What side effects are associated with this type of intervention?

Common treatments for Fibrodysplasia Ossificans Progressiva (FOP) include anti-inflammatory drugs, corticosteroids, and experimental therapies like garetosmab (an anti-Activin A antibody). Known side effects of these treatments can include immune reactions such as the development of antibodies against the drug, which may reduce its effectiveness or cause adverse effects. Other potential side effects may include injection site reactions, increased risk of infections, and general symptoms like fatigue or gastrointestinal issues. It is crucial to monitor patients closely for these side effects during treatment.

What prior FDA approvals exist?

As of now, there are no FDA-approved treatments specifically for Fibrodysplasia Ossificans Progressiva (FOP), also known as Stone Man Syndrome. Garetosmab, an anti-Activin A antibody, is currently being studied for its safety and efficacy in treating FOP. Other treatments for FOP are primarily supportive and symptomatic, focusing on managing pain and preventing flare-ups. Research is ongoing to find effective therapies for this rare and debilitating condition.

What other types of research exist?

Currently, there are no specific novel alternatives to Garetosmab mentioned in the provided context for treating Fibrodysplasia Ossificans Progressiva (FOP). Patients should consult with their healthcare provider to explore ongoing clinical trials or emerging therapies targeting similar pathways.

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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 weeks 28, 56 and 84

Awards & highlights

Pivotal Trial

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 weeks3 visits

Treatment ~ Varies

Follow Up ~ at weeks 28, 56 and 84

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~at weeks 28, 56 and 84

This trial's timeline: 3 weeks for screening, Varies for treatment, and at weeks 28, 56 and 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

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

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

0 / 12Eligibility criteria met

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.