Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta

Running, not enrolling · Phase 3

Conditions studied: Osteogenesis Imperfecta

In brief

The primary objective of the study is to evaluate the effect of setrusumab vs intravenous bisphosphonates (IV-BP) on reduction in fracture rate, including morphometric vertebral fractures in pediatric participants.

Key facts

Study ID

NCT05768854

Run by

Ultragenyx Pharmaceutical Inc

People needed

69

Starts

2023-06-14

Expected to finish

2027-04-01

Last updated by the study team

2026-02-25

Who can join

Age: 2 and older, up to 6. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Phoenix Children's Hospital — Phoenix, Arizona, United States
• Childrens Hospital LA — Los Angeles, California, United States
• Children's Hospital Colorado — Aurora, Colorado, United States
• Yale New Haven Hospital — New Haven, Connecticut, United States
• Nemours/ Alfred i. duPoint Hospital for Children — Wilmington, Delaware, United States
• Children's National Hospital DC — Washington D.C., District of Columbia, United States
• University of South Florida — Tampa, Florida, United States
• Shriners Hospitals for Children Chicago — Chicago, Illinois, United States
• Washington University School of Medicine — St Louis, Missouri, United States
• University of North Carolina at Chapel Hill (UNC) — Chapel Hill, North Carolina, United States
• Vanderbilt University Medical Center (VUMC) — Nashville, Tennessee, United States
• Cook Children's Medical Center — Fort Worth, Texas, United States
• Baylor College of Medicine — Houston, Texas, United States
• Hospital de Clinicas de Porto Alegre (HCPA) — Porto Alegre, Rio Grande do Sul, Brazil
• Children's Hospital at London Health Sciences Centre — London, Ontario, Canada
• Childrens Hospital Of Eastern Ontario Research Institute, University Of Ottawa — Ottawa, Canada
• Institut Imagine — Paris, France
• Azienda Ospedaliera Universitaria Policlinico Umberto I — Roma, Italy
• Universitair Medisch Centrum Utrecht (UMCU) - Wilhelmina Kinderziekenhuis — Utrecht, Netherlands
• Uniwersytet Medyczny w Lodzi - Klinika Endokrynologii i Chorob Metabolicznych — Lodz, Poland

Full record on ClinicalTrials.gov

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