Observing the effects of anti-IL1 therapies on flare activity in FOP patients
An Observational Study of IL1 Inhibition for Blocking ACVR1-Induced Flare Activity and Heterotopic Ossification in Fibrodysplasia Ossificans Progressiva (FOP)
This study is testing if using certain anti-inflammatory medications can help kids and teens with Fibrodysplasia Ossificans Progressiva manage their flare-ups and bone growth issues.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 11 (estimated) |
| Ages | 6 Years to 30 Years |
| Sex | All |
| Sponsor | University of California, San Francisco Academic / other |
| Drugs / interventions | canakinumab |
| Locations | 1 site (San Francisco, California) |
| Trial ID | NCT06724562 on ClinicalTrials.gov |
What this trial studies
This observational study aims to assess the impact of off-label anti-IL1 therapies, such as anakinra and canakinumab, on flare activity and heterotopic ossification in patients with severe Fibrodysplasia Ossificans Progressiva (FOP). The study will involve 11 subjects aged 6-17 years who experience frequent flares or persistent severe flares. Participants will be monitored for up to one year after starting treatment, with evaluations including imaging, bloodwork, and patient-reported outcomes to gather preliminary data for future studies.
Who should consider this trial
Good fit: Ideal candidates are patients aged 6-17 years with a genetic diagnosis of classical FOP and unusually severe disease activity.
Not a fit: Patients with mild FOP symptoms or those not requiring anti-IL1 therapy may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to improved management of flare activity and reduced heterotopic ossification in patients with FOP.
How similar studies have performed: Preliminary data from other studies suggest potential benefits of anti-IL1 therapies in reducing flare frequency, but this specific approach is novel.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Patients with a clinical presentation consistent with FOP and a genetic diagnosis of classical FOP (ACVR1R206H variant) (2), male or female aged 6-30 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. * 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
Where this trial is running
San Francisco, California
- UCSF — San Francisco, California, United States (Recruiting)
Study contacts
- Principal investigator: Edward Hsiao, MD, PhD — University of California, San Francisco
- Study coordinator: Samantha Klein
- Email: Samantha.klein@ucsf.edu
- Phone: 415-254-5748
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.