Garetosmab for Stone Man Syndrome

(OPTIMA Trial)

Recruiting at 21 trial locations

Overseen By Clinical Trials Administrator

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Regeneron Pharmaceuticals

Must not be taking: Bisphosphonates, Denosumab, Imatinib, Isotretinoin

Disqualifiers: Cancer, Renal disease, Hypertension, Diabetes, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 13

Duration: 28 weeks

63 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

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.

What makes the drug Garetosmab unique for treating Stone Man Syndrome?

Garetosmab is unique for treating Stone Man Syndrome because it specifically targets the underlying cause of the condition, which is the overactivity of a protein called activin A, unlike other treatments that may not address this specific mechanism. ^{1 2 3 4 5}

Research Team

Clinical Trial Management

Principal Investigator

Regeneron Pharmaceuticals

Eligibility Criteria

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

Willing and able to undergo CT imaging procedures and other procedures as defined in the protocol

I have been diagnosed with FOP, showing toe malformations, swelling, or bone growth in muscles.

I've had FOP symptoms or worsening conditions in the past year.

Exclusion Criteria

I am a man unwilling to use condoms with my partner who can become pregnant.

I do not have severe heart problems or recent heart attacks.

Pregnant or breastfeeding women

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive garetosmab or placebo intravenously every 4 weeks

28 weeks

7 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

28 weeks

3 visits (in-person)

Extended Treatment

Participants on extended treatment are monitored for new HO lesions and flare-ups

28 weeks

3 visits (in-person)

Treatment Details

Interventions

• Garetosmab
• Placebo

Trial Overview 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.

Participant Groups

Group I: Placebo Experimental Treatment 1 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 Garetosmab Experimental Treatment 1 Intervention

Garetosmab is administered by IV administration Q4W

Group III: High dose Garetosmab Experimental Treatment 1 Intervention

Garetosmab is administered by intravenous (IV) administration every 4 weeks (Q4W)

Who Is Running the Clinical Trial?

Regeneron Pharmaceuticals

Lead Sponsor

Trials

690

Recruited

948,000+

Founded

1988

Headquarters

Tarrytown, USA

Known For

Precision medicine

Top Products

Dupixent, EYLEA, Libtayo, Praluent

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

References

1. New Zealand pubmed.ncbi.nlm.nih.gov

Immunogenicity of Biosimilars for Rheumatic Diseases, Plaque Psoriasis, and Inflammatory Bowel Disease: A Review from Clinical Trials and Regulatory Documents. [2020]

2. England pubmed.ncbi.nlm.nih.gov

Impact of immunogenicity on efficacy and tolerability of tumour necrosis factor inhibitors: pooled analysis of biosimilar studies in rheumatoid arthritis. [2021]

3. United States pubmed.ncbi.nlm.nih.gov

Comparing Effects of Biologic Agents in Treating Patients with Rheumatoid Arthritis: A Multiple Treatment Comparison Regression Analysis. [2022]

4. England pubmed.ncbi.nlm.nih.gov

Long-Term Safety and Efficacy of Subcutaneously Administered Tocilizumab for Adult Rheumatoid Arthritis: A Multicenter Phase 3b Long-term Extension Study. [2022]

5. United States pubmed.ncbi.nlm.nih.gov

Efficacy and Safety of Tabalumab, an Anti-B-Cell-Activating Factor Monoclonal Antibody, in a Heterogeneous Rheumatoid Arthritis Population: Results From a Randomized, Placebo-Controlled, Phase 3 Trial (FLEX-O). [2021]