NCT06724562

IL1 Inhibition in FOP

An Observational Study of IL1 Inhibition for Blocking ACVR1-Induced Flare Activity and Heterotopic Ossification in Fibrodysplasia Ossificans Progressiva (FOP)

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 11 (estimated)
• Sponsor: University of California, San Francisco · Academic / Other
• Sex: All
• Age: 6 Years – 30 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

The investigators will perform an observational study on patients with FOP who have decided, along with their primary medical team, to start anti-IL1 therapy with either anakinra or canakinumab due to intractable or unusually severe FOP disease progression. The investigators will study 11 subjects aged 6-30 years old, with a self-reported flare frequency of at least 4 flares/year \[2 times above the average reported FOP population flare frequency of 2 flares/year\] or with an intractable flare that has lasted greater than 1 month. Subjects will begin an observational period during the medication prescription and insurance approval process and will then be followed for up to 1 year after treatment has been initiated by the medical management team. Low-dose whole-body CT (WBCT) imaging, bloodwork, patient-reported outcomes, pain, and flare activity will be assessed during this study. In addition, patients who are currently on anti-IL1 therapy, or are unable to attain anti-IL1 therapy, will be enrolled in a separate observation-only arm to collect historical data related to their experiences on therapy.

Conditions

• Fibrodysplasia Ossificans Progressiva (FOP)

Interventions

Type	Name	Description
OTHER	Anti-IL1 Therapy	Anti-IL1 is a rescue therapy for FOP patients that is hypothesized to reduce flare activity and subsequent ossification in these patients

Timeline

• Start date: 2025-04-01
• Primary completion: 2027-03-01
• Completion: 2027-03-01
• First posted: 2024-12-09
• Last updated: 2026-03-03

Locations

1 site across 1 country: United States