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Clinical Trial 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.


Clinical Trial 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 (when the participant has completed 24 months in the double-blind period or when the Sponsor determines the timing of the primary analysis, whichever is sooner). 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 (≥ 8years) consisting of a bone biopsy following at least 12 months of setrusumab exposure to investigate the impact of setrusumab on bone histomorphology. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT05125809
Study type Interventional
Source Ultragenyx Pharmaceutical Inc
Contact Patient Contact: Trial Recruitment
Phone 1-888-756-8657
Email trialrecruitment@ultragenyx.com
Status Recruiting
Phase Phase 2/Phase 3
Start date February 21, 2022
Completion date March 2026

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