What is anti-IL1 therapy studying for Fibrodysplasia Ossificans Progressiva (FOP)?

Anti-IL1 therapy is being studied to see if it can reduce flare activity and abnormal bone growth in patients with severe FOP. This observational study aims to gather important data on how this treatment affects disease progression and symptoms. The University of California, San Francisco is leading this research to help inform future treatment studies.

Who may be eligible for this Fibrodysplasia Ossificans Progressiva (FOP) clinical trial?

Participants must be aged 6 to 30 years with a diagnosis of classical FOP caused by the ACVR1R206H variant. They should have unusually severe disease, such as more than 4 flares per year or a persistent flare lasting over one month, and their medical team must have recommended anti-IL1 therapy. Please review eligibility criteria for more details.

Is there any cost to participate in this Fibrodysplasia Ossificans Progressiva (FOP) clinical trial?

Study-related care, including the study treatment and all trial visits, is provided at no cost. However, personal expenses like travel costs may vary and are not guaranteed to be covered. Participants should plan accordingly for any additional expenses.

Actively Recruiting

Age: 6Years - 30Years

All Genders

ID06724562

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

Led by University of California, San Francisco · Updated on 2026-03-03

Participants Needed

Research Sites

N/A

Total Duration

AI-Summary

What this Trial Is About

Researchers are conducting an observational study to evaluate the off-label use of anti-IL1 therapies, such as anakinra or canakinumab, in patients with severe Fibrodysplasia Ossificans Progressiva FOP. The study aims to observe whether these therapies can reduce ACVR1-induced flare activity and heterotopic ossification. It also seeks to gather important preliminary data and tools for designing a future Phase II study. Participants are individuals with unusually severe FOP disease progression or frequent flares. The study involves 11 subjects aged 6 to 30 years who either are starting anti-IL1 therapy under their medical teams guidance, are currently on anti-IL1 therapy, or are unable to obtain this treatment. Participants will be observed during the medication prescription and insurance approval process, then followed for up to one year after treatment begins. Assessments include low-dose whole-body CT imaging, blood tests, patient-reported outcomes, flare activity, and pain. A separate observation-only group will collect data from those already on or unable to access anti-IL1 therapy. Participants will undergo periodic evaluations including imaging and bloodwork, along with reporting on flare frequency, pain levels, mobility, and medication use. The primary outcome is the number of flares experienced over one year, with secondary outcomes tracking new bone formation, changes in inflammatory markers, mobility, and treatment-related adverse events. The study lasts up to one year post-treatment initiation, providing comprehensive monitoring of disease activity and therapy effects.

CONDITIONS

Brief Title

IL1 Inhibition in FOP

Who Can Participate

Age: 6Years - 30Years

All Genders

Eligibility Criteria

You may qualify if you...

Patients aged 6 to 30 years with classical FOP diagnosed by the ACVR1R206H genetic variant
Patients experiencing unusually severe FOP, defined as more than 4 flares per year or a flare lasting longer than 1 month
Patients whose medical team has decided to start anti-IL1 therapy as rescue treatment
Ability to participate in all study assessments, including blood draws, radiology, and travel
No history of unexplained infections, autoimmune diseases, or contraindications to anti-IL1 therapy
Written informed consent and ability to comply with study requirements

You will not qualify if you...

Pregnant or breastfeeding individuals, or those unwilling to use birth control during the study
Conditions or abnormalities that may compromise patient safety or data quality
Inability to travel to study site for assessments
Pre-existing autoimmune or autoinflammatory diseases other than FOP
Inability to tolerate study assessments such as blood draws
History of unexplained infections
Current participation in other interventional trials or studies with disease-modifying medications
Inability to take prescribed medications as directed by the managing physician

Research Team

Samantha Klein

Judy Gonzalez-Vargas

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