Long-term Extension Study of Setrusumab in Adults With Type I, III, or IV Osteogenesis Imperfecta

NCT05312697 | Terminated Phase 2 Results DMC FDA Drug

• 1 other identifier: UX143-CL203
• Study Type: interventional
• Target: 2
• Locations: 1 country
• Sites: 1

Brief Summary

The primary objective of the study is to evaluate bone mineral density (BMD) after 12 months of retreatment with monthly setrusumab in adults with osteogenesis imperfecta (OI).

Conditions

Osteogenesis Imperfecta

Outcome Measures

Primary Outcomes (1)

• Percentage Change From Retreatment Baseline in Lumbar Spine Bone Mineral Density (BMD) Measured by Dual-Energy X-Ray Absorptiometry (DXA) After 12 Months of Setrusumab

  Retreatment Baseline, Month 12

Secondary Outcomes (2)

• Percentage Change From Retreatment Baseline in Total Hip BMD Measured by DXA at Month 12 of the Retreatment Period

  Retreatment Baseline, Month 12

• Annualized New Fracture Rate as Confirmed by Radiograph During the Retreatment Period

  Up to Month 12

Study Arms (2)

Setrusumab QM -->Setrusumab QM

EXPERIMENTAL

During Retreatment Period: Setrusumab will be administered via intravenous (IV) infusion once a month (QM) for 12 months. During Extension Period: Setrusumab will be administered via IV infusion QM for at least 12 months or until commercially available.

Biological: Setrusumab

Setrusumab QM --> Setrusumab TBD

EXPERIMENTAL

During Retreatment Period: Setrusumab will be administered via IV infusion QM for 12 months. During Extension Period: Setrusumab will be administered via IV infusion at a dose frequency to be determined (TBD) for at least 12 months or until commercially available.

Biological: Setrusumab

Interventions

Setrusumab BIOLOGICAL

A fully human anti-sclerostin monoclonal antibody (mAb)

Also known as: UX143, BPS804

Setrusumab QM --> Setrusumab TBD; Setrusumab QM -->Setrusumab QM

Eligibility Criteria

• Age: 19 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Males or females who participated in the Phase 2b ASTEROID study
• Females of childbearing potential must consent to use highly effective contraception during the Observation, Retreatment, and Extension Periods through 2 months after the last dose of setrusumab and agree not to become pregnant

You may not qualify if:

• Known hypersensitivity to setrusumab or its excipients that, in the judgment of the Investigator, places the participant 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
• Pregnant or breastfeeding or planning to become pregnant (self or partner) at any time during the study
• Not willing or able to discontinue bisphosphonates 6 months prior to the Retreatment Screening Visit
• Concurrent participation in any study that is examining the safety and efficacy of any investigational product or investigational medical device that alters bone health during the Retreatment and Extension Periods, per discretion of the Investigator in consultation with the Medical Monitor.
• Calcium levels outside the normal range at the Retreatment Screening Visit. If the participant's calcium level is not within the normal range, decision to retest should be made in conjunction with the Medical Monitor
• Glomerular filtration rate (GFR) ≤ 29 mL/min at the Retreatment Screening Visit. If the participant's GFR is ≤ 29 mL/min, decision to retest should be made in conjunction with the Medical Monitor
• History of skeletal malignancies or bone metastases at any time
• History of neural foraminal stenosis (except if due to scoliosis)
• History of myocardial infarction, angina pectoris, ischemic stroke or transient ischemic attack (Investigators should consider whether the potential benefits of treatment outweigh the potential risks in patients with other cardiovascular risk factors such as hypertension, hyperlipidemia, familial hyperlipidemia, family history of premature ischemic cardiovascular disease, smoking, diabetes mellitus, and metabolic syndrome.)
• History of or concomitant uncontrolled diseases such as hypo-/hyperparathyroidism, hypo-/hyperthyroidism, Paget's disease, abnormal thyroid function or thyroid disease or other endocrine disorders or conditions that could affect bone metabolism eg, Stage IV/V renal disease
• A history of rickets or osteomalacia or any skeletal condition (other than OI) leading to long-bone deformities and/or increased risk of fractures
• 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/baseline assessments
• Documented history of significant psychiatric or medical disorder that would prevent the participant complying with the requirements of the protocol or would make it unsafe for the participant to participate in the study as judged by the investigator
• Current/previously reported allergy to the study drug or any of its excipients or the class of drug under investigation

Sponsors & Collaborators

• Ultragenyx Pharmaceutical Inc: lead
• Mereo BioPharma: collaborator

Study Sites (1)

New Mexico Clinical Research & Osteoporosis Center

Albuquerque, New Mexico, 87106, United States

Location

Related Links

• Ultragenyx Patient Advocacy/OI Disease Information

MeSH Terms

Conditions

Osteogenesis Imperfecta

Interventions

setrusumab

Condition Hierarchy (Ancestors)

Osteochondrodysplasias; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Collagen Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases

Results Point of Contact

• Title: Medical Information
• Organization: Ultragenyx Pharmaceutical Inc

medinfo@ultragenyx.com

1-888-756-8567

Study Officials

• Medical Director

  Ultragenyx Pharmaceutical Inc

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 28, 2022
• First Posted: April 5, 2022
• Study Start: April 28, 2022
• Primary Completion: July 7, 2022
• Study Completion: July 7, 2022
• Last Updated: July 23, 2025
• Results First Posted: July 23, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)