NCT05768854

Brief Summary

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.

Trial Health

82
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
69

participants targeted

Target at below P25 for phase_3

Timeline
11mo left

Started Jun 2023

Typical duration for phase_3

Geographic Reach
7 countries

20 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress76%
Jun 2023Apr 2027

First Submitted

Initial submission to the registry

March 3, 2023

Completed
11 days until next milestone

First Posted

Study publicly available on registry

March 14, 2023

Completed
3 months until next milestone

Study Start

First participant enrolled

June 14, 2023

Completed
2.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 23, 2025

Completed
1.4 years until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2027

Expected
Last Updated

February 25, 2026

Status Verified

February 1, 2026

Enrollment Period

2.4 years

First QC Date

March 3, 2023

Last Update Submit

February 24, 2026

Conditions

Osteogenesis Imperfecta

Outcome Measures

Primary Outcomes (1)

  • Annualized Rate of All Radiographically-Confirmed Fractures, Including Morphometric Vertebral Fractures, at the Primary Analysis

    Up to 24 Months

Secondary Outcomes (9)

  • Annualized Rate of Radiographically-Confirmed Fractures, Excluding Morphometric Vertebral Fractures, but Including Fractures of the Fingers, Toes, Face and Skull at the Primary Analysis

    Up to 24 Months

  • Annualized Rate of All Radiographically-Confirmed Fractures, Excluding Morphometric Vertebral Fractures, and Fractures of the Fingers, Toes, Face and Skull, at the Primary Analysis

    Up to 24 Months

  • Change from Baseline in Dual-energy X-ray Absorptiometry (DXA) Bone Mineral Density (BMD) Z-score at the Lumbar Spine at the Primary Analysis

    Up to 24 Months

  • Proportion of Participants Experiencing New Radiographically-Confirmed Fractures, including Morphometric Vertebral Fractures, at the Primary Analysis

    Up to 24 Months

  • Change from Baseline in Pediatric Orthopedic Society of North America Pediatric Outcomes Data Collection Instrument (POSNA-PODCI) Sports/Physical Functioning and Pain/Comfort Subscale Scores at the Primary Analysis

    Baseline, Up to 24 Months

  • +4 more secondary outcomes

Study Arms (2)

Intravenous Bisphosphonates (IV-BP) -> Setrusumab

ACTIVE COMPARATOR

Participants on IV-BP will continue their existing dose/regimen per investigator discretion; for participants not on IV-BP, the dose/regimen will be determined by the investigator. After the active-controlled period, participants will receive Setrusumab during the extension period

Drug: BisphosphonateBiological: Setrusumab

Setrusumab

EXPERIMENTAL

Participants will receive Setrusumab during the active-controlled and extension period

Biological: Setrusumab

Interventions

BisphosphonateDRUG

Administered per investigator discretion via intravenous (IV) infusion

Intravenous Bisphosphonates (IV-BP) -> Setrusumab
SetrusumabBIOLOGICAL

A fully human sclerostin neutralizing monoclonal antibody (mAb) administered once a month (QM) via intravenous (IV) infusion

Also known as: BPS804, UX143
Intravenous Bisphosphonates (IV-BP) -> SetrusumabSetrusumab

Eligibility Criteria

Age2 Years - 6 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (20)

Phoenix Children's Hospital

Phoenix, Arizona, 85206, United States

Location

Childrens Hospital LA

Los Angeles, California, 90027, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Yale New Haven Hospital

New Haven, Connecticut, 06510, United States

Location

Nemours/ Alfred i. duPoint Hospital for Children

Wilmington, Delaware, 19803, United States

Location

Children's National Hospital DC

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

Location

University of South Florida

Tampa, Florida, 33612, United States

Location

Shriners Hospitals for Children Chicago

Chicago, Illinois, 60707, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

University of North Carolina at Chapel Hill (UNC)

Chapel Hill, North Carolina, 27599, United States

Location

Vanderbilt University Medical Center (VUMC)

Nashville, Tennessee, 37212, United States

Location

Cook Children's Medical Center

Fort Worth, Texas, 76104, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Hospital de Clinicas de Porto Alegre (HCPA)

Porto Alegre, Rio Grande do Sul, 90035-903, Brazil

Location

Children's Hospital at London Health Sciences Centre

London, Ontario, N6A 5W9, Canada

Location

Childrens Hospital Of Eastern Ontario Research Institute, University Of Ottawa

Ottawa, KIH 8L1, Canada

Location

Institut Imagine

Paris, 75015, France

Location

Azienda Ospedaliera Universitaria Policlinico Umberto I

Roma, 00161, Italy

Location

Universitair Medisch Centrum Utrecht (UMCU) - Wilhelmina Kinderziekenhuis

Utrecht, 3584 EA, Netherlands

Location

Uniwersytet Medyczny w Lodzi - Klinika Endokrynologii i Chorob Metabolicznych

Lodz, 91-738, Poland

Location

Related Links

MeSH Terms

Conditions

Osteogenesis Imperfecta

Interventions

Diphosphonatessetrusumab

Condition Hierarchy (Ancestors)

OsteochondrodysplasiasBone Diseases, DevelopmentalBone DiseasesMusculoskeletal DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesCollagen DiseasesConnective Tissue DiseasesSkin and Connective Tissue Diseases

Intervention Hierarchy (Ancestors)

OrganophosphonatesOrganophosphorus CompoundsOrganic Chemicals

Study Officials

  • Medical Director

    Ultragenyx Pharmaceutical Inc

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 3, 2023

First Posted

March 14, 2023

Study Start

June 14, 2023

Primary Completion

October 23, 2025

Study Completion (Estimated)

April 1, 2027

Last Updated

February 25, 2026

Record last verified: 2026-02

Data Sharing

IPD Sharing
Will not share

Locations

CA(2)BR(1)NL(1)US(13)PL(1)IT(1)FR(1)