Table 8.
Clinical experience
| Study | Number | Description | Endpoints | Results |
|---|---|---|---|---|
| A. With the S‐303 and GSH PI‐RBC system | ||||
| First generation | ||||
| US Phase III chronic study | 50 | Transfusion‐dependent SCD patients; two‐arm double‐blinded crossover design | Blood utilization | Terminated |
| US Phase III acute study124 | 148 | CV surgery patients | Composite endpoint of MI, renal failure, and mortality124 | Met primary endpoint, early termination |
| Second generation | ||||
| US Phase II study 122 | 27 | Healthy volunteers; crossover design | 24‐hour recovery: 88.0 ± 8.5 days (T) vs. 90.1 ± 6.9 (C) | Met primary endpoint, completed |
| EU Phase III acute study125 | 50 | CV surgery patients; two‐arm design |
Primary efficacy: mean Hb content per RBC component Primary safety: adverse events over 90 days (related and unrelated to study RBC components) compared between the treatment groups |
Met primary endpoint with similar AE profile between arms126 |
| EU Phase III chronic study127 | 70 | Transfusion‐dependent thalassemia Major patients; crossover design |
Primary efficacy: Hb consumption (g Hb/kg body weight/day). Primary safety: incidence of a treatment‐emergent antibody with confirmed specificity to S‐303 RBCs over 12 months |
In progress |
| B. With the riboflavin and UV PI‐WB system | ||||
| US Phase II Study–IMPROVE123, 128 |
12 (4/4/3) |
Feasibility trial to evaluate recovery and survival in RBCs obtained from WB units treated with the Mirasol system. Three study arms each using a different UV dose (22, 33, and 44 J/mLRBC) |
Primary: 24‐hr posttransfusion RBC recovery Secondary: RBC survival; SAE |
Terminated Recovery ≥ 75% 22‐J dose: 1 of 4 33‐J dose: 1 of 4 44‐J dose: 1 of 3 Survival ≥ 28 days 22‐J dose: 2 of 4 33‐J dose: 2 of 4 44‐J dose: 0 of 4 |
| US Phase II study–IMPROVE II1 29 | 29 | To evaluate, as per FDA criteria, the 24‐hr posttransfusion RBC recovery in healthy adult subjects of LR‐RBCs, derived from Mirasol‐treated fresh WB units, and stored refrigerated for 21 days. |
Primary: 24‐hr posttransfusion RBC recovery Secondary: RBC survival, AUC; SAE; neoantigenicity |
Completed Data not yet reported |
| Ghana Phase‐III Study–AIMS1 30 | 250 | Treatment of WB with the Mirasol system: prevention of Malaria caused by transfusion |
Primary: TT malaria Secondary: TT bacterial infections |
Completed |
AUC = area under curve; CV = cardiovascular; MI = myocardial infarction; SAE = severe adverse event.