pathogen Reduction Technology (PRT) System

Mirasol platelets (MIR PLTs): The final product to be transfused to the subject will be leukoreduced (LR), apheresis (Aph) single-donor platelets (PLTs) at the standard therapeutic dose of 1 unit of Aph PLTs containing ≥ 3.0 × 1.0E11 PLTs. MIR PLTs will be treated with the Mirasol pathogen reduction technology system.

Randomized to leukoreduced, apheresis platelets stored in 100% plasma

Reference platelets (REF PLTs): The final product to be transfused to the subject will be LR-Aph single-donor PLTs at the standard therapeutic dose of 1 unit of Aph PLTs containing ≥ 3.0 × 1.0E11 PLTs.

Overall Number of Participants Analyzed 136 141
Measure Type: Count of Participants
Unit of Measure: Participants
4
   2.9%
2
   1.4%
3.Secondary Outcome
Title Number and Percentage of Subjects With ≥ Grade 2 Bleeding
Hide Description The number and percentage of subjects with at least 1 day of ≥ Grade 2 bleeding from Day 0 through Day 27 (or until transfusion independence was achieved) by treatment group
Time Frame From the first post-randomization platelet transfusion through 28 days following the first transfusion.
Hide Outcome Measure Data
Hide Analysis Population Description
The Modified Intent-to-Treat Analysis Set was used for this analysis. Modified Intent-to-Treat Analysis Set = all randomized subjects who had at least 1 study transfusion according to randomized study group.
Arm/Group Title MIRASOL CONTROL
Hide Arm/Group Description:

Randomized to leukoreduced, Trima Accel® apheresis platelets stored in 100% plasma, pathogen reduced with the Mirasol® Pathogen Reduction Technology (PRT) System

Mirasol platelets (MIR PLTs): The final product to be transfused to the subject will be leukoreduced (LR), apheresis (Aph) single-donor platelets (PLTs) at the standard therapeutic dose of 1 unit of Aph PLTs containing ≥ 3.0 × 1.0E11 PLTs. MIR PLTs will be treated with the Mirasol pathogen reduction technology system.

Randomized to leukoreduced, apheresis platelets stored in 100% plasma

Reference platelets (REF PLTs): The final product to be transfused to the subject will be LR-Aph single-donor PLTs at the standard therapeutic dose of 1 unit of Aph PLTs containing ≥ 3.0 × 1.0E11 PLTs.

Overall Number of Participants Analyzed 145 152
Measure Type: Count of Participants
Unit of Measure: Participants
58
  40.0%
46
  30.3%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection MIRASOL, CONTROL
Comments The null hypothesis was H0: pt/pc > 1.2 (ie, MIRASOL had more than a 20% higher probability of a patient experiencing at least one WHO ≥ Grade 2 bleed compared to CONTROL).
Type of Statistical Test Non-Inferiority
Comments A non-inferiority margin of 1.2 was used to evaluated this endpoint.
Statistical Test of Hypothesis P-Value 0.7274
Comments [Not Specified]
Method Wald Non-inferiority Test
Comments [Not Specified]
Method of Estimation Estimation Parameter Relative Risk
Estimated Value 1.32
Confidence Interval (2-Sided) 95%
0.97 to 1.81
Estimation Comments The MIRASOL group represents the numerator and the CONTROL group represents the denominator for the relative risk.
4.Secondary Outcome
Title Number and Percentage of Subjects at the First Timepoint of ≥ Grade 2 Bleeding
Hide Description The time to first ≥ Grade 2 bleeding was analyzed using a log-rank test comparing survival curves stratified by treatment group.
Time Frame From the first post-randomization platelet transfusion through 28 days following the first transfusion.
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT Set was used for this analysis. Subjects that did not experience a ≥ Grade 2 bleed were censored at Day 27 or at date of transfusion independence (10th day without a PLT transfusion prior to last follow-up day or Day 27, whichever occurred earlier), where appropriate. Subjects that did not complete the study or were lost to follow-up were censored on the date of their last study visit in the treatment period.
Arm/Group Title MIRASOL CONTROL
Hide Arm/Group Description:

