New drug aims to cut fractures in kids with brittle bones
NCT ID NCT05768854
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a new medicine called setrusumab against standard bone-strengthening drugs (bisphosphonates) in children aged 2 to under 7 with osteogenesis imperfecta (brittle bone disease) types I, III, or IV. The goal is to see if setrusumab can lower the number of bone breaks, including spine fractures. About 69 children who have had at least one fracture in the past year will take part.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 3
Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.
- Participants
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69 people
The number who actually took part.
- Started
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Jun 2023
- Expected to finish
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Apr 2027
An estimate. End dates often move.
- Lead sponsor
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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
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2 to 6 years
- Sex
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Anyone
- Healthy volunteers
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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 Hide 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: * Male or female 2 to \< 7 years of age at time of informed consent * Clinical diagnosis of OI Types I, III, or IV confirmed by identification of genetic mutation in COL1A1 or COL1A2 * History of ≥ 1 fracture in the past 12 months, ≥ 2 fractures in the past 24 months, or ≥ 1 femur, tibia, or humerus fracture in the past 24 months * Any prior exposure to, or currently receiving, IV-bisphosphonate therapy for treatment of OI * Serum 25-hydroxyvitamin D level ≥ 20 ng/mL at the Screening visit. If 25-hydroxyvitamin D levels are below 20 ng/mL, the subject may be rescreened after a minimum of 14 days of vitamin D supplementation as directed by the Investigator Exclusion Criteria: * Contraindication for the use of IV bisphosphonates based on clinical judgment of the Investigator * 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 clinically unstable within past 2 years requires review by the Medical Monitor. * History of or current uncontrolled concomitant diseases that may impact bone metabolism, such as hypo/hyperparathyroidism, abnormal thyroid function, nephrotic syndrome, or Stage IV/V renal disease * Any skeletal condition (other than OI) leading to bone deformity and/or increased risk of fractures, such as rickets, osteopetrosis, idiopathic juvenile osteoporosis, or skeletal dysplasia * History of known cardiovascular disease such as coronary artery anomaly, Kawasaki disease, myocarditis, cardiomyopathy, myocardial infarction, stroke, or thromboembolic disease. Individuals with other congenital or acquired cardiovascular disease necessitating echocardiogram require Medical Monitor review. Investigators should consider whether the potential benefits of treatment outweigh the potential risks in patients with cardiovascular risk factors such as confirmed arterial hypertension. * Hypocalcemia, defined as serum calcium levels below the age-adjusted normal limit reference ranges after a recommended ≥ 4 hour fast, at Screening * Estimated glomerular filtration rate \<=35 mL/min/1.73 m2 at Screening * Prior treatment with growth hormone, denosumab, anti-sclerostin antibody, or other anabolic or anti-resorptive medications impacting the bone (other than bisphosphonates) at any time * History of external radiation therapy * Known hypersensitivity to setrusumab or its excipients that, in the judgment of the Investigator, places the subject at increased risk for adverse effects * 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 * Use of any investigational product or investigational medical device within 4 weeks or 5 half-lives (whichever is longer) of investigational drug 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 study Medical Monitor
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Azienda Ospedaliera Universitaria Policlinico Umberto I
Roma, 00161, Italy
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Baylor College of Medicine
Houston, Texas, 77030, United States
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Children's Hospital at London Health Sciences Centre
London, Ontario, N6A 5W9, Canada
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Children's National Hospital DC
Washington D.C., District of Columbia, 20010, United States
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Childrens Hospital LA
Los Angeles, California, 90027, United States
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Childrens Hospital Of Eastern Ontario Research Institute, University Of Ottawa
Ottawa, KIH 8L1, Canada
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Cook Children's Medical Center
Fort Worth, Texas, 76104, United States
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Hospital de Clinicas de Porto Alegre (HCPA)
Porto Alegre, Rio Grande do Sul, 90035-903, Brazil
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Institut Imagine
Paris, 75015, France
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Nemours/ Alfred i. duPoint Hospital for Children
Wilmington, Delaware, 19803, United States
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Phoenix Children's Hospital
Phoenix, Arizona, 85206, United States
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Shriners Hospitals for Children Chicago
Chicago, Illinois, 60707, United States
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Universitair Medisch Centrum Utrecht (UMCU) - Wilhelmina Kinderziekenhuis
Utrecht, 3584 EA, Netherlands
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University of North Carolina at Chapel Hill (UNC)
Chapel Hill, North Carolina, 27599, United States
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University of South Florida
Tampa, Florida, 33612, United States
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Uniwersytet Medyczny w Lodzi - Klinika Endokrynologii i Chorob Metabolicznych
Lodz, 91-738, Poland
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Vanderbilt University Medical Center (VUMC)
Nashville, Tennessee, 37212, United States
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Washington University School of Medicine
St Louis, Missouri, 63110, United States
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Yale New Haven Hospital
New Haven, Connecticut, 06510, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can genetics predict the course of brittle bone disease?
- Dental scans and AI could spot rare bone diseases faster
- Wearable sensors shed light on movement in brittle bone disease
- BONeMOVE: exercise boosts stamina in kids with brittle bones
- New shot aims to toughen fragile bones in rare disease
- Gentle exercise with cuffs may strengthen bones and muscles in brittle bone disease