4 results
The objectives of the study are to:* Identify a dose and dosing regimen of KRN23, based on safety and PD effect, in pediatric XLH patients* Establish the safety profile of KRN23 for the treatment of children with XLH including ectopic mineralization…
Primary Objective:The primary safety objective of the study is to assess the safety and tolerability of REGN2477 in male and female patients with fibrodysplasia ossificans progressiva (FOP).The primary efficacy objective of the study is to assess…
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 evaluate the effect of crizanlizumab + standard of care compared to standard of care alone on albuminuria (ACR) decrease at 12 months