Randomized to leukoreduced, Trima Accel® apheresis platelets stored in 100% plasma, pathogen reduced with the Mirasol® Pathogen Reduction Technology (PRT) System

Mirasol platelets (MIR PLTs): The final product to be transfused to the subject will be leukoreduced (LR), apheresis (Aph) single-donor platelets (PLTs) at the standard therapeutic dose of 1 unit of Aph PLTs containing ≥ 3.0 × 1.0E11 PLTs. MIR PLTs will be treated with the Mirasol pathogen reduction technology system.

Randomized to leukoreduced, apheresis platelets stored in 100% plasma

Reference platelets (REF PLTs): The final product to be transfused to the subject will be LR-Aph single-donor PLTs at the standard therapeutic dose of 1 unit of Aph PLTs containing ≥ 3.0 × 1.0E11 PLTs.

Overall Number of Participants Analyzed 145 152
Measure Type: Count of Participants
Unit of Measure: Participants
Day 4
115
  79.3%
128
  84.2%
Day 8
92
  63.4%
110
  72.4%
Day 12
72
  49.7%
85
  55.9%
Day 16
33
  22.8%
48
  31.6%
Day 20
11
   7.6%
27
  17.8%
Day 24
5
   3.4%
13
   8.6%
Day 28
4
   2.8%
11
   7.2%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection MIRASOL, CONTROL
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.07
Comments [Not Specified]
Method Log-rank test
Comments [Not Specified]
5.Secondary Outcome
Title Number and Percentage of Subjects With ≥ Grade 3 Bleeding
Hide Description The number and percentage of subjects with at least 1 day of ≥ Grade 3 bleeding from Day 0 through Day 27 (or until transfusion independence was achieved).
Time Frame From the first post-randomization platelet transfusion through 28 days following the first transfusion.
Hide Outcome Measure Data
Hide Analysis Population Description
The Modified Intent-to-Treat Analysis Set was used for this analysis. Modified Intent-to-Treat Analysis Set = all randomized subjects who had at least 1 study transfusion according to randomized study group.
Arm/Group Title MIRASOL CONTROL
Hide Arm/Group Description:

Randomized to leukoreduced, Trima Accel® apheresis platelets stored in 100% plasma, pathogen reduced with the Mirasol® Pathogen Reduction Technology (PRT) System

Mirasol platelets (MIR PLTs): The final product to be transfused to the subject will be leukoreduced (LR), apheresis (Aph) single-donor platelets (PLTs) at the standard therapeutic dose of 1 unit of Aph PLTs containing ≥ 3.0 × 1.0E11 PLTs. MIR PLTs will be treated with the Mirasol pathogen reduction technology system.

Randomized to leukoreduced, apheresis platelets stored in 100% plasma

Reference platelets (REF PLTs): The final product to be transfused to the subject will be LR-Aph single-donor PLTs at the standard therapeutic dose of 1 unit of Aph PLTs containing ≥ 3.0 × 1.0E11 PLTs.

Overall Number of Participants Analyzed 145 152
Measure Type: Count of Participants
Unit of Measure: Participants
6
   4.1%
2
   1.3%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection MIRASOL, CONTROL
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1649
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
6.Secondary Outcome
Title Number and Percentage of Subjects With PLT Refractoriness
Hide Description The number and percentage of subjects with PLT refractoriness defined as 2 sequential transfusions, each with corrected count increments (CCIs) < 5000 measured 1 hour post-transfusion.
Time Frame From the first post-randomization platelet transfusion through 28 days following the first transfusion.
Hide Outcome Measure Data
Hide Analysis Population Description
The Modified Intent-to-Treat Analysis Set was used for this analysis. Modified Intent-to-Treat Analysis Set = all randomized subjects who had at least 1 study transfusion according to randomized study group.
Arm/Group Title MIRASOL CONTROL
Hide Arm/Group Description:

