Phase 2 Study of INCB000928 to Test Safety and Effectiveness in People With Fibrodysplasia Ossificans Progressiva

What this Trial Is About

Researchers are evaluating the drug INCB000928 in people diagnosed with fibrodysplasia ossificans progressiva FOP, a rare condition that causes bone to form outside the skeleton. This Phase 2 study is designed to assess how well INCB000928 works, its safety, tolerability, and how the body processes the drug. The study includes a placebo comparison and aims to provide important information on treatment effects in participants with FOP. Participants aged 2 years and older are divided into three age cohorts and randomly assigned to receive either INCB000928 or a placebo daily by mouth for a 24-week double-blind period. Those who complete this initial phase may continue in an open-label extension where all participants receive INCB000928 for an additional 292 weeks. Imaging scans excluding the head are used to monitor changes in bone formation during the study. During the trial, participants will have assessments to detect new bone lesions, measure their volume, and track flare-ups related to FOP at regular intervals up to week 48 and beyond. The study also monitors treatment side effects and the drugs pharmacokinetics at multiple time points. Overall participation can last several years due to the long extension phase, with ongoing safety and efficacy evaluations throughout.

To Assess the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva