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

NCT ID: NCT03329404

Last Updated: 2024-02-14

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: NA
• Total Enrollment: 9 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-04-12
• Study Completion Date: 2018-12-19

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.

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: Period 1 = Mirasol-treated RBCs followed by conventional (reference) RBCs; Period 2 = Reference RBCs followed by Mirasol-treated RBCs. 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.

Conditions

Transfusion Dependent Thalassemia

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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

Group Type: EXPERIMENTAL

Mirasol Red Blood Cells (MIR RBCs)

Intervention Type: DEVICE

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.

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

Group Type: ACTIVE_COMPARATOR

Reference Red Blood Cells (REF RBCs)

Intervention Type: DEVICE

Reference Red Blood Cells (REF RBCs) will be acquired from routine use inventory and transfused according to the patient's transfusion schedule.

Interventions

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.

Intervention Type: 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.

Intervention Type: DEVICE

Eligibility 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.

• Minimum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

United States Department of Defense

FED

Sponsor Role: collaborator

Joint Warfighter Medical Research Program

OTHER

Sponsor Role: collaborator

U.S. Army Medical Research and Development Command

FED

Sponsor Role: collaborator

Terumo BCTbio

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Ned Cosgriff, MD

Role: STUDY_DIRECTOR

Terumo BCT

Steve Sloan, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Boston Children's Hospital

Locations

UCSF Benioff Children's Hospital Oakland

Oakland, California, United States

Boston Children's Hospital

Boston, Massachusetts, United States

Weill-Cornell Medical College

New York, New York, United States

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Rambam Health Care Campus

Haifa, , Israel

Hadassah Ein Kerem Hospital

Jerusalem, , Israel

Centro della Microcitemia ed Anemie Congenite Ospedale Gallieria

Genova, Genoa, Italy

U.O.C. di Ematologia e Malattie Rare del Sangue e degli Organi Ematopoietici V. Cervello Hospital

Palermo, , Italy

Ege University Children's Hospital

Bornova, İzmir, Turkey (Türkiye)

Countries

United States; Israel; Italy; Turkey (Türkiye)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

CTS-5056

Identifier Type: -

Identifier Source: org_study_id