The objective of this study is to assess the efficacy of SC411 in reducing the number of sickle cell crisis (SCC) events in sickle cell disease (SCD) subjects receiving SC411 compared to those subjects receiving placebo.
This Phase 3, prospective, randomized, double-blinded, placebo-controlled, multi-center study will enroll approximately 210 subjects at up to 70 sites in the United States. Participation will consist of a Screening Period, followed by a minimum 12-month Treatment Period. SC411 is administered orally as a soft gel mini capsule. This study will enroll subjects aged ≥5 to ≤17 years who have a diagnosis of SCD that includes the phenotypes hemoglobin SS homozygous (HbSS), hemoglobin SC (HbSC), and hemoglobin S/β°-thalassemia (HbS/ β°-thalassemia); and have had at least 2 but no more than 10 documented SCC events (as defined above) within 12 months prior to the Screening Visit.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
210
Assessment of the efficacy of SC411 in reducing the number of sickle cell crisis (SCC) events in subjects compared to placebo will be measured by counting the number of sickle cell crises that occur after randomization.
Assessment of the efficacy of SC411 in reducing the number of sickle cell crisis (SCC) events in subjects compared to placebo will be measured by counting the number of sickle cell crises that occur after randomization. The primary objective of this study is to assess the efficacy of orally administered SC411 in reducing the number of sickle cell crisis (SCC) events in sickle cell disease (SCD) subjects compared to placebo. A SCC event will be defined as either an acute painful crisis or an acute chest syndrome.
Time frame: 52 weeks
Evaluation of the effect of SC411 compared to placebo by measuring the time until the patient's first sickle cell event.
Evaluation of days to the first event from randomization for each patient.
Time frame: 52 weeks
Evaluation of the effect of SC411 compared to placebo by measuring the the number of visits to a medical facility (hospital, clinic, or emergency room) for SCC event or complications of SCD.
Evaluation of the effect of SC411 compared to placebo by measuring the the number of visits to a medical facility (hospital, clinic, or emergency room) for SCC event or complications of SCD.
Time frame: 52 weeks
Evaluation of the effect of SC411 compared to placebo by measuring the number of days with electronic diary (eDiary)-recorded opioid or non opioid analgesic use at home to manage sickle cell pain.
To evaluate the effect of SC411 compared to placebo by measuring the number of days with electronic diary (eDiary)-recorded opioid or non opioid analgesic use at home to manage sickle cell pain.
Time frame: 52 weeks
Evaluation of the effect of SC411 compared to placebo by measuring the number of crisis-free days.
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To evaluate the effect of SC411 compared to placebo by measuring the number of crisis-free days. A crisis-free day is defined as any day with a zero entry on the eDiary pain intensity scale.
Time frame: 52 weeks