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Promising drug may halt extra bone formation in rare 'Stone Man' disease

NCT ID NCT05394116

Disease control Sponsor: Regeneron Pharmaceuticals Source: ClinicalTrials.gov ↗

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Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jul 17, 2026 · Updated 1 time

Summary

This study tests an experimental drug, garetosmab, in 63 adults with fibrodysplasia ossificans progressiva (FOP), a rare condition where soft tissues turn into bone. The goal is to see if the drug safely reduces new bone growth and painful flare-ups. Participants receive either the drug or a placebo, and researchers monitor side effects and drug levels in the blood.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

63 people

The number who actually took part.

Started

Nov 2022

Expected to finish

Feb 2029

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key 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 Key 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 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 Note: Other protocol defined Inclusion/Exclusion Criteria apply

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Conditions

The condition(s) this trial relates to.

fibrodysplasia ossificans progressiva myositis ossificans Ossification, Heterotopic

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Fibrodysplasia ossificans progressiva

Contacts and locations

Locations

  • Amsterdam University Medical Center

    Amsterdam, North Holland, 1081 HV, Netherlands

  • Clinica Universidad de La Sabana

    Chía, Cundinamarca, 140013, Colombia

  • HUS Children and Adolescents Park Hospital Clinical Trial Unit

    Helsinki, Stenbäckinkatu 11, 00029, Finland

  • Hopital Lariboisiere

    Paris, 75010, France

  • Hospital Israelita Albert Einstein

    São Paulo, 05652-900, Brazil

  • Hospital Kuala Lampur

    Kuala Lumpur, 50586, Malaysia

  • Hospital Universitario Ramon y Cajal

    Madrid, 28034, Spain

  • Hôpital Lapeyronie

    Montpellier, 34090, France

  • IRCCS Istituto Giannina Gaslini

    Genoa, 16147, Italy

  • Kyushu University Hospital

    Fukuoka, 812-8582, Japan

  • Nagoya University Hospital

    Nagoya, Aichi-ken, 466-8560, Japan

  • Oita University Hospital

    Yufu, Oita Prefecture, 879-5593, Japan

  • Queen Mary Hospital

    Hong Kong, 22553838, Hong Kong

  • Royal National Orthropaedic Hospital NHS Trust

    Middlesex, Greater London, HA7 4LP, United Kingdom

  • Royal North Shore Hospital

    St Leonards, New South Wales, 2065, Australia

  • Seoul National University Hospital

    Seoul, 03080, South Korea

  • Szpital Centrum Medyczne Medyk

    Rzeszów, Podkarpackie Voivodeship, 35-326, Poland

  • Tongji Hospital of Tongji University

    Shanghai, 200065, China

  • Universidad de Concepcion

    Concepción, Biobio, 4030000, Chile

  • University of California Los Angeles (UCLA) Medical Center

    Los Angeles, California, 90095, United States

  • University of Cape Town

    Rondebosch, Cape Town, 7700, South Africa

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232, United States

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