IL1 Inhibition in FOP
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age6-30
SponsorUniversity of California, San Francisco
About this trial
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.
Eligibility criteria
Qualifiers
Patients with a clinical presentation consistent with FOP and a genetic diagnosis of classical FOP (ACVR1R206H variant) (2), male or female aged 6-30 years old.
Patients with unusually severe FOP disease activity. This will be determined by FOP flare frequency of >4 flares per year, which is 2 times higher than the reported average in prior FOP studies ; or by a persistent flare that has failed to resolve after 1 month of standard-of-care therapy.
Patients whose primary medical team has decided that rescue therapy with an anti-IL1 medication should be initiated. Once the primary medical team has decided that anti-IL1 therapy should be pursued, the subject will be told about this clinical-observational study and enrolled in the pre-treatment phase while access to the anti-IL1 therapy is being obtained by the clinical management team.
Ability to participate in all assessments, including blood draws, radiology assessments, and travel. Age 6 is chosen as the lower limit to avoid the need for anesthesia for whole body CT in younger subjects.
Disqualifiers
Pregnant, breastfeeding, or unwilling to practice birth control during participation in the study.
Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the patient or the quality of the data.
Inability to travel to site for assessments
Pre-existing autoimmune or autoinflammatory disease (aside from FOP)
Trial design
Treatments tested in this trial
- Anti-IL1 Therapy
Treatment groups
11 Participants
are divided into 3 treatment groupsSponsors and collaborators
University of California, San Francisco
Lead sponsor
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Collaborator
Source ClinicalTrials.gov