Observing the effects of anti-IL1 therapies on flare activity in FOP patients

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• 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

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Evaluating INCB000928 for treating Fibrodysplasia Ossificans Progressiva

  This study is testing a new drug called INCB000928 to see if it can help people with Fibrodysplasia Ossificans Progressiva manage their symptoms better.

  Recruiting at 11 sites Phase 2 Incyte Corporation · Rochester, Minnesota and 23 other locations · NCT05090891

• Global registry for patients with Fibrodysplasia Ossificans Progressiva

  This study is creating a global registry to collect information from people with Fibrodysplasia Ossificans Progressiva and their caregivers to help improve understanding and treatment of the condition.

  Recruiting The International FOP Association · North Kansas City, Missouri · NCT02745158

• Documenting long-term safety and effectiveness of palovarotene in patients with fibrodysplasia ossificans progressiva

  This study is trying to see how safe and effective the medication palovarotene is for children and adults with fibrodysplasia ossificans progressiva who are already using it.

  Recruiting at 4 sites Ipsen · San Francisco, California and 4 other locations · NCT06089616

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.