NCT05768854

Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta

An Open-label, Randomized, Active-Controlled, Phase 3 Study of Setrusumab Compared With Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta Types I, III or IV

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 69 (actual)
• Sponsor: Ultragenyx Pharmaceutical Inc · Industry
• Sex: All
• Age: 2 Years – 6 Years
• Healthy volunteers: Not accepted

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.

Detailed description

Participants will be randomized 1:1 to receive either setrusumab or IV-BP. Following randomization, participants will receive setrusumab or IV-BP for up to 24 months during the Active-controlled Period. At the end of the Active-controlled Period all participants will enter the Extension Period and participants assigned to IV-BP will transition to setrusumab. During the Extension Period, all participants will receive setrusumab for a minimum of 12 months or until setrusumab becomes commercially available in their respective country or the study is discontinued. The use of any bisphosphonate is prohibited throughout the Extension Period.

Conditions

• Osteogenesis Imperfecta

Interventions

Type	Name	Description
DRUG	Bisphosphonate	Administered per investigator discretion via intravenous (IV) infusion
BIOLOGICAL	Setrusumab	A fully human sclerostin neutralizing monoclonal antibody (mAb) administered once a month (QM) via intravenous (IV) infusion

Timeline

• Start date: 2023-06-14
• Primary completion: 2025-10-23
• Completion: 2027-04-01
• First posted: 2023-03-14
• Last updated: 2026-02-25

Locations

20 sites across 7 countries: United States, Brazil, Canada, France, Italy, Netherlands, Poland

Regulatory

• FDA-regulated drug study