Randomized to leukoreduced, Trima Accel® apheresis platelets stored in 100% plasma, pathogen reduced with the Mirasol® Pathogen Reduction Technology (PRT) System

Mirasol platelets (MIR PLTs): The final product to be transfused to the subject will be leukoreduced (LR), apheresis (Aph) single-donor platelets (PLTs) at the standard therapeutic dose of 1 unit of Aph PLTs containing ≥ 3.0 × 1.0E11 PLTs. MIR PLTs will be treated with the Mirasol pathogen reduction technology system.

Randomized to leukoreduced, apheresis platelets stored in 100% plasma

Reference platelets (REF PLTs): The final product to be transfused to the subject will be LR-Aph single-donor PLTs at the standard therapeutic dose of 1 unit of Aph PLTs containing ≥ 3.0 × 1.0E11 PLTs.

Overall Number of Participants Analyzed 145 152
Measure Type: Count of Participants
Unit of Measure: Participants
41
  28.3%
20
  13.2%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection MIRASOL, CONTROL
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0015
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
Method of Estimation Estimation Parameter Risk Ratio (RR)
Estimated Value 2.15
Confidence Interval (2-Sided) 95%
1.32 to 3.49
Estimation Comments [Not Specified]
7.Secondary Outcome
Title Number and Percentage of Subjects With Immune Platelet Refractoriness
Hide Description The number and percentage of subjects with PLT refractoriness for each treatment group. Subjects were defined as immune PLT refractoriness based on 2 sequential transfusion episodes, each with CCIs < 5000 measured 1 hour post transfusion, and who also had a positive antibody test within 14 days before or after the onset of PLT refractoriness.
Time Frame Initial post-randomization platelet transfusion through high Class I HLA development.
Hide Outcome Measure Data
Hide Analysis Population Description
The Modified Intent-to-Treat Analysis Set was used for this analysis. Modified Intent-to-Treat Analysis Set = all randomized subjects who had at least 1 study transfusion according to randomized study group.
Arm/Group Title MIRASOL CONTROL
Hide Arm/Group Description:

Randomized to leukoreduced, Trima Accel® apheresis platelets stored in 100% plasma, pathogen reduced with the Mirasol® Pathogen Reduction Technology (PRT) System

Mirasol platelets (MIR PLTs): The final product to be transfused to the subject will be leukoreduced (LR), apheresis (Aph) single-donor platelets (PLTs) at the standard therapeutic dose of 1 unit of Aph PLTs containing ≥ 3.0 × 1.0E11 PLTs. MIR PLTs will be treated with the Mirasol pathogen reduction technology system.

Randomized to leukoreduced, apheresis platelets stored in 100% plasma

Reference platelets (REF PLTs): The final product to be transfused to the subject will be LR-Aph single-donor PLTs at the standard therapeutic dose of 1 unit of Aph PLTs containing ≥ 3.0 × 1.0E11 PLTs.

Overall Number of Participants Analyzed 136 141
Measure Type: Count of Participants
Unit of Measure: Participants
1
   0.7%
1
   0.7%
8.Other Pre-specified Outcome
Title Number and Percentage of Subjects With Unanticipated Adverse Device Effects (UADEs)
Hide Description UADEs are identified as treatment emergent adverse events reported by the investigator as serious, unanticipated, at least possibly related to study device or at least possibly related to treatment. UADEs were coded using Medical Dictionary for Regulatory Activities (MedDRA) Version 19.1
Time Frame From initial post-randomization PLT transfusion through 72 hours following the last per protocol PLT transfusion.
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Set was used for this analysis. Safety Set = randomized subjects who received at least 1 PLT transfusion post-randomization, independent of the outcome or successful completions of the procedure.
Arm/Group Title MIRASOL CONTROL
Hide Arm/Group Description:

