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Efficacy and Safety of RBCs Derived From Mirasol-treated Whole Blood in Patients Requiring Chronic Transfusion (PRAISE) (PRAISE)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03329404
Recruitment Status : Terminated (Study suspended due to blood supply challenges. Subsequently approved by FDA to reopen but will not do so because of changing clinical need.)
First Posted : November 6, 2017
Results First Posted : April 28, 2021
Last Update Posted : April 28, 2021
Sponsor:
Collaborators:
United States Department of Defense
Joint Warfighter Medical Research Program
U.S. Army Medical Research and Development Command
Information provided by (Responsible Party):
Terumo BCTbio

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Crossover Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Transfusion Dependent Thalassemia
Interventions Device: Mirasol Red Blood Cells (MIR RBCs)
Device: Reference Red Blood Cells (REF RBCs)
Enrollment 12
Recruitment Details A subject was considered enrolled upon signing the informed consent. The first subject was enrolled on 12 April 2018 and the last subject was enrolled on 26 October 2018.
Pre-assignment Details This was a crossover study, all 9 randomized subjects were to receive both Mirasol and Reference RBCs in either period 1 or 2. 4 subjects were randomized to the MIR/REF RBC treatment sequence and 5 subjects were randomized to the REF/MIR RBC sequence. 4 subjects completed period 1, no subjects completed period 2 due to study suspension. Because no subjects completed the study, the primary endpoint was not evaluated and results are summarized by treatment type rather than treatment sequence.
Arm/Group Title Mirasol Red Blood Cells (MIR RBCs) Followed by Reference RBCs (REF RBCs) Reference Red Blood Cells (REF RBCs) Followed by Mirasol RBCs (MIR RBCs)
Hide Arm/Group Description

MIR RBCs: RBCs were derived from whole blood (WB) collected in citrate phosphate dextrose (CPD) solution, treated with the Mirasol System for WB, Leukoreduced (LR), and stored in Additive Solution Formula 3 (AS-3) for ≤ 21 days at 1 - 6°C.

REF RBCs: LR apheresis RBCs or WB-derived RBCs were per site standard inventory.

REF RBCs: Leukoreduced (LR) apheresis RBCs or whole blood (WB)-derived RBCs were per site standard inventory.

MIR RBCs: RBCs were derived from WB collected in citrate phosphate dextrose (CPD) solution, treated with the Mirasol System for WB, LR, and stored in Additive Solution Formula 3 (AS-3) for ≤ 21 days at 1 - 6°C.

Period Title: Overall Study
Started 4 5
Completed [1] 3 1
Not Completed 1 4
Reason Not Completed
The study was suspended.             1             4
[1]

4 subjects were randomized to the MIR/REF RBC treatment sequence, 3 of the 4 subjects completed the first arm of the study and 1 subject did not complete the first arm of the study.

5 subjects were randomized to the REF/MIR RBC treatment sequence, 1 of the 5 subjects completed the first arm of the study and 4 subjects did not complete the first arm of the study.

In summary- no subjects completed the entire study and 4 subjects completed one arm of the study.

Arm/Group Title Mirasol Red Blood Cells (MIR RBCs)/Reference Red Blood Cells (REF RBCs) Treatment Sequence Reference Red Blood Cells (REF RBCs)/Mirasol Red Blood Cells (MIR RBCs) Treatment Sequence Total
Hide Arm/Group Description

MIR RBCs: RBCs were derived from whole blood (WB) collected in citrate phosphate dextrose (CPD) solution, treated with the Mirasol System for WB, Leukoreduced (LR), and stored in Additive Solution Formula 3 (AS-3) for ≤ 21 days at 1 - 6°C.

REF RBCs: LR apheresis RBCs or WB-derived RBCs were per site standard inventory.

REF RBCs: Leukoreduced (LR) apheresis RBCs or whole blood (WB)-derived RBCs were per site standard inventory.

MIR RBCs: RBCs were derived from WB collected in citrate phosphate dextrose (CPD) solution, treated with the Mirasol System for WB, LR, and stored in Additive Solution Formula 3 (AS-3) for ≤ 21 days at 1 - 6°C.

