ICA-17043 is being developed for the chronic treatment of patients with sickle cell disease (SCD) in both adults and children. ICA-17043 is a potent and specific inhibitor of a channel in human red blood cells (RBCs) that blocks RBC dehydration. ICA-17043 is expected to inhibit RBC dehydration and thus should prevent or delay the sickling process. By reducing sickled cells, an improvement in anemia, a reduction in painful crises, and ultimately, less end-organ disease is anticipated.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
90
Low dose arm
150 mg Loading Dose; 10 mg daily dose
Placebo Loading dose capsules and maintenance dose tablets matched 10 mg active treatment group
Study Site
Birmingham, Alabama, United States
Study Site
Oakland, California, United States
The primary efficacy endpoint was the change from Baseline in hemoglobin (Hb)
Time frame: 12 Weeks
Changes in other hematologic measurements
Time frame: 12 weeks
Changes in RBC indices, including: mean corpuscular volume (MCV), mean corpuscular Hb concentration (MCHC), and mean corpuscular Hb (MCH
Time frame: 12 weeks
Other laboratory measures associated with sickle cell crises activity including: direct and indirect bilirubin and lactic dehydrogenase (LDH)
Time frame: 12 weeks
Rate of painful crises
Time frame: 12 weeks
Time to first painful crisis
Time frame: 12 weeks
Morbidity of painful crises (maximum morbidity index, derived variable)
Time frame: 12 weeks
Pain intensity scores
Time frame: 12 weeks
Quality of Life (SF 36)
Time frame: 12 Weeks
Health economic data
Time frame: 12 weeks
Average plasma concentration
Time frame: 12 weeks
Correlation between the average plasma concentration and the change in Hb from Baseline to study endpoint
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Study Site
San Francisco, California, United States
Study Site
Washington D.C., District of Columbia, United States
Study Site
Augusta, Georgia, United States
Study Site
Chicago, Illinois, United States
Study Site
Baltimore, Maryland, United States
Study Site
Boston, Massachusetts, United States
Study Site
Detroit, Michigan, United States
Study Site
Jackson, Mississippi, United States
...and 9 more locations
Time frame: 12 weeks