7 results
Comparison of steady-state cysteamine-trough WBC cystine levels between Cystagon® and RP103 over 3 months for each treatment period.
To characterize the safety, tolerability, and recommended phase 2 dose (RP2D) of each combination partner used with ruxolitinib (Part 1)To evaluate the preliminary efficacy of each novel ruxolitinib combination treatment arm (Parts 2 & 3)
To compare the efficacy of 7.5 mg/kg of crizanlizumab versus placebo on the annualized rate of VOC leading to healthcare visit, in addition to standard of careTo compare the efficacy of 5.0 mg/kg of crizanlizumab versus placebo on the annualized…
- To assess the pharmacokinetic profile of cysteamine base after single dose administration of the new sustained-release cysteamine bitartrate (PO-001) to healthy volunteers, in comparison with Cystagon® and Procysbi®.- To collect safety/…
Primary:To assess the safety and tolerability of DS-1211b compared with placebo in subjects with PXE.To assess the dose response by assessing the treatment changes in PD endpoints.
To evaluate the effect of crizanlizumab + standard of care compared to standard of care alone on albuminuria (ACR) decrease at 12 months
To study the effect of crizanlizumab on the hemodynamics of the cerebral vasculature (CBF and CVR)