Transfusion Dependent Thalassemia Clinical Trial
— PRAISEOfficial title:
Evaluate the Efficacy and Safety of RBCs Derived From Mirasol-treated Whole Blood Compared With Conventional RBCs in Patients Requiring Chronic Transfusion Support
| Verified date | January 2024 |
| Source | Terumo BCTbio |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
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.
| Status | Terminated |
| Enrollment | 9 |
| Est. completion date | December 19, 2018 |
| Est. primary completion date | December 19, 2018 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 12 Years and older |
| Eligibility | 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. |
| Country | Name | City | State |
|---|---|---|---|
| Israel | Rambam Health Care Campus | Haifa | |
| Israel | Hadassah Ein Kerem Hospital | Jerusalem | |
| Italy | 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 | |
| Turkey | Ege University Children's Hospital | Bornova | Izmir |
| United States | Boston Children's Hospital | Boston | Massachusetts |
| United States | Weill-Cornell Medical College | New York | New York |
| United States | UCSF Benioff Children's Hospital Oakland | Oakland | California |
| United States | The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania |
| Lead Sponsor | Collaborator |
|---|---|
| Terumo BCTbio | Joint Warfighter Medical Research Program, U.S. Army Medical Research and Development Command, United States Department of Defense |
United States, Israel, Italy, Turkey,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Other | Percentage Decline in Post-transfusion Hb Level | Percentage decline in post-transfusion Hb level | 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. | |
| Other | RBC Mass Infused | volume x Hb/unit | 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 | |
| Other | Number of Antibody Screening Test With Confirmed Specificity to RBCs Derived From Mirasol-treated WB | Antibody screening was performed in a total of 8 (100%) subjects in the SS at a total of 97 intervals (Pre-Transfusion, 7 Days Post-Transfusion or End of Study Treatment Follow-up Visit). | 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 | |
| Other | Number of Participants With Human Leukocyte Antigen (HLA) Alloimmunization Post Transfusion | The highest of three different normalized background ratio (NBG) cut-offs was used to quantify positivity for Class I HLA antibodies prior to transfusion as it was used to identify conversion from HLA antibody negative prior to transfusion to positivity after transfusion(s) within the applicable treatment group. | 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. | |
| Primary | 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. | 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 | Hb Increment | (post-transfusion Hb - pre-transfusion Hb)/Hb transfused]/RBC volume in subject at pre-transfusion | Endpoint assessments was evaluated at 15 min Post Transfusion, 1 Day Post Transfusion, 7 Day Post Transfusion, and End of Transfusion Episode. | |
| Secondary | Actual Hb Level Post-transfusion (15 Min) | Actual Hb level post-transfusion (15 min) | 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 |