NCT03329404

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

Evaluate the Efficacy and Safety of RBCs Derived From Mirasol-treated Whole Blood Compared With Conventional RBCs in Patients Requiring Chronic Transfusion Support

• Status: Terminated
• Phase: N/A
• Study type: Interventional
• Enrollment: 9 (actual)
• Sponsor: Terumo BCTbio · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

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

Interventions

Type	Name	Description
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.
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.

Timeline

• Start date: 2018-04-12
• Primary completion: 2018-12-19
• Completion: 2018-12-19
• First posted: 2017-11-06
• Last updated: 2024-02-14
• Results posted: 2021-04-28

Locations

9 sites across 4 countries: United States, Israel, Italy, Turkey (Türkiye)

Regulatory

• FDA-regulated device study