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
Purpose
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.
Condition
- Fibrodysplasia Ossificans Progressiva (FOP)
Eligibility
- Eligible Ages
- Between 6 Years and 17 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Patients with a clinical presentation consistent with FOP and a genetic diagnosis of classical FOP (ACVR1R206H variant) (2), male or female aged 6-17 years old. - Patients with unusually severe FOP disease activity. This will be determined by FOP flare frequency of >6 flares per year, which is 3 times higher than the reported average in prior FOP studies ; or by a persistent flare that has failed to resolve after 3 months 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. - No history of unexplained infections, known autoimmune disease, or contraindication to anti-IL1 therapy. - Written informed consent (and assent when applicable) obtained from subject or subject's legal representative and ability for subject to comply with the requirements of the study.
Exclusion Criteria
- 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) - Inability to tolerate assessments (such as phlebotomy) - Unexplained infections - Current participation in an interventional trial, or study of a potentially disease modifying medication - Inability to take medications as prescribed by managing physician
Study Design
- Phase
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
| Anti-IL1 Observational Arm | FOP patients that are treated with Anti-IL1 Therapy |
|
| Optional Non-Treatment Observational Arm | FOP patients unable to obtain Anti-IL1 therapy |
|
Recruiting Locations
San Francisco, California 94143
More Details
- NCT ID
- NCT06724562
- Status
- Recruiting
- Sponsor
- University of California, San Francisco
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 6-17 years old, with a self-reported flare frequency of at least 6 flares/year [3 times above the average reported FOP population flare frequency of 2 flares/year] or with an intractable flare that has lasted greater than 3 months. 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.