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Monoclonal Antibodies
Garetosmab for Stone Man Syndrome (OPTIMA Trial)
Phase 3
Recruiting
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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up at week 28, week 56 and week 84
Awards & highlights
OPTIMA Trial Summary
This trial will study the effects of garetosmab on Fibrodysplasia ossificans progressiva (FOP) patients and their safety/tolerability.
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.Check my eligibility
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.See study design
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.
OPTIMA Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have been diagnosed with FOP, showing toe malformations, swelling, or bone growth in muscles.
Select...
I've had FOP symptoms or worsening conditions in the past year.
Select...
My FOP is confirmed with a specific ACVR1 mutation.
OPTIMA Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ at week 28, week 56 and week 84
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~at week 28, week 56 and week 84
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Incidence and severity of treatment-emergent adverse events of special interest (AESIs)
Number of new HO lesions adjudicated as positive based on computed tomography (CT)
Secondary outcome measures
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)
+13 moreOPTIMA 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
Find a Location
Who is running the clinical trial?
Regeneron PharmaceuticalsLead Sponsor
622 Previous Clinical Trials
380,665 Total Patients Enrolled
Clinical Trial ManagementStudy DirectorRegeneron Pharmaceuticals
263 Previous Clinical Trials
251,348 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility 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.My diabetes is not under control, with an HbA1c level over 9%.I have a known history of abnormal blood vessels in my brain.I haven't taken investigational drugs or specific treatments like denosumab, imatinib, or isotretinoin recently.I have been diagnosed with FOP, showing toe malformations, swelling, or bone growth in muscles.I have used bisphosphonates in the past year and am currently using them.My kidney function is severely impaired.I have had serious breathing problems needing oxygen or machines to help me breathe.I have had cancer before.I have a history of high blood pressure that is hard to control.I've had FOP symptoms or worsening conditions in the past year.My FOP is confirmed with a specific ACVR1 mutation.
Research Study Groups:
This trial has the following groups:- Group 1: High dose Garetosmab
- Group 2: Low dose Garetosmab
- Group 3: Placebo
Awards:
This trial has 1 awards, including:- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Is there a recruitment process open for this experiment?
"According to the information provided on clinicaltrials.gov, this ongoing medical trial is currently open for candidates. Participants in the study were first accepted as of November 21st 2022 and there have been recent edits made to the notes on July 13th 2023."
Answered by AI
What is the aggregate amount of participants in this research endeavor?
"Affirmative. According to the clinicaltrials.gov website, this medical trial has opened applications since November 21st 2022 and is currently searching for 66 volunteers from 16 different sites."
Answered by AI
How widely distributed is the execution of this experiment?
"Currently, the trial is recruiting from a total of 16 medical facilities across Europe. Specifically, Hospital Universitario Ramon y Cajal in Madrid and HUS Children and Adolescents Park Hospital Clinical Trial Unit in Helsinki are among them, with an additional 14 sites also offering enrollment opportunities."
Answered by AI
Has the FDA endorsed high dose Garetosmab for therapeutic use?
"Garetosmab has been evaluated in multiple clinical studies, and its safety is believed to be substantial. Therefore it received a rating of 3 on our scale."
Answered by AI
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