Randomized to leukoreduced, Trima Accel® apheresis platelets stored in 100% plasma, pathogen reduced with the Mirasol® Pathogen Reduction Technology (PRT) System

Mirasol platelets (MIR PLTs): The final product to be transfused to the subject will be leukoreduced (LR), apheresis (Aph) single-donor platelets (PLTs) at the standard therapeutic dose of 1 unit of Aph PLTs containing ≥ 3.0 × 1.0E11 PLTs. MIR PLTs will be treated with the Mirasol pathogen reduction technology system.

Randomized to leukoreduced, apheresis platelets stored in 100% plasma

Reference platelets (REF PLTs): The final product to be transfused to the subject will be LR-Aph single-donor PLTs at the standard therapeutic dose of 1 unit of Aph PLTs containing ≥ 3.0 × 1.0E11 PLTs.

Overall Number of Participants Analyzed 141 161
Measure Type: Count of Participants
Unit of Measure: Participants
Blood and Lymphatic System Disorders/ Febrile Neutropenia
1
   0.7%
0
   0.0%
No UADEs
140
  99.3%
161
 100.0%
Time Frame Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs) that occurred from initial post randomization platelet transfusion through seventy-two (72) hours following the transfusion end time of the last on-protocol PLT transfusion were reported. Deaths that occurred (including deaths due to bleeding) thirty days (30) following the transfusion end time of the last on-protocol PLT transfusion were reported.
Adverse Event Reporting Description Treatment Emergent definition: an event that first appears during treatment, which was absent before or which worsened relative to the pre-treatment state. Treatment emergent are those events that occur during or following the first post-randomization PLT transfusion. All TEAEs/TESAEs were followed until resolution, stabilization, or the end of the subject's study participation which occurred first.
 
Arm/Group Title MIRASOL CONTROL
Hide Arm/Group Description

Randomized to leukoreduced, Trima Accel® apheresis platelets stored in 100% plasma, pathogen reduced with the Mirasol® Pathogen Reduction Technology (PRT) System

Mirasol platelets (MIR PLTs): The final product to be transfused to the subject will be leukoreduced (LR), apheresis (Aph) single-donor platelets (PLTs) at the standard therapeutic dose of 1 unit of Aph PLTs containing ≥ 3.0 × 1.0E11 PLTs. MIR PLTs will be treated with the Mirasol pathogen reduction technology system.

Randomized to leukoreduced, apheresis platelets stored in 100% plasma

Reference platelets (REF PLTs): The final product to be transfused to the subject will be LR-Aph single-donor PLTs at the standard therapeutic dose of 1 unit of Aph PLTs containing ≥ 3.0 × 1.0E11 PLTs.

