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New drug aims to slash fractures in brittle bone disease

NCT ID NCT05125809

Disease control Sponsor: Ultragenyx Pharmaceutical Inc Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 27, 2026

Summary

This study tests a medicine called setrusumab in people with osteogenesis imperfecta (brittle bone disease). The goal is to see if it can lower the number of bone fractures. About 183 participants with types I, III, or IV of the disease who have had recent fractures will receive either the drug or a placebo. The study is in phases 2 and 3, meaning it checks both the right dose and how well it works compared to no treatment.

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 2/3

Runs two stages together: whether the treatment works, then large-scale confirmation.

Participants

183 people

The number who actually took part.

Started

Feb 2022

Expected to finish

Apr 2027

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

5 to 25 years

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.

Inclusion Criteria: * Diagnosis of OI Type I, III, or IV as confirmed by identification of pathogenic or likely pathogenic genetic variants in COL1A1 or COL1A2. If a variant of uncertain significance is identified, then clinical presence of the expected phenotype can be used to confirm the diagnosis * ≥ 1 fracture in the past 12 months, ≥ 2 fractures in the past 24 months or ≥ 1 tibia, femur or humerus fracture in the past 24 months * Serum 25-hydroxyvitamin D ≥ 20 ng/mL at the Screening Visit. If 25-hydroxyvitamin D levels are below 20 ng/mL, 25-hydroxyvitamin D testing can repeated after a minimum of 14 days of vitamin D supplementation as directed by the treating physician * Willing to not receive bisphosphonate therapy during the study * From the period following informed consent to 60 days after the last dose of the study drug, females of childbearing potential and fertile males must consent to use highly effective contraception. If female, agree not to become pregnant. If male, agree not to father a child or donate sperm * Willing and able to provide informed consent for subjects greater than or equal to 18 years of age, or provide assent (if possible) and have a legally authorized representative provide informed consent, after the nature of the study has been explained and prior to any research-related procedures * Willing to provide access to medical records for the collection of radiographic data, fracture data, growth data, and disease history * Must, in the opinion of the Investigator, be willing and able to complete all aspects of the study, adhere to the study visit schedule, and comply with the assessments Exclusion Criteria: * History of skeletal malignancies or bone metastases at any time * History of neural foraminal stenosis (except if due to scoliosis) * Clinical manifestations of Chiari malformation or basilar invagination. Presence of any other neurologic disease that has been unstable within past 2 years requires review by the Medical Monitor * History of or uncontrolled concomitant diseases such as hypo/hyperparathyroidism, Paget's disease, abnormal thyroid function, thyroid disease or other endocrine disorders or conditions that could affect bone metabolism such as Stage IV/V renal disease * Rickets or any skeletal condition (other than OI) leading to long-bone deformities and/or increased risk of fractures * History of stroke, myocardial infarction, transient ischemic attack or angina. * Hypocalcemia, defined as serum calcium levels below the age-adjusted normal limits after a ≥ 4 hour fast * Estimated glomerular filtration rate ≤ 29 mL/min/1.73 m2 * Prior treatment with the following: 1. Teriparatide, growth hormone, bone anabolic, or anti-resorptive medications (other than bisphosphonates) within 6 months of the first dose with study drug (Month 0) 2. Denosumab within 24 months of Screening 3. Romosozumab at any time * Documented alcohol and/or drug abuse within 12 months prior to dosing or evidence of such abuse as indicated by the laboratory results during the Screening assessments * Presence or history of any condition that, in the view of the Investigator, would interfere with participation, pose undue risk, or would confound interpretation of results * Known hypersensitivity to setrusumab or excipients that, in the judgment of the Investigator, places the subject at increased risk for adverse effects * History of external radiation therapy * Pregnant or breastfeeding or planning to become pregnant (self or partner) at any time during the study * Use of any investigational product or investigational medical device within 4 weeks or 5 half-lives of investigational drug (whichever is longer) prior to Screening, or during the study (per discretion of the Investigator in consultation with the Medical Monitor) * Concurrent participation in another clinical study without prior approval from the Investigator in consultation with the Medical Monitor * For Phase 2 Only: A history of bone surgery within the previous 6 months prior to Screening or planned bone surgery for the first 3 months of the study

