The purpose of this study is to determine the safety and clinical effects of SCD-101 when given to adults with sickle cell disease.
This is single site, dose- escalation study of SCD-101 in participants with homozygous sickle cell disease (S/S) or S/beta 0 Thalassemia. All participants will be monitored for safety, tolerability, and dose-limiting toxicities. The study is divided into two parts. Part A is an open-label, non-randomized, non-placebo-controlled dose escalation study with a 28-day treatment phase and 14-day follow-up phase with five cohorts . Part B is a randomized, placebo-controlled, confirmatory 2x2 crossover cohort with a 28 day washout between periods, and a 28-day follow-up phase.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
60
Administered as gelatin capsules
King's County Hospital
Brooklyn, New York, United States
Determine the safety, tolerability, and dose limiting toxicities of escalating doses of SCD-101, assessed by frequency and severity of adverse events (AEs), and changes in vital signs, 12-lead ECGs and laboratory assessments as compared to baseline
Time frame: From the time the participant is administered the first dose through the final follow-up (18 weeks)
Determine the effect of escalating doses of SCD-101 on the mean change in hemoglobin form base line
Time frame: From the time the participant is accessed at baseline through the final follow-up (18 weeks)
Determine the effect of escalating doses of SCD-101 on the mean change in percent reticulocytes from baseline
Time frame: From the time the participant is accessed at baseline through the final follow-up (18 weeks)
Determine the effect of escalating doses of SCD-101 on the mean change from baseline in red blood cell hemolysis as measured by lactate dehydrogenase (LDH) and indirect bilirubin.
Time frame: From the time the participant is accessed at baseline through the final follow-up (18 weeks)
Determine the effect of escalating doses of SCD-101 on the mean change from baseline in fatigue as measured by the PROMIS fatigue questionnaire
Time frame: From the time the participant is accessed at baseline through the final follow-up (18 weeks)
Determine the effect of escalating doses of SCD-101 on the mean change from baseline in the percent of venous circulating sickle red blood cells
Time frame: From the time the participant is accessed at baseline through the final follow-up (18 weeks)
Determine the effect of escalating doses of SCD-101 on the mean change from baseline in functional capacity as measured by the 6-Minute Walk Test
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Time frame: From the time the participant is accessed at baseline through the final follow-up (18 weeks)