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
This trial does not accept healthy volunteersQualifiers
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 population
Subjects with a diagnosis of FOP who meet the inclusion and exclusion criteria will be eligible for participation in this study.
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
Anti-IL1 Therapy
Other interventionAnti-IL1 is a rescue therapy for FOP patients that is hypothesized to reduce flare activity and subsequent ossification in these patients
Treatment groups
11 Participants
are divided into 3 treatment groupsGroup A: Anti-IL1 Observational Arm1 intervention
Group B: Optional Non-Treatment Observational Arm0 interventions
Group C: "On Treatment" Observational Arm0 interventions
Trial outcomes
Primary outcomes
1
Number of flares that a patient experiences.
Surveys will be used to track the number of clinical flares that a patient experiences before and during treatment with anti-IL1 therapy.
Time frame
1 yearSecondary outcomes
1
Change in new heterotopic ossification bone formation over time
Low dose whole body CT (WBCT) without the head, will be used to create detailed images of the skeletal system. The images will be used to measure new HO bone formation from baseline.
Time frame
1 year2
Changes in blood inflammatory cytokine levels with anti-IL1 therapy
Blood samples will be taken to measure changes in the inflammatory cytokines present in the blood over time, and if this changes with anti-IL1 treatment.
Time frame
1 year3
Change in patient mobility
The Cumulative Analog Joint Involvement Scale (CAJIS) will be used to assess 15 major joints for their mobility. This will be compared from baseline vs. 1 year of anti-IL1 treatment.
Time frame
1 year4
Number of participants with treatment emergent adverse events (TEAEs)
The number of participants with adverse events (ie adverse events not at baseline) will be collected during the treatment period.
Time frame
1 yearSponsors and contacts
Click on the lead sponsor to view all of their trials.
University of California, San Francisco
Lead sponsor
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Collaborator
Source ClinicalTrials.gov