Transfusion Dependent ThalassemiaPRAISENCT03329404
Efficacy and Safety of RBCs Derived From Mirasol-treated Whole Blood in Patients Requiring Chronic Transfusion (PRAISE)
A study testing Mirasol Red Blood Cells (MIR RBCs) and Reference Red Blood Cells (REF RBCs) in people with Transfusion Dependent Thalassemia, run by Terumo BCTbio at 9 sites in 4 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Transfusion Dependent Thalassemia
- What is being tested?
- Mirasol Red Blood Cells (MIR RBCs) and Reference Red Blood Cells (REF RBCs)
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 9 sitesin 4 countries
- Study dates
- 12 Apr 2018to 19 Dec 2018
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- Historical RBC transfusion requirement of more than 250 mL/kg/year.
- 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.
- Prior treatment with pathogen-reduced RBCs with subsequent development of known antibodies to the associated RBCs.
- Planned treatment requirement of frozen RBC products.
- Treatment requirements for any medication that is known to cause hemolysis.
- Receiving cardiac medications for heart failure.
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- Patients anticipated to receive massive transfusion, per the treating physician's clinical judgment.
- Known HIV infection (defined as HIV RNA positive) with changes to antiviral regimen within the 12 months prior to screening.
- Acute or chronic medical disorder that, in the opinion of the Investigator, would impair the ability of the patient to receive study treatment.
- 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.
- 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.
- Pregnant or breastfeeding.
- Planned concurrent treatment with other pathogen reduction treated blood products during participation in this study.
- Patients who received prior treatment with pathogen-reduced RBCs within the past 120 days.
- Inability to comply with study procedures and/or follow-up.
Trial locations and contacts
9 sites in 4 countries.
9 sites in 4 countries
United States4 sites
Boston Children's Hospital
Boston, Massachusetts, 02116
Weill-Cornell Medical College
New York, New York, 10065
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Israel2 sites
Rambam Health Care Campus
Haifa, 3109601
Hadassah Ein Kerem Hospital
Jerusalem, 91120
Italy2 sites
Centro della Microcitemia ed Anemie Congenite Ospedale Gallieria
Genova, Genoa, 16128
U.O.C. di Ematologia e Malattie Rare del Sangue e degli Organi Ematopoietici V. Cervello Hospital
Palermo, 90146
Turkey (Türkiye)1 site
Ege University Children's Hospital
Bornova, İzmir, 35040
About this study
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
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.
Read the 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
- Being tested
- Mirasol Red Blood Cells (MIR RBCs), Reference Red Blood Cells (REF RBCs)
- Keywords
- Thalassemia, pathogen reduction therapy
- Medical subject headings
- Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Mirasol Red Blood Cells (MIR RBCs)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)Device
Reference Red Blood Cells (REF RBCs) will be acquired from routine use inventory and transfused according to the patient's transfusion schedule.
| Study group | Role | What they receive |
|---|---|---|
| Mirasol Red Blood Cells (MIR RBCs) | Experimental | 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 Receives: Device: Mirasol Red Blood Cells (MIR RBCs) |
| Reference Red Blood Cells (REF RBCs) | Active comparator | Reference Red Blood Cells (REF RBCs); LR apheresis RBCs or WB-derived RBCs will be per site standard inventory Receives: Device: Reference Red Blood Cells (REF RBCs) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Normalized Hemoglobin (Hb AUC) Calculated From Normalized Hb Between Successive Transfusions as a Measure of Percent Surviving RBCs
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.
Measured: 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.
Secondary outcomes (2)
Hb Increment
(post-transfusion Hb - pre-transfusion Hb)/Hb transfused]/RBC volume in subject at pre-transfusion
Measured: Endpoint assessments was evaluated at 15 min Post Transfusion, 1 Day Post Transfusion, 7 Day Post Transfusion, and End of Transfusion Episode.
Actual Hb Level Post-transfusion (15 Min)
Actual Hb level post-transfusion (15 min)
Measured: 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
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Nov 2017
- Study start12 Apr 2018
- Primary completion19 Dec 2018
- Study completion19 Dec 2018
Why it stopped: Study suspended due to blood supply challenges. Subsequently approved by FDA to reopen but will not do so because of changing clinical need.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Transfusion Dependent ThalassemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Terumo BCTbio
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- United States Department of Defense, Joint Warfighter Medical Research Program, U.S. Army Medical Research and Development Command
- Responsible party
- Sponsor
- Investigators
- Ned Cosgriff, MD, Study director, Terumo BCTSteve Sloan, MD, PhD, Principal investigator, Boston Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03329404
- Sponsor's protocol ID
- CTS-5056
- Acronym
- PRAISE
- Status last verified
- Jan 2024
- First submitted
- 23 Oct 2017
- Last updated
- 14 Feb 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03329404
- Acronym / sponsor ID
- PRAISE / CTS-5056
- Conditions
- Transfusion Dependent Thalassemia
- Keywords
- Thalassemia, pathogen reduction therapy
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study suspended due to blood supply challenges. Subsequently approved by FDA to reopen but will not do so because of changing clinical need.
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Terumo BCTbioIndustry (pharma, biotech, devices)
- Collaborators
- United States Department of Defense, Joint Warfighter Medical Research Program, U.S. Army Medical Research and Development Command
- Study start
- 12 Apr 2018 (actual)
- Primary completion
- 19 Dec 2018 (actual)
- Study completion
- 19 Dec 2018 (actual)
- First posted
- 6 Nov 2017 (actual)
- Results
- Posted 28 Apr 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Mirasol Red Blood Cells (MIR RBCs) | Experimental | Device: 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 |
| Reference Red Blood Cells (REF RBCs) | Active comparator | Device: 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 |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (15 listed)
- Historical RBC transfusion requirement of more than 250 mL/kg/year.
- 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.
- Prior treatment with pathogen-reduced RBCs with subsequent development of known antibodies to the associated RBCs.
- Planned treatment requirement of frozen RBC products.
- Treatment requirements for any medication that is known to cause hemolysis.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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 | Mirasol Red Blood Cells (MIR RBCs) |
| 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. | Device | Reference Red Blood Cells (REF RBCs) |
Primary outcome
Normalized Hemoglobin (Hb AUC) Calculated From Normalized Hb Between Successive Transfusions as a Measure of Percent Surviving RBCs
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.
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.
2 secondary outcomes
Hb Increment
Time frame: Endpoint assessments was evaluated at 15 min Post Transfusion, 1 Day Post Transfusion, 7 Day Post Transfusion, and End of Transfusion Episode.
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
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Feb 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ned Cosgriff, MD study director
Terumo BCT
Steve Sloan, MD, PhD principal investigator
Boston Children's Hospital
9 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Mirasol Red Blood Cells (MIR RBCs) and Reference Red Blood Cells (REF RBCs) in people with Transfusion Dependent Thalassemia, run by Terumo BCTbio at 9 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 14 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States, Israel, Italy, Turkey (Türkiye). Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 28 Apr 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Normalized Hemoglobin (Hb AUC) Calculated From Normalized Hb Between Successive Transfusions as a Measure of Percent Surviving RBCs (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.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Feb 2024.