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

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ClinicalTrials.gov Identifier: NCT03329404
Recruitment Status : Recruiting
First Posted : November 6, 2017
Last Update Posted : September 28, 2018
Sponsor:
Collaborators:
United States Department of Defense
Joint Warfighter Medical Research Program
U.S. Army Medical Research and Materiel Command
Information provided by (Responsible Party):
Terumo BCTbio

Brief Summary:
This is a prospective, multi-center, randomized, crossover trial to evaluate the clinical effectiveness of red blood cells (RBCs) derived from Mirasol-treated whole blood (WB) versus conventional RBCs in transfusion dependent thalassemia patients. Throughout the clinical study, RBC transfusion volume and frequency will be determined by each subject's treating physician.

Condition or disease Intervention/treatment Phase
Transfusion Dependent Thalassemia Device: Mirasol Red Blood Cells (MIR RBCs) Device: Reference Red Blood Cells (REF RBCs) Not Applicable

Detailed Description:

Patients will be randomized 1:1 to receive either Mirasol-treated RBCs followed by conventional RBCs, or to receive conventional RBCs followed by Mirasol-treated RBCs. The blood centers will collect the donor RBCs and supply the Mirasol-treated RBCs to the hospital sites for transfusion into patients. Hospital sites will order conventional RBCs as per their normal process, from their standard vendor.

Blood transfusion is the mainstay of care for individuals with thalassemia major. The purpose of transfusion is twofold: to improve the anemia and to suppress the ineffective erythropoiesis. A transfusion episode for these thalassemia patients are the routine transfusions administered on a regular schedule for the life of the patient.

The crossover trial design will consist of 2 treatment periods. Each period will include a 50 day wash-in phase (Day 0 of the wash-in = Day 0 of the treatment period) followed by 2 transfusion episodes. An end of study treatment follow-up visit will occur 2-4 weeks after the last per protocol transfusion, prior to the next standard of care transfusion. A final study visit will occur at least 60 days after the last per protocol transfusion.

The primary objective of the PRAISE study is to determine if percent survival of RBCs derived from Mirasol-treated WB is non-inferior to conventional RBCs when transfused into patients requiring chronic RBC transfusion support. The secondary objectives include comparing other efficacy and safety endpoints between treatment groups.


Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 97 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Evaluate the Efficacy and Safety of RBCs Derived From Mirasol-treated Whole Blood Compared With Conventional RBCs in Patients Requiring Chronic Transfusion Support
Actual Study Start Date : April 23, 2018
Estimated Primary Completion Date : June 2020
Estimated Study Completion Date : June 2020

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Mirasol Red Blood Cells (MIR RBCs)
MIR RBCs: RBCs will be derived from WB collected in CPD solution, treated with the Mirasol System for WB, LR, and stored in AS-3 for ≤ 21 days at 1 6°C
Device: Mirasol Red Blood Cells (MIR RBCs)
Mirasol Red Blood Cells (MIR RBCs) derived from Mirasol-treated WB; WB will be Mirasol treated, centfifuged and leukoreduced and the derived RBCs will be stored before transfusion for up to 21 days and transfused according to the patient's transfusion schedule.

Active Comparator: Reference Red Blood Cells (REF RBCs)
Reference Red Blood Cells (REF RBCs); LR apheresis RBCs or WB-derived RBCs will be per site standard inventory
Device: Reference Red Blood Cells (REF RBCs)
Reference Red Blood Cells (REF RBCs) will be acquired from routine use inventory and transfused according to the patient's transfusion schedule.




Primary Outcome Measures :
  1. Normalized hemoglobin (Hb AUC) calculated from normalized Hb between successive transfusions as a measure of percent surviving RBCs [ 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 ]
    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.


Secondary Outcome Measures :
  1. Hb increment [ 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 ]
    (post-transfusion Hb - pre-transfusion Hb)/Hb transfused]/RBC volume in subject at pre-transfusion

  2. 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 ]
    Actual Hb level post-transfusion (15 min)


Other Outcome Measures:
  1. 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 ]
    Proportional decline in post-transfusion Hb level

  2. RBC mass infused [ 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 ]
    volume x Hb/unit

  3. Incidence of treatment-emergent antibody with confirmed specificity to RBCs derived from Mirasol-treated WB [ 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 ]
  4. Human leukocyte antigen (HLA) alloimmunization rates [ 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 ]
  5. Treatment emergent adverse events (TEAEs). [ 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 ]
  6. Transfusion-related adverse events (AEs). [ 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 ]
  7. Serious adverse events (SAEs). [ 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 ]
  8. Unanticipated adverse device effects (UADEs). [ 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 ]


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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 1. Transfusion dependent thalassemia patient with mean 2-4 week transfusion intervals for the prior 6 months.