Total of all reporting groups
Overall Number of Baseline Participants 4 5 9
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 5 participants 9 participants
<=18 years
1
  25.0%
1
  20.0%
2
  22.2%
Between 18 and 65 years
3
  75.0%
4
  80.0%
7
  77.8%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 5 participants 9 participants
Female
2
  50.0%
1
  20.0%
3
  33.3%
Male
2
  50.0%
4
  80.0%
6
  66.7%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 5 participants 9 participants
Hispanic or Latino
0
   0.0%
0
   0.0%
0
   0.0%
Not Hispanic or Latino
3
  75.0%
5
 100.0%
8
  88.9%
Unknown or Not Reported
1
  25.0%
0
   0.0%
1
  11.1%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 4 participants 5 participants 9 participants
4 5 9
1.Primary Outcome
Title Normalized Hemoglobin (Hb AUC) Calculated From Normalized Hb Between Successive Transfusions as a Measure of Percent Surviving RBCs
Hide Description The Hb AUC is calculated using the trapezoidal method on normalized Hb. The normalization is accomplished by dividing all posttransfusion Hb values by the 15-minute posttransfusion Hb level. The ratio is expressed as a percentage. A natural log-transform of the observed normalized Hb AUC will be utilized.
Time Frame Crossover design with 2 treatment periods. Each period included a 50-day wash-in followed by 2 transfusion episodes for primary endpoint assessment. Expected participation was approximately 7-10 months, depending on the subject's transfusion schedule.
Hide Outcome Measure Data
Hide Analysis Population Description
The full Statistical Analysis Plan was not executed for this study because the study was stopped prematurely. 97 subjects were planned to be enrolled however only 12 subjects enrolled and 9 were randomized. Of the 9 randomized, no subjects completed the study due to early termination of the study. The primary outcome measure was not analyzed due to the small sample size and premature termination of the study.
Arm/Group Title Mirasol Red Blood Cells (MIR RBCs) Reference Red Blood Cells (REF RBCs)
Hide Arm/Group Description:

MIR RBCs: RBCs were derived from whole blood (WB) collected in citrate phosphate dextrose (CPD) solution, treated with the Mirasol System for WB, Leukoreduced (LR), and stored in Additive Solution Formula 3 (AS-3) for ≤ 21 days at 1 - 6°C.

REF RBCs: LR apheresis RBCs or WB-derived RBCs were per site standard inventory.

REF RBCs: Leukoreduced (LR) apheresis RBCs or whole blood (WB)-derived RBCs will be per site standard inventory.

MIR RBCs: RBCs will be derived from WB collected in citrate phosphate dextrose (CPD) solution, treated with the Mirasol System for WB, LR, and stored in Additive Solution Formula 3 (AS-3) for ≤ 21 days at 1 - 6°C.

Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
2.Secondary Outcome
Title Hb Increment
Hide Description (post-transfusion Hb - pre-transfusion Hb)/Hb transfused]/RBC volume in subject at pre-transfusion
Time Frame An average of 30 weeks consisting of 2 crossover treatment periods with each period including a 50 day wash-in phase followed by 2 transfusion episodes for endpoint assessment
Outcome Measure Data Not Reported
3.Secondary Outcome
Title Actual Hb Level Post-transfusion (15 Min)
Hide Description Actual Hb level post-transfusion (15 min)
Time Frame An average of 30 weeks consisting of 2 crossover treatment periods with each period including a 50 day wash-in phase followed by 2 transfusion episodes for endpoint assessment
Outcome Measure Data Not Reported
4.Other Pre-specified Outcome
Title Proportional Decline in Post-transfusion Hb Level
Hide Description Proportional decline in post-transfusion Hb level
Time Frame An average of 30 weeks consisting of 2 crossover treatment periods with each period including a 50 day wash-in phase followed by 2 transfusion episodes for endpoint assessment
Outcome Measure Data Not Reported
5.Other Pre-specified Outcome
Title RBC Mass Infused
Hide Description volume x Hb/unit
Time Frame An average of 30 weeks consisting of 2 crossover treatment periods with each period including a 50 day wash-in phase followed by 2 transfusion episodes for endpoint assessment
Outcome Measure Data Not Reported
6.Other Pre-specified Outcome
Title Incidence of Treatment-emergent Antibody With Confirmed Specificity to RBCs Derived From Mirasol-treated WB
Hide Description [Not Specified]
Time Frame Up to 40 weeks consisting of 2 crossover treatment periods with each period including a 50 day wash-in phase followed by 2 transfusion episodes for endpoint assessment and a final study visit 60 days after last study transfusion
Outcome Measure Data Not Reported
7.Other Pre-specified Outcome
Title Human Leukocyte Antigen (HLA) Alloimmunization Rates
Hide Description [Not Specified]
Time Frame An average of 15 weeks consisting of the first treatment period including a 50 day wash-in phase followed by 2 transfusion episodes
Outcome Measure Data Not Reported
8.Other Pre-specified Outcome
Title Treatment Emergent Adverse Events (TEAEs).
Hide Description [Not Specified]
Time Frame Up to 40 weeks consisting of 2 crossover treatment periods with each period including a 50 day wash-in phase followed by 2 transfusion episodes for endpoint assessment and a final study visit 60 days after last study transfusion
Outcome Measure Data Not Reported
9.Other Pre-specified Outcome
Title Transfusion-related Adverse Events (AEs).
Hide Description [Not Specified]
Time Frame Up to 40 weeks consisting of 2 crossover treatment periods with each period including a 50 day wash-in phase followed by 2 transfusion episodes for endpoint assessment and a final study visit 60 days after last study transfusion
Outcome Measure Data Not Reported
10.Other Pre-specified Outcome
Title Serious Adverse Events (SAEs).
Hide Description [Not Specified]
Time Frame Up to 40 weeks consisting of 2 crossover treatment periods with each period including a 50 day wash-in phase followed by 2 transfusion episodes for endpoint assessment and a final study visit 60 days after last study transfusion
Outcome Measure Data Not Reported
11.Other Pre-specified Outcome
Title Unanticipated Adverse Device Effects (UADEs).
Hide Description [Not Specified]
Time Frame Up to 40 weeks consisting of 2 crossover treatment periods with each period including a 50 day wash-in phase followed by 2 transfusion episodes for endpoint assessment and a final study visit 60 days after last study transfusion
Outcome Measure Data Not Reported
Time Frame 8 months
Adverse Event Reporting Description This was a crossover study, therefore adverse events are reported by treatment received, some subjects received both treatments. Of the 9 randomized subjects, 1 subject did not receive any study transfusions, 4 of the 9 subjects received some MIR RBC transfusions and 8 of the 9 subjects received some REF RBC transfusions.
 
