NCT05125809

Setrusumab vs Placebo for Osteogenesis Imperfecta

An Operationally Seamless, Randomized Phase 2/3 Study Consisting of a Phase 2 Single-Blind, Dose-Evaluation Phase and a Phase 3 Double-Blind, Placebo-Controlled Phase to Assess the Efficacy and Safety of Setrusumab in Subjects With Osteogenesis Imperfecta

• Status: Active Not Recruiting
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 183 (actual)
• Sponsor: Ultragenyx Pharmaceutical Inc · Industry
• Sex: All
• Age: 5 Years – 25 Years
• Healthy volunteers: Not accepted

Summary

The primary objectives of the study are to identify a setrusumab dosing strategy in participants with OI and to evaluate the effect of setrusumab vs placebo on reduction in fracture rate.

Detailed description

Participants in Phase 2 will be randomized 1:1 to receive low dose or high dose setrusumab. Phase 2 participants will continue receiving their assigned dose of setrusumab until all Phase 2 participants have completed the Month 6 study visit. After this point, Phase 2 participants will begin receiving the selected dosing strategy in the Phase 2 open-label Treatment Extension Period. Phase 3 participants will be randomized 2:1 to receive setrusumab or placebo during the double-blind treatment period. Phase 3 participants will transition to the open-label Treatment Extension Period after the end of the double-blind period. Participants in the Phase 2 and Phase 3 treatment extension periods will receive open-label setrusumab treatment for at least 12 months, and have the option to remain in the open-label treatment period until setrusumab is commercially available in their region. An optional substudy will be conducted in approximately 10 participants (≥ 8 years) consisting of a bone biopsy to investigate the impact of setrusumab on bone histomorphology.

Conditions

• Osteogenesis Imperfecta

Interventions

Type	Name	Description
BIOLOGICAL	Setrusumab	A fully human sclerostin neutralizing monoclonal antibody (mAb) administered once a month (QM) via intravenous (IV) infusion
OTHER	Placebo	A 5% dextrose/glucose solution administered QM via IV infusion

Timeline

• Start date: 2022-02-21
• Primary completion: 2025-10-20
• Completion: 2027-04-01
• First posted: 2021-11-18
• Last updated: 2026-02-25

Locations

44 sites across 11 countries: United States, Australia, Canada, France, Germany, Italy, Netherlands, Poland, Portugal, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study