[Translation] A Phase II, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of INCB000928 in Subjects with Fibrodysplasia Ossificans Progressiva (PROGRESS)
主要目的为确定INCB000928在抑制FOP受试者发生新异位骨化的疗效。
关键次要目的为评估INCB000928在减少疾病发作和改善疾病发作相关性症状的疗效。
次要目的包括评估INCB000928用于治疗FOP受试者的安全性及耐受性;在第 48 周时,确认INCB000928在双盲期内被随机到安慰剂组的受试者的疗效;表征分析INCB000928的PK;进一步评估INCB000928对FOP受试者的疗效。
[Translation] The primary objective is to determine the efficacy of INCB000928 in inhibiting the occurrence of new heterotopic ossification in FOP subjects.
The key secondary objective is to evaluate the efficacy of INCB000928 in reducing disease attacks and improving symptoms associated with disease attacks.
Secondary objectives include evaluating the safety and tolerability of INCB000928 for the treatment of FOP subjects; confirming the efficacy of INCB000928 in subjects randomized to the placebo group during the double-blind period at week 48; characterizing the PK of INCB000928; and further evaluating the efficacy of INCB000928 in FOP subjects.