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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

Study Purpose

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.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

 No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

 Observational
Eligible Ages 6 Years - 17 Years
Gender All
More 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 6-17 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

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

 NCT06724562
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

 University of California, San Francisco
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

 Edward Hsiao, MD, PhD
Principal Investigator Affiliation University of California, San Francisco
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

 Other, NIH
Overall Status Recruiting
Countries United States
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

 Fibrodysplasia Ossificans Progressiva (FOP)
Additional Details

The investigators will perform an observational study on patients with FOP who have decided, along with their primary medical team, to start anti-IL1 therapy with either anakinra or canakinumab due to intractable or unusually severe FOP disease progression. The investigators will study 11 subjects aged 6-17 years old, with a self-reported flare frequency of at least 6 flares/year [3 times above the average reported FOP population flare frequency of 2 flares/year] or with an intractable flare that has lasted greater than 3 months. Subjects will begin an observational period during the medication prescription and insurance approval process and will then be followed for up to 1 year after treatment has been initiated by the medical management team. Low-dose whole-body CT (WBCT) imaging, bloodwork, patient-reported outcomes, pain, and flare activity will be assessed during this study.

Arms & Interventions

Arms

: Anti-IL1 Observational Arm

FOP patients that are treated with Anti-IL1 Therapy

: Optional Non-Treatment Observational Arm

FOP patients unable to obtain Anti-IL1 therapy

Interventions

Other: - 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

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

UCSF, San Francisco, California

Status

Recruiting

Address

UCSF

San Francisco, California, 94143

Site Contact

Edward Hsiao, MD, PhD

[email protected]

415-476-9732

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