Clinical Research Directory

An Expanded Access Program of Garetosmab in Adult Patients With Fibrodysplasia Ossificans Progressiva (FOP)

The objective of this Expanded Access Program (EAP) is to provide garetosmab to adult patients with Fibrodysplasia Ossificans Progressiva (FOP), including patients who have completed the double-blind treatment period of the parent study, OPTIMA (R2477-FOP-2175 \[NCT05394116\]), prior to marketing authorization approval.

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

This Phase 2, Randomized, Double-Blind, Placebo-Controlled Study is intended to evaluate the Efficacy, Safety, and Tolerability and PK of INCB000928 administered to participants with a clinical diagnosis of fibrodysplasia ossificans progressiva (FOP).

IL1 Inhibition in FOP

This is an observational pre-post study to observe if the off label use of anti-IL1 therapies, such as anakinra or canakinumab, can block ACVR1-induced flare activity and heterotopic ossification in FOP. It will also generate key tools and preliminary data that are needed to design a future Phase II study. This study specifically focuses on patients with severe FOP who are being considered by their medical team for rescue therapy with anti-IL1 therapy. Preliminary data suggests patients experience significant decreases in flare frequency when taking anti-IL1 therapy, but other measures of efficacy remain unassessed, such as changes in heterotopic ossification formation, changes in pain medication use, and changes in functionality.

The Fibrodysplasia Ossificans Progressiva (FOP) Registry

The Fibrodysplasia Ossificans Progressiva (FOP) Registry is a global, non-interventional, voluntary database that captures demographic and disease data directly from FOP patients and their caregivers via a secure, web-based patient portal. A physician portal (in development) will allow physicians to enter clinical data about their patients. The objectives are to organize the international FOP community for participation in clinical trials; to enable FOP patients worldwide to report data in a shared forum; to improve the collective understanding of FOP natural history; and to advance the understanding of FOP treatment outcomes.