UX143-CL301: A Phase 2/3 Study of Setrusumab in Subjects with OI
Research type
Research Study
Full title
A Randomized, Double-Blind, Placebo-controlled, Phase 2/3 Study to Assess the Efficacy and Safety of Setrusumab in Subjects with Osteogenesis Imperfecta.
IRAS ID
1005781
Contact name
Barbara Winslow
Contact email
Sponsor organisation
Ultragenyx Pharmaceutical Inc.
Eudract number
2021-006597-23
Clinicaltrials.gov Identifier
Research summary
This is a phase2/3 study into setrusumab, a new treatment for Osteogenesis Imperfecta (OI). OI is a rare genetic disorder of bone development resulting in bones that break easily, loose joints and weakened teeth and is sometimes called ‘brittle bone disease’. Osteogenesis imperfecta is a genetic bone disorder that is present at birth and is caused by changes in one of the two genes, COL1A1 or COL1A2.
Phase 2 of the study will look at the effects of setrusumab to find the best dose for treating OI and will see how well it is tolerated by comparing it to a placebo (a placebo is a treatment that looks like setrusumab but contains no active medicine).
Phase 3 of the study is to use the best dose level of setrusumab as determined during Phase 2 and to collect more information about how well it is tolerated and how well it works in treating OI (reducing fracture rate, and improving bone mineral density, health-related quality of life/physical health status).
The study will be conducted at multiple sites globally. The study doctor or study staff will tell participants which Phase they will take part in.
The duration of participation in the study will be a minimum of about 13 months and a maximum of about 25 months. All study participants will then enter the Open-label Treatment Extension Period, which will continue for at least 12 months or until setrusumab is available for general use in the UK (i.e., available with prescription through a pharmacy) whichever comes first. ‘Open-label’ means that both the participants and the study doctor will know that participants are receiving setrusumab. During the Open-label Treatment Extension, no one will be receiving placebo.
Study procedures will include: Physical examinations, vital signs, x-rays, DXA scans, ECGs, blood and urine tests and completion of questionnaires.REC name
Yorkshire & The Humber - Leeds West Research Ethics Committee
REC reference
22/YH/0168
Date of REC Opinion
2 Dec 2022
REC opinion
Further Information Favourable Opinion