Anti-IL1 Therapy for Stone Man Syndrome
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to determine if anti-IL1 therapies, such as anakinra or canakinumab, can reduce flare-ups and abnormal bone growth in individuals with Fibrodysplasia Ossificans Progressiva (FOP), a rare bone condition. The trial examines the impact of these treatments on flare frequency, bone growth, pain medication use, and daily functionality. Participants should have severe FOP, with frequent or unresolved flare-ups, and their doctors should have recommended anti-IL1 therapy. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, providing an opportunity to contribute to early evidence of its efficacy.
Do I have to stop taking my current medications for the trial?
The trial information does not specify if you need to stop taking your current medications. However, it mentions that you cannot participate if you are currently in another trial or taking a potentially disease-modifying medication.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research has shown that treatments like anakinra or canakinumab are usually safe for patients. In earlier studies, participants using these treatments experienced fewer flare-ups, periods when symptoms worsen, and showed little new unwanted bone growth. While these results suggest the treatments are generally safe, long-term side effects remain under investigation. Prospective trial participants should discuss potential risks and benefits with their doctor.12345
Why are researchers excited about this trial?
Researchers are excited about Anti-IL1 therapies, like Anakinra and Canakinumab, because they offer a novel approach to treating Fibrodysplasia Ossificans Progressiva (FOP) by targeting the inflammatory pathway. Unlike standard treatments that mainly focus on symptom management, these therapies aim to block the ACVR1-induced flare activity and heterotopic ossification, which are key drivers of the condition. Anakinra and Canakinumab work by inhibiting interleukin-1, a cytokine involved in inflammation, potentially reducing bone formation outside the normal skeleton. This targeted action could provide significant relief and improve quality of life for those affected by FOP, sparking optimism in the medical community.
What evidence suggests that anti-IL1 therapy could be effective for FOP?
Research has shown that certain treatments, such as anakinra and canakinumab, may benefit people with Fibrodysplasia Ossificans Progressiva (FOP). One study found that these treatments significantly reduced flare-ups, which are sudden worsening of symptoms, leading to better overall health for patients. Early results also suggest these therapies can lessen the frequency of flares. However, more research is needed to understand their effects on new bone growth and pain relief. In this trial, participants in the Anti-IL1 Observational Arm will receive Anti-IL1 therapy, including treatments like anakinra and canakinumab. Meanwhile, the Optional Non-Treatment Observational Arm will include participants unable to obtain Anti-IL1 therapy. Overall, these treatments show promise in managing some symptoms of FOP.23678
Who Is on the Research Team?
Edward Hsiao, MD, PhD
Principal Investigator
University of California, San Francisco
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Observational Period
Participants begin an observational period during the medication prescription and insurance approval process
Treatment
Participants receive anti-IL1 therapy with either anakinra or canakinumab and are monitored for flare activity, pain, and other outcomes
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Anakinra
- Canakinumab
How Is the Trial Designed?
3
Treatment groups
Experimental Treatment
FOP patients unable to obtain Anti-IL1 therapy
FOP patients that are beginning treatment with Anti-IL1 Therapy
FOP patients that are already using Anti-IL1 therapy
Find a Clinic Near You
Who Is Running the Clinical Trial?
University of California, San Francisco
Lead Sponsor
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Collaborator
Citations
Long-term use of interleukin-1 inhibitors reduce flare ...
This case series demonstrates significant benefits from IL-1 inhibitors for reducing flare activity and improving the general health of patients with FOP.
NCT06724562 | This is an Observational Pre-post Study to ...
Preliminary data suggests patients experience significant decreases in flare frequency when taking anti-IL1 therapy, but other measures of efficacy remain ...
Observe if the Off Label Use of Anti-IL1 Therapies, Such as ...
Preliminary data suggests patients experience significant decreases in flare frequency when taking anti-IL1 therapy, but other measures of ...
Project Details
PROJECT SUMMARY Abnormal responses to inflammatory stimuli can lead to severe inflammation, acute tissue damage, and long- term scarring or fibrosis.
Successful experience of tofacitinib treatment in patients with ...
Tofacitinib is a highly efficient, well-tolerated option that may prevent FOP flares. Further studies of the therapeutic potential of JAK-kinase inhibitors in ...
6.
reporter.nih.gov
reporter.nih.gov/search/14E9CE024F8FC5D27598B8961CAA4A01A2FFCEB861BF/project-details/11161515An Observational Study of IL1 Inhibition for ... - NIH RePORTER
The Project Outcomes shown here are displayed verbatim as submitted by the Principal Investigator (PI) for this award. Any opinions, findings, and conclusions ...
Long-term use of interleukin-1 inhibitors reduce flare activity in ...
All 4 patients showed persistent improvement in flare activity during treatment with IL-1 inhibitors, with minimal formation of new HO sites.
A Narrative Review of Phase II and III Clinical Trials for the ...
These studies investigated pharmacological treatments for Fibrodysplasia Ossificans Progressiva (FOP) in Phase II and III clinical trials, ...
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