Garetosmab for Stone Man Syndrome
(OPTIMA Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new drug, garetosmab, for people with fibrodysplasia ossificans progressiva (FOP), a rare condition where muscles and tissues gradually turn into bone. The goal is to assess the safety of garetosmab and its effectiveness in managing FOP symptoms. Participants will receive either a high dose, a low dose, or a placebo (a substance with no active drug). Ideal participants have been diagnosed with FOP, experience symptoms like pain or swelling, and have had disease activity in the past year. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to potentially groundbreaking treatment.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications, but it does exclude those who have used certain drugs like bisphosphonates in the past year or other investigational drugs recently. It's best to discuss your specific medications with the study team.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research shows that garetosmab has been tested in people with fibrodysplasia ossificans progressiva (FOP), a rare condition where muscles gradually turn into bone. In previous studies, patients received garetosmab through an IV every four weeks, and the safety results supported this schedule. Some patients with very severe FOP passed away due to the seriousness of their condition, not the treatment. Overall, garetosmab was well-tolerated. The current study is in a later phase, indicating that earlier research generally found the treatment to be safe.12345
Why do researchers think this study treatment might be promising for Stone Man Syndrome?
Unlike the standard treatments for Stone Man Syndrome, which primarily focus on managing symptoms and complications, garetosmab offers a novel approach by targeting the underlying cause of the condition. Garetosmab is a monoclonal antibody that specifically inhibits activin A, a protein involved in the abnormal bone formation characteristic of the disease. This targeted action could potentially prevent new bone growth, offering hope for a more effective intervention. Researchers are excited about garetosmab because it addresses the root of the problem, potentially leading to better outcomes for patients with this rare and challenging condition.
What evidence suggests that garetosmab might be an effective treatment for fibrodysplasia ossificans progressiva?
Research has shown that garetosmab holds promise for treating fibrodysplasia ossificans progressiva (FOP), a rare condition where muscles and tissues turn into bone. One study found that garetosmab reduced new bone formation by up to 94%. It also decreased the length and severity of flare-ups, which are painful episodes of bone growth. Patients taking garetosmab required less corticosteroid therapy, a common treatment for managing symptoms. In this trial, participants will receive either a high dose or low dose of garetosmab, or a placebo. These findings suggest that garetosmab could effectively manage and slow down FOP.12567
Who Is on the Research Team?
Clinical Trial Management
Principal Investigator
Regeneron Pharmaceuticals
Are You a Good Fit for This Trial?
Adults with fibrodysplasia ossificans progressiva (FOP) who have had disease activity within the past year can join this trial. They must be able to undergo CT scans and meet specific health criteria, excluding those with cancer, severe kidney issues, uncontrolled diabetes, significant heart or respiratory conditions, or women who are pregnant/breastfeeding.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive garetosmab or placebo intravenously every 4 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
Extended Treatment
Participants on extended treatment are monitored for new HO lesions and flare-ups
What Are the Treatments Tested in This Trial?
Interventions
- Garetosmab
- Placebo
Find a Clinic Near You
Who Is Running the Clinical Trial?
Regeneron Pharmaceuticals
Lead Sponsor
Leonard Schleifer
Regeneron Pharmaceuticals
Chief Executive Officer since 1988
MD and PhD in Medicine
George Yancopoulos
Regeneron Pharmaceuticals
Chief Medical Officer since 1997
MD from Harvard Medical School