This is a clinical study evaluating the efficacy and safety of rivipansel (GMI-1070) in treating subjects with sickle cell disease (SCD) who are 6 years of age or older experiencing a pain crisis necessitating hospitalization.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
345
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 (phosphate buffered saline) will be infused intravenously every 12 hours up to 15 doses maximum.
Time to Readiness for Discharge From Hospital
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.
Time frame: Day 1 up to the latest day when all 6 criteria of readiness-for-discharge were met (up to an average of Day 8)
Time to Discharge From Hospital
Time to discharge from hospital was defined as the difference (in hours) between the time and date of the hospital discharge order from a qualified healthcare provider and the start time and date of the first infusion (loading dose) of study drug.
Time frame: Day 1 up to the latest day when the order of hospital discharge was issued by a qualified healthcare provider (up to an average of Day 8)
Cumulative Intravenous (IV) Opioids Consumption From Time of Loading Dose of Study Drug to Discharge From Hospital
Cumulative IV opioid consumption was reported as cumulative IV opioid use (standardized using morphine equivalent units \[MEU\]), from the start of the first infusion (loading dose) of study drug until hospital discharge.
Time frame: Day 1 up to the latest day when IV opioid was discontinued (up to an average of Day 8)
Time to Discontinuation of Intravenous (IV) Opioids
Time to discontinuation of IV opioids was defined as the difference (in hours) between the stop time and date of the latest IV opioid dose and the start time and date of the first infusion (loading dose) of study drug.
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University of South Alabama Children's and Women's Hospital
Mobile, Alabama, United States
Arkansas Children's Hospital Research Pharmacy
Little Rock, Arkansas, United States
Arkansas Children's Hospital
Little Rock, Arkansas, United States
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States
UC Davis Medical Center Main Hospital
Sacramento, California, United States
UC Davis Medical Center
Sacramento, California, United States
University of California Davis Medical Center
Sacramento, California, United States
University of Colorado CTRC
Aurora, Colorado, United States
University of Colorado Hospital
Aurora, Colorado, United States
Howard University Center for Sickle Cell Disease
Washington D.C., District of Columbia, United States
...and 132 more locations
Time frame: Day 1 up to the latest day when IV opioid was discontinued (up to an average of Day 8)
Cumulative Intravenous (IV) Opioids Consumption Within First 24 Hours Post-Loading Dose of Study Drug
Cumulative IV opioid consumption (standardized using MEU) was reported as IV opioid use in the first 24 hours from the start time of the first IV infusion (loading dose) of study drug.
Time frame: 24 hours post first IV infusion of loading dose of study drug on Day 1
Percentage of Participants Re-hospitalized for Vaso-Occlusive Crisis (VOC) Within 3 Days of Discharge From Hospital
Percentage of participants who were re-hospitalized for a vaso-occlusive crisis (VOC) within 3 days of discharge from hospital are reported.
Time frame: Within 3 days of discharge from hospital, where discharge from hospital was any day from Day 1 to an average of Day 8