    2. Age ≥ 12 years.

    3. Negative pregnancy test for women of childbearing potential and agreement to practice a medically acceptable contraception regimen throughout the participation in the clinical trial. Not required if female subjects are not of child-bearing potential (ie, prior to menses onset, surgically sterilized, 1-year postmenopausal).

    4. Signed informed consent from the patient, or if the patient is < 18 years of age, signed assent from patient and consent from parent/guardian, according to local Institutional Review Board/Ethics Committee (IRB/EC) requirements.

Exclusion Criteria:

  1. Historical RBC transfusion requirement of more than 250 mL/kg/year.
  2. Presence of RBC antibodies that make procurement of compatible RBC units not feasible per the treating physician's clinical judgment for reasonable execution of the study.
  3. Prior treatment with pathogen-reduced RBCs with subsequent development of known antibodies to the associated RBCs.
  4. Planned treatment requirement of frozen RBC products.
  5. Treatment requirements for any medication that is known to cause hemolysis.
  6. Receiving cardiac medications for heart failure.
  7. Patients anticipated to receive massive transfusion, per the treating physician's clinical judgment.
  8. Known HIV infection (defined as HIV RNA positive) with changes to antiviral regimen within the 12 months prior to screening.
  9. Acute or chronic medical disorder that, in the opinion of the Investigator, would impair the ability of the patient to receive study treatment.
  10. Participation in another clinical study, either concurrently or within the previous 28 days, in which the study drug or device may influence study endpoints or patient safety, according to Investigator discretion.
  11. Participation in another clinical study within the past 3 months if investigational RBCs or treatment or drugs were received that are likely to have long term effect on RBCs function.
  12. Pregnant or breastfeeding.
  13. Planned concurrent treatment with other pathogen reduction treated blood products during participation in this study.
  14. Patients who received prior treatment with pathogen-reduced RBCs within the past 120 days.
  15. Inability to comply with study procedures and/or follow-up.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03329404


Contacts
Contact: Rosie Gunter 303-542-5293 Rosie.Gunter@terumobct.com
Contact: Shannon Godbold, RN, BSN 303-239-2010 Shannon.Godbold@terumobct.com

Locations
United States, California
UCSF Benioff Children's Hospital Oakland Recruiting
Oakland, California, United States, 94609
Contact: Kacie Smith    510-428-3885 ext 2752    KaSmith@mail.cho.org   
United States, Massachusetts
Boston Children's Hospital Not yet recruiting
Boston, Massachusetts, United States, 02116
Contact: Ashley Cronkright    617-355-7203    Ashley.Cronkright@childrens.harvard.edu   
United States, New York
Weill-Cornell Medical College Recruiting
New York, New York, United States, 10065
Contact: Peter Keefe    212-746-4933    pek2010@med.cornell.edu   
United States, Pennsylvania
The Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Abinaya Arulselvan    215-590-3582    Arulselvaa@email.chop.edu   
Israel
Rambam Health Care Campus Not yet recruiting
Haifa, Israel, 3109601
Contact: Lilach Bonstein    972 4 7772052    l_bonstein@rambam.health.gov.il   
Hadassah Ein Kerem Hospital Not yet recruiting
Jerusalem, Israel, 91120
Contact: Tali Zelikovich    +972-2-6776047    Orlyz@hadassah.org.il   
Italy
Centro della Microcitemia ed Anemie Congenite Ospedale Gallieria Not yet recruiting
Genova, Genoa, Italy, 16128
Contact: Giulia Albasini    003-0105634583    giulia.albasini@galliera.it   
U.O.C. di Ematologia e Malattie Rare del Sangue e degli Organi Ematopoietici V. Cervello Hospital Not yet recruiting
Palermo, Italy, 90146
Contact: Angela Vitrano    39 091 6802841    angelavitranos4@gmail.com   
Turkey
Ege University Children's Hospital Not yet recruiting
Bornova, Izmir, Turkey, 35040
Contact: Yesim Aydinok, Prof. Dr.    +90(232) 3904219    yesim.aydinok@yahoo.com   
Sponsors and Collaborators
Terumo BCTbio
United States Department of Defense
Joint Warfighter Medical Research Program
U.S. Army Medical Research and Materiel Command
Investigators
Study Director: Ned Cosgriff, MD Terumo BCT
Principal Investigator: Steve Sloan, MD, PhD Boston Children’s Hospital

Responsible Party: Terumo BCTbio
ClinicalTrials.gov Identifier: NCT03329404     History of Changes
Other Study ID Numbers: CTS-5056
First Posted: November 6, 2017    Key Record Dates
Last Update Posted: September 28, 2018
Last Verified: May 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: Yes
Device Product Not Approved or Cleared by U.S. FDA: Yes
Pediatric Postmarket Surveillance of a Device Product: No

Keywords provided by Terumo BCTbio:
Thalassemia
pathogen reduction therapy

Additional relevant MeSH terms:
Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn