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

Launched by UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · Dec 4, 2024

Keywords

ClinConnect Summary

This clinical trial is studying whether a type of medication called anti-IL1 therapies, specifically anakinra or canakinumab, can help patients with a rare condition called Fibrodysplasia Ossificans Progressiva (FOP). FOP causes painful flare-ups and abnormal bone growth in places where bones shouldn’t form. The trial aims to see if these medications can reduce flare-ups and slow down the formation of extra bone growth, which can help improve patients' overall well-being. It is specifically looking at patients aged 6 to 17 who have severe symptoms and are already being considered for this type of therapy.

If eligible, participants will be part of an observational study, meaning their response to the medication will be closely watched without any experimental treatment being given. The study will involve various assessments, including blood tests and imaging, to gather important information. Importantly, patients must be able to travel for these assessments and have a medical team that has approved the use of anti-IL1 therapy. This study is a stepping stone to potentially larger trials in the future, helping to gather crucial data on the effectiveness of these treatments for FOP.

Gender

ALL

Eligibility 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 6-17 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