All-Cause Mortality
MIRASOL CONTROL
Affected / at Risk (%) Affected / at Risk (%)
Total   1/141 (0.71%)      3/161 (1.86%)    
Hide Serious Adverse Events
MIRASOL CONTROL
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   22/141 (15.60%)      33/161 (20.50%)    
Blood and lymphatic system disorders     
Febrile neutropenia  1  4/141 (2.84%)  4 11/161 (6.83%)  12
Cardiac disorders     
Cardiomyopathy  1  0/141 (0.00%)  0 1/161 (0.62%)  1
Right ventricular dysfunction  1  0/141 (0.00%)  0 1/161 (0.62%)  1
Sinus tachycardia  1  1/141 (0.71%)  1 0/161 (0.00%)  0
Tachycardia  1  1/141 (0.71%)  1 0/161 (0.00%)  0
Gastrointestinal disorders     
Abdominal pain  1  1/141 (0.71%)  1 0/161 (0.00%)  0
Diaphragmatic hernia  1  1/141 (0.71%)  1 0/161 (0.00%)  0
Vomiting  1  0/141 (0.00%)  0 1/161 (0.62%)  1
General disorders     
Pyrexia  1  0/141 (0.00%)  0 4/161 (2.48%)  4
Hepatobiliary disorders     
Acute hepatic failure  1  0/141 (0.00%)  0 1/161 (0.62%)  1
Bile duct stone  1  0/141 (0.00%)  0 1/161 (0.62%)  1
Venoocclusive liver disease  1  0/141 (0.00%)  0 1/161 (0.62%)  1
Immune system disorders     
Cytokine release syndrome  1  1/141 (0.71%)  1 0/161 (0.00%)  0
Engraftment syndrome  1  0/141 (0.00%)  0 1/161 (0.62%)  1
Graft versus host disease in gastrointestinal tract  1  0/141 (0.00%)  0 1/161 (0.62%)  1
Infections and infestations     
Septic Shock  1  2/141 (1.42%)  2 2/161 (1.24%)  3
Streptococcal bacteremia  1  2/141 (1.42%)  2 2/161 (1.24%)  2
Bacteraemia  1  3/141 (2.13%)  3 0/161 (0.00%)  0
Staphylococcal bacteraemia  1  1/141 (0.71%)  1 1/161 (0.62%)  1
Stomatococcal infection  1  2/141 (1.42%)  2 0/161 (0.00%)  0
Bronchopulmonary aspergillosis  1  1/141 (0.71%)  1 0/161 (0.00%)  0
Candida infection  1  1/141 (0.71%)  1 0/161 (0.00%)  0
Cellulitis  1  0/141 (0.00%)  0 1/161 (0.62%)  1
Clostridium difficile infection  1  0/141 (0.00%)  0 1/161 (0.62%)  1
Cystitis viral  1  1/141 (0.71%)  1 0/161 (0.00%)  0
Device related infection  1  0/141 (0.00%)  0 1/161 (0.62%)  1
Diverticulitis  1  0/141 (0.00%)  0 1/161 (0.62%)  1
Enterococcal bacteraemia  1  1/141 (0.71%)  1 0/161 (0.00%)  0
Enterococcal infection  1  0/141 (0.00%)  0 1/161 (0.62%)  1
Human herpesvirus 6 infection  1  1/141 (0.71%)  1 0/161 (0.00%)  0
Pneumonia  1  0/141 (0.00%)  0 1/161 (0.62%)  1
Pseudomonal bacteraemia  1  1/141 (0.71%)  1 0/161 (0.00%)  0
Septic arthritis staphylococcal  1  0/141 (0.00%)  0 1/161 (0.62%)  1
Escherichia bacteraemia  1  1/141 (0.71%)  1 1/161 (0.62%)  1
Injury, poisoning and procedural complications     
Allergic transfusion reaction  1  0/141 (0.00%)  0 1/161 (0.62%)  1
Delayed engraftment  1  0/141 (0.00%)  0 1/161 (0.62%)  1
Febrile nonhaemolytic transfusion reaction  1  1/141 (0.71%)  1 0/161 (0.00%)  0
Procedural hypotension  1  0/141 (0.00%)  0 1/161 (0.62%)  1
Transfusion-associated dyspnoea  1  0/141 (0.00%)  0 1/161 (0.62%)  1
Transfusion-related circulatory overload  1  0/141 (0.00%)  0 1/161 (0.62%)  1
Transplant failure  1  0/141 (0.00%)  0 1/161 (0.62%)  1
Investigations     
Blood culture positive  1  1/141 (0.71%)  1 0/161 (0.00%)  0
Musculoskeletal and connective tissue disorders     
