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Monoclonal Antibodies

Garetosmab for Stone Man Syndrome (OPTIMA Trial)

Phase 3
Waitlist Available
Research Sponsored by Regeneron Pharmaceuticals
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Clinical diagnosis of Fibrodysplasia Ossificans Progressiva (FOP) based on findings of congenital malformation of the great toes, episodic soft tissue swelling, and/or progressive Heterotopic Ossification (HO)
FOP disease activity within 1 year of screening visit. FOP disease activity is defined as pain, swelling, stiffness, or other signs and symptoms associated with FOP flare-ups; or worsening of joint function, or radiographic progression of HO lesions (increase in size or number of HO lesions) with/without being associated with flare-up episodes
Must not have
Cardiovascular conditions such as New York Heart Association class III or IV heart failure, cardiomyopathy, intermittent claudication, myocardial infarction, or acute coronary syndrome within 6 months prior to screening; symptomatic ventricular cardiac arrhythmia
Uncontrolled diabetes defined as hemoglobin A1C (HbA1c) >9% at screening
Timeline
Screening 3 weeks
Treatment Varies
Follow Up at week 28, week 56 and week 84
Awards & highlights

Summary

This trial is testing garetosmab, an experimental drug, in adults with fibrodysplasia ossificans progressiva (FOP). The drug aims to stop or slow down abnormal bone growth by blocking a specific protein. The study will also look at side effects and how the body reacts to the drug.

Who is the study for?
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.
What is being tested?
The trial is testing garetosmab versus a placebo in adults with FOP to evaluate its safety and effectiveness. It will also examine potential side effects, how much of the drug stays in the blood over time, and if the body creates antibodies against it.
What are the potential side effects?
Potential side effects from garetosmab may include reactions at the infusion site, general discomfort or changes in blood tests indicating organ function. The study will monitor for any new antibodies that might reduce drug effectiveness or cause other side effects.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have been diagnosed with FOP, showing toe malformations, swelling, or bone growth in muscles.
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I've had FOP symptoms or worsening conditions in the past year.
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My FOP is confirmed with a specific ACVR1 mutation.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I do not have severe heart problems or recent heart attacks.
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My diabetes is not under control, with an HbA1c level over 9%.
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I have a known history of abnormal blood vessels in my brain.
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I haven't taken investigational drugs or specific treatments like denosumab, imatinib, or isotretinoin recently.
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I have used bisphosphonates in the past year and am currently using them.
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My kidney function is severely impaired.
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I have had serious breathing problems needing oxygen or machines to help me breathe.
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I have had cancer before.
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I have a history of high blood pressure that is hard to control.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~at week 28, week 56 and week 84
This trial's timeline: 3 weeks for screening, Varies for treatment, and at week 28, week 56 and week 84 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Secondary study objectives
Change in disease severity as assessed by the Clinician's Global Impression of Change (CGIC)
Change in disease severity as assessed by the Patient Global Impression of Severity (PGIS)
Change in disease severity as assessed by the Patient's Global Impression of Change (PGIC)
+9 more

Trial Design

3Treatment groups
Experimental Treatment
Group I: PlaceboExperimental Treatment1 Intervention
Placebo to match garetosmab, is supplied as a liquid solution without the monoclonal antibody (or the protein) and is administered IV Q4W.
Group II: Low dose GaretosmabExperimental Treatment1 Intervention
Garetosmab is administered by IV administration Q4W
Group III: High dose GaretosmabExperimental Treatment1 Intervention
Garetosmab is administered by intravenous (IV) administration every 4 weeks (Q4W)
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Placebo
1995
Completed Phase 3
~2670

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Garetosmab is an anti-Activin A antibody that works by inhibiting the activity of Activin A, a protein involved in the abnormal bone formation seen in Fibrodysplasia Ossificans Progressiva (FOP). By blocking Activin A, garetosmab aims to prevent or reduce the formation of extra-skeletal bone, which is a hallmark of FOP. Understanding this mechanism is crucial for patients as it provides insight into how the treatment may help manage their condition by targeting a specific pathway involved in disease progression, potentially leading to more effective and tailored therapies.
[Diagnosis and therapy of chronic glomerulonephritis in long-term follow-up].Plasmapheresis and immunosuppressive drug therapy in the Eaton-Lambert syndrome.TAFRO Syndrome: A Case of Significant Endocrinopathy in a Caucasian Patient.

Find a Location

Who is running the clinical trial?

Regeneron PharmaceuticalsLead Sponsor
655 Previous Clinical Trials
385,204 Total Patients Enrolled
Clinical Trial ManagementStudy DirectorRegeneron Pharmaceuticals
280 Previous Clinical Trials
254,592 Total Patients Enrolled

Media Library

Garetosmab (Monoclonal Antibodies) Clinical Trial Eligibility Overview. Trial Name: NCT05394116 — Phase 3
Stone Man Syndrome Research Study Groups: High dose Garetosmab, Low dose Garetosmab, Placebo
Stone Man Syndrome Clinical Trial 2023: Garetosmab Highlights & Side Effects. Trial Name: NCT05394116 — Phase 3
Garetosmab (Monoclonal Antibodies) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05394116 — Phase 3
~18 spots leftby Jul 2025