Arm/Group Title Mirasol Red Blood Cells (MIR RBCs) Reference Red Blood Cells (REF RBCs)
Hide Arm/Group Description MIR RBCs: RBCs were derived from whole blood (WB) collected in citrate phosphate dextrose (CPD) solution, treated with the Mirasol System for WB, leukoreduced, and stored in Additive Solution Formula 3 (AS-3) for ≤ 21 days at 1 - 6°C. REF RBCs: Leukoreduced apheresis RBCs or whole blood-derived RBCs were per site standard inventory.
All-Cause Mortality
Mirasol Red Blood Cells (MIR RBCs) Reference Red Blood Cells (REF RBCs)
Affected / at Risk (%) Affected / at Risk (%)
Total   0/4 (0.00%)      0/8 (0.00%)    
Hide Serious Adverse Events
Mirasol Red Blood Cells (MIR RBCs) Reference Red Blood Cells (REF RBCs)
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/4 (0.00%)      0/8 (0.00%)    
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Mirasol Red Blood Cells (MIR RBCs) Reference Red Blood Cells (REF RBCs)
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/4 (25.00%)      4/8 (50.00%)    
Blood and lymphatic system disorders     
Thrombocytosis  1  1/4 (25.00%)  1 0/8 (0.00%)  0
Thrombocytopenia  1  0/4 (0.00%)  0 1/8 (12.50%)  1
Infections and infestations     
Viral upper respiratory tract infection  1  0/4 (0.00%)  0 1/8 (12.50%)  1
Investigations     
Aspartate aminotransferase increased  1  0/4 (0.00%)  0 1/8 (12.50%)  1
Alanine aminotransferase increased  1  0/4 (0.00%)  0 1/8 (12.50%)  1
Metabolism and nutrition disorders     
Hypoglycemia  1  1/4 (25.00%)  1 0/8 (0.00%)  0
Skin and subcutaneous tissue disorders     
Rash  1  0/4 (0.00%)  0 1/8 (12.50%)  1
1
Term from vocabulary, MedDRA Version 19.0
Indicates events were collected by systematic assessment
The primary endpoint was not evaluated due to the early termination of the study and subsequent small sample size with no subjects completing both Period 1 and Period 2 of the study preventing any meaningful results.
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: NCT03329404    
Other Study ID Numbers: CTS-5056
First Submitted: October 23, 2017
First Posted: November 6, 2017
Results First Submitted: March 31, 2021
Results First Posted: April 28, 2021
Last Update Posted: April 28, 2021