Safety and effect of SANGUINATE on Sickle Cell Disease patients experiencing a vaso-occlusive crisis
Patients who are experiencing a vaso-occlusive crisis will report to the clinic for treatment and screening into the SGSC-005 study. Patients who meet all inclusion/exclusion criteria will be randomized to receive either SANGUINATE 320 mg/kg or placebo (saline) over a 2 hour infusion period. Assessments of vital signs, ECGs, safety labs, adverse events as well as patient and physician questionnaires will be completed up to 6 hours after the start of the infusion. Patients will then be assessed for discharge either to home or admitted to the hospital for further treatment depending on their disease severity. Follow-up phone calls will be completed 24 hours and 7 days after initiation of treatment . A follow-up visit will be completed in the clinic at 72 hours after initiation of treatment.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Enrollment
24
Single two-hour infusion of SANGUINATE
Single two-hour infusion of placebo
FSCDR
Hollywood, Florida, United States
Florida Health Tampa General Hospital
Tampa, Florida, United States
University of Maryland School of Medicine
Baltimore, Maryland, United States
Time to readiness for discharge from ambulatory site
Defined as the time from the start of study drug infusion until the time of participant's response that their pain episode has improved enough for discharge; investigator's assessment of participant's readiness for discharge; and participant no longer requires IV opioid administration
Time frame: 7 Days
Safety of treatment
as determined by changes in vital signs, EKG, biochemical, hematological, and urinalysis measures, and reported AEs
Time frame: 1 Day
Extent of reduction in pain score during ambulatory visit as assessed by the participant on a 10-point pain scale
Time frame: 1 Day
Total pain treatment received including the opioid dose (mg/kg) received during ambulatory visit
Time frame: 1 Day
Global assessment of the participant's improvement by the Investigator or study staff (at the time of readiness for discharge from the ambulatory visit, and at the 72-hour follow-up visit)
Time frame: 7 Days
Reduction in the rate of recurrent ambulatory visit(s) for the VOC within 7 days post discharge
Time frame: 7 Days
Reduction in the rate of hospitalization(s) for VOC after treatment with SANGUINATE and within 7 days post-discharge
Time frame: 7 Days
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