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Conditions

The condition(s) this trial relates to.

osteogenesis imperfecta osteogenesis imperfecta type 1 osteogenesis imperfecta type 3 osteogenesis imperfecta type 4

As listed by the trial registrant

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

Osteogenesis imperfecta

Contacts and locations

Locations

  • Arkansas Children's Hospital

    Little Rock, Arkansas, 72202, United States

  • Atrium Health Levine Children's Hospital

    Charlotte, North Carolina, 28203, United States

  • Azienda Ospedaliera Universitaria Policlinico Umberto I

    Rome, 00161, Italy

  • Baylor College of Medicine

    Houston, Texas, 77030, United States

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • Centro Hospitalar do Porto

    Porto, 4099-001, Portugal

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027, United States

  • Children's Hospital and Medical Center

    Omaha, Nebraska, 68114, United States

  • Children's Hospital of Eastern Ontario

    Ottawa, Ontario, K1H 8L1, Canada

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20010, United States

  • Connecticut Children's Medical Center

    Hartford, Connecticut, 06106, United States

  • Cook Children's Medical Center

    Fort Worth, Texas, 76104, United States

  • Gazi University

    Ankara, Turkey (Türkiye)

  • Hospital de Santa Maria

    Lisbon, 1649-028, Portugal

  • Indiana University Hospital

    Indianapolis, Indiana, 46202, United States

  • Institut Imagine

    Paris, France

  • Istituto Ortopedico Rizzoli

    Bologna, Italy

  • Kennedy Krieger Institute

    Baltimore, Maryland, 21205, United States

  • London Health Sciences Center

    London, Ontario, Canada

  • Marmara University

    Istanbul, Turkey (Türkiye)

  • McGill University Health Centre

    Montreal, H4A 3J1, Canada

  • Musculoskeletal Center Würzburg

    Würzburg, 97074, Germany

  • Nationwide Children's Hospital- Ohio State University College of Medicine

    Columbus, Ohio, 43205, United States

  • Nemours/ Alfred i. duPoint Hospital for Children

    Wilmington, Delaware, 19803, United States

  • New Mexico Clinical Research & Osteoporosis Center, Inc.

    Albuquerque, New Mexico, 87106, United States

  • Otto von Guericke University Magdeburg

    Magdeburg, 39106, Germany

  • Queensland Paediatric Endocrinology

    South Brisbane, Queensland, QLD 4101, Australia

  • Royal Children's Hospital

    Melbourne, Australia

  • Royal Manchester Childrens Hospital

    Manchester, United Kingdom

  • Sheffield Children's NHS Foundation Trust

    Sheffield, S10 2TH, United Kingdom

  • Shriners Hospital for Children - Northern California

    Sacramento, California, 95817, United States

  • Shriners Hospitals for Children - Chicago

    Chicago, Illinois, 60707, United States

  • The Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • UW Health University Hospital

    Madison, Wisconsin, 53792, United States

  • Universita Degli Studi Di Verona

    Verona, Italy

  • University of Cologne

    Cologne, Germany

  • University of South Florida

    Tampa, Florida, 33612, United States

  • University of Toronto- The Hospital for Sick Children (SickKids)

    Toronto, Canada

  • Uniwersytet Medyczny w Lodzi - Klinika Endokrynologii i Chorob Metabolicznych

    Lodz, Poland

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232, United States

  • Washington University School of Medicine

    St Louis, Missouri, 63110, United States

  • Wilhelmina Children's Hospital

    Utrecht, Netherlands

  • Yale New Haven Hospital

    New Haven, Connecticut, 06510, United States

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