Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02187003 | Efficacy and Safety of Rivipansel (GMI-1070) in the Treatment of Vaso-Occlusive Crisis in Hospitalized Subjects With Sickle Cell Disease | PHASE3 | COMPLETED | 345 | — | — | Jun 17, 2015 | Jun 27, 2019 | Mar 11, 2025 | 142 | United States, Canada |
Time to readiness-for-discharge from hospital was defined as the difference (in hours) between the time and date when all criteria for readiness-for-discharge were met and the start time and date of the first infusion (loading dose) of study drug. Criteria for readiness-for-discharge were met when all of the applicable 6 criteria (in relation to treatment of VOC and complications related to the VOC) were documented to have occurred. The six criteria were: 1) only oral pain medication was required, 2) acute complications related to the VOC (such as acute chest syndrome, stroke, priapism) had resolved to the extent that management could be in an outpatient setting, 3) IV opioids had been discontinued, 4) IV hydration had been discontinued, 5) IV antibiotics had been discontinued and 6) red blood cell (RBC) transfusion was no longer required for treatment of this VOC.
| Arm | Type | Description |
|---|---|---|
| Rivipansel Treatment Arm | EXPERIMENTAL | - |
| Placebo Treatment Arm | PLACEBO_COMPARATOR | - |
| Name | Type | Description |
|---|---|---|
| Rivipansel | DRUG | Rivipansel (GMI-1070) will be infused intravenously every 12 hours up to 15 doses maximum. Subjects aged 12 and over who weigh more than 40 kilograms will receive a dose of 1680 mg of rivipansel, followed by a dose of 840 mg of rivipansel every 12 hours. All subjects aged 6 to 11 years and any subject who weighs 40 kilograms or less, will receive weight-based dosing (mg/kg) of 40 mg/kg of rivipansel (maximum of 1680 mg) followed by a dose of 20 mg/kg of rivipansel (maximum of 840 mg) every 12 hours. |
| Placebo | OTHER | Placebo (phosphate buffered saline) will be infused intravenously every 12 hours up to 15 doses maximum. |
Inclusion Criteria: * At least 6 years of age. * Documented diagnosis of sickle cell disease. * Diagnosis of vaso-occlusive crisis necessitating admission to the hospital with treatment including IV opioids. * Able to receive the first dose of study drug within 24 hours from the administration of I...