RECRUITING

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

Conditions

Fibrodysplasia Ossificans Progressiva (FOP)heterotopic ossification canakinumab anakinra FOP

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

An Observational Study of IL1 Inhibition for Blocking ACVR1-Induced Flare Activity and Heterotopic Ossification in Fibrodysplasia Ossificans Progressiva (FOP)

Quick Facts

Study Start:2025-04

Study Completion:2027-03

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06724562

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:6 Years to 17 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD

Inclusion Criteria	Exclusion 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.	* 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

Inclusion Criteria

Exclusion 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.

* 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

Contacts and Locations

Study Contact

Samantha Klein

CONTACT

415-254-5748

Samantha.klein@ucsf.edu

Judy Gonzalez-Vargas

CONTACT

415-254-5048

Judy.Gonzalez-Vargas@ucsf.edu

Principal Investigator

Edward Hsiao, MD, PhD

PRINCIPAL_INVESTIGATOR

University of California, San Francisco

Study Locations (Sites)

UCSF

San Francisco, California, 94143

United States

Collaborators and Investigators

Sponsor: University of California, San Francisco

Edward Hsiao, MD, PhD, PRINCIPAL_INVESTIGATOR, University of California, San Francisco

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-04

Study Completion Date2027-03

Study Record Updates

Study Start Date2025-04

Study Completion Date2027-03

Terms related to this study

Keywords Provided by Researchers

heterotopic ossification
canakinumab
anakinra
FOP

Additional Relevant MeSH Terms

Fibrodysplasia Ossificans Progressiva (FOP)