A Study to Assess Safety, Tolerability and Efficacy of Garetosmab Versus Placebo Administered Intravenously (IV) in Adult Participants With Fibrodysplasia Ossificans Progressiva (FOP)
NCT ID: NCT05394116
Last Updated: 2026-01-16
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE3
63 participants
INTERVENTIONAL
2022-11-21
2029-02-27
Brief Summary
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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)
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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High dose Garetosmab
Garetosmab is administered by intravenous (IV) administration every 4 weeks (Q4W)
Garetosmab
Garetosmab is supplied as a liquid drug product and will be administered IV.
Low dose Garetosmab
Garetosmab is administered by IV administration Q4W
Garetosmab
Garetosmab is supplied as a liquid drug product and will be administered IV.
Placebo
Placebo to match garetosmab, is supplied as a liquid solution without the monoclonal antibody (or the protein) and is administered IV Q4W.
Placebo
Placebo to match garetosmab, is supplied as a liquid solution without the monoclonal antibody (or the protein) and is administered IV.
Interventions
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Garetosmab
Garetosmab is supplied as a liquid drug product and will be administered IV.
Placebo
Placebo to match garetosmab, is supplied as a liquid solution without the monoclonal antibody (or the protein) and is administered IV.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
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
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
9. History of severe respiratory compromise requiring oxygen, respiratory support (eg, bilevel positive airway pressure \[biPAP\] or continuous positive airway pressure \[CPAP\]), or a history of aspiration pneumonia requiring hospitalization
10. Prior use in the past year and concomitant use of bisphosphonates
11. Concurrent participation in another interventional clinical study or a non-interventional study with radiographic measures or invasive procedures (eg, collection of blood or tissue samples)
12. Treatment with another investigational drug, denosumab, imatinib or isotretinoin in the last 30 days or within 5 half-lives of the investigational drug, whichever is longer
13. Pregnant or breastfeeding women
14. Women of childbearing potential (WOCBP) who are unwilling to practice highly effective contraception, as defined in the protocol
15. Male patients with WOCBP partners who are not willing to use condoms with WOCBP partners to prevent potential fetal exposure, as defined in the protocol
18 Years
ALL
No
Sponsors
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Regeneron Pharmaceuticals
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Trial Management
Role: STUDY_DIRECTOR
Regeneron Pharmaceuticals
Locations
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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
Queen Mary Hospital
Hong Kong, , Hong Kong
IRCCS Istituto Giannina Gaslini
Genoa, , Italy
Nagoya University Hospital
Nagoya, Aichi-ken, Japan
Oita University Hospital
Yufu, Oita Prefecture, Japan
Kyushu University Hospital
Fukuoka, , Japan
Hospital Kuala Lampur
Kuala Lumpur, , Malaysia
Amsterdam University Medical Center
Amsterdam, North Holland, Netherlands
Szpital Centrum Medyczne Medyk
Rzeszów, Podkarpackie Voivodeship, Poland
University of Cape Town
Rondebosch, Cape Town, South Africa
Seoul National University Hospital
Seoul, , South Korea
Hospital Universitario Ramon y Cajal
Madrid, , Spain
Royal National Orthropaedic Hospital NHS Trust
Middlesex, Greater London, United Kingdom
Countries
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Other Identifiers
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2022-000880-40
Identifier Type: REGISTRY
Identifier Source: secondary_id
2023-508350-26-00
Identifier Type: REGISTRY
Identifier Source: secondary_id
R2477-FOP-2175
Identifier Type: -
Identifier Source: org_study_id
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