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A Study to Assess Safety, Tolerability and Efficacy of Garetosmab Versus Placebo Administered Intravenously (IV) in Adult Participants With Fibrodysplasia Ossificans Progressiva (FOP) | Clinical Trials | Clareo Health

ACTIVE_NOT_RECRUITINGPHASE3

A Study to Assess Safety, Tolerability and Efficacy of Garetosmab Versus Placebo Administered Intravenously (IV) in Adult Participants With Fibrodysplasia Ossificans Progressiva (FOP)

Phase 3 Randomized, Placebo-Controlled Study to Assess Safety, Tolerability, and Efficacy of Garetosmab in Patients With Fibrodysplasia Ossificans Progressiva

NCT05394116•Regeneron Pharmaceuticals

View on ClinicalTrials.gov

Summary

This study is researching an experimental drug called garetosmab. The study is focused on adult patients with fibrodysplasia ossificans progressiva (FOP). The aim of the study is to see how safe and effective the study drug is in patients with FOP. The study is looking at several other research questions, including: * What side effects may happen from receiving the study drug * How much study drug is in the blood at different times * Whether the body makes antibodies against the study drug (which could make the drug less effective or could lead to side effects)

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. Clinical diagnosis of Fibrodysplasia Ossificans Progressiva (FOP) \[(based on findings of congenital malformation of the great toes, episodic soft tissue swelling, and/or progressive Heterotopic Ossification (HO)\]
•2. Confirmation of FOP diagnosis with documentation of Type I activin A receptor (ACVR1) FOP causing mutation
•3. 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
•4. Willing and able to undergo CT imaging procedures and other procedures as defined in the protocol

Exclusion Criteria

•1. Cumulative Analog Joint Involvement Scale (CAJIS) score at screening \>19
•2. Participant has significant concomitant illness or history of significant illness such as but not limited to cardiac, renal, rheumatologic, neurologic, psychiatric, endocrine, metabolic, or lymphatic disease, that in the opinion of the study investigator might confound the results of the study or pose additional risk to the patient by their participation in the study
•3. Previous history or diagnosis of cancer
•4. Severely impaired renal function defined as estimated glomerular filtration rate \<30 milliliter per minute (mL/min) (/1.73 m\^2 calculated by the Modification of Diet in Renal Disease equation
•5. Uncontrolled diabetes defined as hemoglobin A1C (HbA1c) \>9% at screening
•6. History of poorly controlled hypertension, as defined by:
•1. Systolic blood pressure ≥180 mm Hg or diastolic blood pressure ≥110 mm Hg at the screening visit
•2. Systolic blood pressure of 160 mm Hg to 179 mm Hg or diastolic blood pressure of 100 mm Hg to 10\^9 mm Hg at the screening visit, AND a history of end-organ damage (including history of left-ventricular hypertrophy, heart failure, angina, myocardial infarction, stroke, transient ischemic attack, peripheral arterial disease, end-stage renal disease, and moderate-to-advanced retinopathy
•7. Known history of cerebral vascular malformation
•8. 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
+7 more criteria

Locations (22)

University of California Los Angeles (UCLA) Medical Center

Los Angeles, California, United States

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Royal North Shore Hospital

St Leonards, New South Wales, Australia

Hospital Israelita Albert Einstein

São Paulo, Brazil

Universidad de Concepcion

Concepción, Bio Bio, Chile

Tongji Hospital of Tongji University

Shanghai, China

Clinica Universidad de La Sabana

Chía, Cundinamarca, Colombia

HUS Children and Adolescents Park Hospital Clinical Trial Unit

Helsinki, Stenbäckinkatu 11, Finland

Hôpital Lapeyronie

Montpellier, France

Hopital Lariboisiere

Paris, France

Key Dates

Start Date

November 21, 2022

Primary Completion Date

July 17, 2025

Completion Date

February 27, 2029

Last Updated

January 16, 2026

Enrollment

ACTUAL participants

Conditions

Fibrodysplasia Ossificans Progressiva

Interventions

Garetosmab

DRUG

Placebo

DRUG

Saracatinib Trial TO Prevent FOP

NCT04307953

Fibrodysplasia Ossificans Progressiva

View study

COMPLETEDPHASE3

A Rollover Study to Further Evaluate the Safety and Efficacy of Palovarotene Capsules in Male and Female Participants Aged ≥14 Years With Fibrodysplasia Ossificans Progressiva (FOP) Who Have Completed the Relevant Parent Studies.

NCT05027802

Fibrodysplasia Ossificans Progressiva (FOP)

View study

COMPLETED

An International Cross-sectional Survey to Evaluate the Burden of Fibrodysplasia Ossificans Progressiva (FOP) on Patients and Their Families.

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: January 16, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.