Musculoskeletal chest pain  1  0/141 (0.00%)  0 1/161 (0.62%)  1
Nervous system disorders     
Cerebrovascular accident  1  1/141 (0.71%)  1 0/161 (0.00%)  0
Encephalopathy  1  0/141 (0.00%)  0 1/161 (0.62%)  1
Syncope  1  0/141 (0.00%)  0 1/161 (0.62%)  1
Psychiatric disorders     
Mental status changes  1  2/141 (1.42%)  2 0/161 (0.00%)  0
Renal and urinary disorders     
Acute kidney injury  1  1/141 (0.71%)  1 1/161 (0.62%)  1
Renal impairment  1  0/141 (0.00%)  0 1/161 (0.62%)  1
Urinary retention  1  1/141 (0.71%)  1 0/161 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Respiratory failure  1  0/141 (0.00%)  0 2/161 (1.24%)  3
Dyspnoea  1  1/141 (0.71%)  1 0/161 (0.00%)  0
Pneumonia aspiration  1  0/141 (0.00%)  0 1/161 (0.62%)  1
Vascular disorders     
Hypotension  1  3/141 (2.13%)  3 2/161 (1.24%)  2
Deep vein thrombosis  1  1/141 (0.71%)  1 1/161 (0.62%)  1
Embolism  1  1/141 (0.71%)  1 0/161 (0.00%)  0
1
Term from vocabulary, MedDRA (13.0)
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
MIRASOL CONTROL
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   97/141 (68.79%)      100/161 (62.11%)    
Blood and lymphatic system disorders     
Febrile neutropenia  1  24/141 (17.02%)  27 17/161 (10.56%)  17
Anaemia  1  12/141 (8.51%)  12 11/161 (6.83%)  11
Gastrointestinal disorders     
Diarrhoea  1  21/141 (14.89%)  21 35/161 (21.74%)  35
Vomiting  1  19/141 (13.48%)  19 14/161 (8.70%)  14
Nausea  1  12/141 (8.51%)  12 8/161 (4.97%)  8
Abdominal pain  1  6/141 (4.26%)  6 11/161 (6.83%)  11
Stomatitis  1  11/141 (7.80%)  11 6/161 (3.73%)  6
General disorders     
Pyrexia  1  19/141 (13.48%)  20 16/161 (9.94%)  17
Mucosal inflammation  1  15/141 (10.64%)  15 19/161 (11.80%)  19
Oedema peripheral  1  15/141 (10.64%)  18 13/161 (8.07%)  14
Fatigue  1  13/141 (9.22%)  13 9/161 (5.59%)  9
Injury, poisoning and procedural complications     
Febrile nonhaemolytic transfusion reaction  1  7/141 (4.96%)  13 8/161 (4.97%)  9
Metabolism and nutrition disorders     
Hypokalaemia  1  14/141 (9.93%)  15 20/161 (12.42%)  20
Decreased appetite  1  8/141 (5.67%)  8 8/161 (4.97%)  8
Hypomagnesaemia  1  8/141 (5.67%)  8 8/161 (4.97%)  8
Nervous system disorders     
Headache  1  9/141 (6.38%)  9 11/161 (6.83%)  12
Dizziness  1  7/141 (4.96%)  8 9/161 (5.59%)  11
Respiratory, thoracic and mediastinal disorders     
Oropharyngeal pain  1  11/141 (7.80%)  11 10/161 (6.21%)  10
1
Term from vocabulary, MedDRA (13.0)
Indicates events were collected by systematic assessment
The clinical trial was terminated early on the recommendation of the Data Monitoring Committee and in agreement with the Clinical Trial Steering Committee and the Sponsor.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Robert Cortes, Jr. MD
Organization: Terumo Blood and Cell Technologies
Phone: +1.303.231.4353
EMail: Robert.Cortes@terumobct.com
Layout table for additonal information
Responsible Party: Terumo BCTbio
ClinicalTrials.gov Identifier: NCT02964325    
Other Study ID Numbers: CTS-5030
First Submitted: November 9, 2016
First Posted: November 16, 2016
Results First Submitted: June 25, 2021
Results First Posted: July 16, 2021
Last Update Posted: August 19, 2021