Phase 2b Dose Finding Study in Adult Patients Osteogenesis Imperfecta
Research type
Research Study
Full title
A Phase 2b, Multicentre, Multinational, Placebo-controlled, Double-blind, Dose-finding Study in Adult Patients with Type I, III or IV Osteogenesis Imperfecta Treated with BPS804
IRAS ID
222778
Contact name
Muhammad Kassim Javaid
Contact email
Sponsor organisation
Mereo BioPharma Group Limited
Eudract number
2016-005096-27
Duration of Study in the UK
0 years, 10 months, 1 days
Research summary
Research Summary
This is a phase 2b study for adults suffering from Osteogenesis Imperfecta (OI). Patients that suffer from OI have a genetic disorder that is characterised by fragile bones that break easily, often with little or no physical trauma. It has been shown that patients with OI have a higher mortality rate.
Current management of OI is directed towards preventing or controlling symptoms, maximising independent mobility and improving bone mass and muscle strength. Current interventions include exercise and physical therapy, rehabilitation, and pain management. Surgery may prove necessary for individuals with severe deformities or basilar impression. Rodding, a procedure in which metal rods are surgically placed in the long bones of patients with OI to prevent fractures, is often used to treat individuals with OI. However, none of these treatments affect the underlying bone pathology.The drug being investigated in this study is BPS804, a monoclonal antibody. Based on significantly increased bone formation in untreated patients with OI, It is hypothesised that OI patients who receive BPS804 will experience an increase in bone mineral density, an improvement in bone structure and strength, and hence a reduction in bone fracture incidence.
The study is split into three sections, screening – to check for eligibility, treatment and follow up. If a patient is deemed eligible according to the screening criteria they will be randomised 1:1:1:1 to 20mg/Kg, 8mg/Kg and 2mg/Kg BPS804 or to placebo. During the treatment phase, patients will complete monthly treatment cycles for 12 months. After the treatment phase, patients have the option of receiving BPS804 treatment in the MBPS2016 Does Maintenance Study or will complete a 60 day follow up period.
The study will take place at 5 NHS site in the UK and approximately 30 patients are expected to be enrolled. Approximately 140 patients will be recruited worldwide.
REC name
North East - Newcastle & North Tyneside 2 Research Ethics Committee
REC reference
17/NE/0117
Date of REC Opinion
6 Jun 2017
REC opinion
Further Information Favourable Opinion