Recruiting

Anti-IL1 Therapies

Sponsor:

UCLA

Code:

NCT06724562

Conditions

Fibrodysplasia Ossificans Progressiva (FOP)

Eligibility Criteria

Sex: All

Age: 6 - 30

Healthy Volunteers: Not accepted

Interventions

Anti-IL1 Therapy

Study Details

Brief summary:

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.

Conditions

Fibrodysplasia Ossificans Progressiva (FOP)

Study ID

NCT06724562

Start date

Apr 1, 2025

Status verified date

Aug, 2026

Completion date

Nov 5, 2027

Anticipated

Primary completion date

Nov 5, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 6 - 30

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

11 participants

Anticipated

Interventions and Outcome Measures

Arms

Anti-IL1 Observational Arm

FOP patients that are beginning treatment with Anti-IL1 Therapy

Optional Non-Treatment Observational Arm

FOP patients unable to obtain Anti-IL1 therapy

"On Treatment" Observational Arm

FOP patients that are already using Anti-IL1 therapy

Interventions

Anti-IL1 Therapy

Anti-IL1 is a rescue therapy for FOP patients that is hypothesized to reduce flare activity and subsequent ossification in these patients

Primary outcome measure

  • Number of flares that a patient experiences. [ Time Frame: 1 year ]

Central Contacts and Locations

Central contacts

Samantha Klein

415-254-5748Samantha.klein@ucsf.edu

Judy Gonzalez-Vargas

415-254-5048Judy.Gonzalez-Vargas@ucsf.edu

Locations

UCSF

Recruiting

San Francisco, California, United States, 94143

Contacts

Edward Hsiao, MD, PhD

415-476-9732edward.hsiao@ucsf.edu

More Information

Sponsor

University of California, San Francisco

Last update posted

Aug 6, 2026

Last verified

Aug, 2026

Keywords

  • heterotopic ossification
  • canakinumab
  • anakinra
  • FOP

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by University of California, San Francisco on 2026-08-06.