This study consists of two parts. Part A will evaluate the safety and tolerability of multiple doses of OXB-102 (AXO-Lenti-PD) in participants with Parkinson's disease. Part B will assess the safety and efficacy of the selected dose of OXB-102 in participants with Parkinson's disease.
This study consists of two parts. Part A is an open-label dose-escalation phase in which participants are enrolled in cohorts and will receive one of approximately three escalating doses of OXB-102 (AXO-Lenti-PD). Part B is a randomized, double-blind phase in which participants will be randomized to either an active group receiving the selected dose from Part A, or to a control group receiving an imitation surgical procedure (ISP).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
6
Neurosurgical delivery of OXB-102 (gene therapy) to the putamen
Participants randomized to the control group in Part B will receive an ISP
Service de Neurochirurgie, Hôpital Henri Mondor
Créteil, France
University of Cambridge, Centre for Brain Repair
Cambridge, Cambridgeshire, United Kingdom
The National Hospital for Neurology and Neurosurgery
London, United Kingdom
Safety of OXB-102 as measured by incidence of treatment emergent adverse events and serious adverse events
Treatment emergent adverse events and serious adverse events will be assessed by National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE), version 4.0, for severity
Time frame: 3 months timepoint
Safety of OXB-102 as measured by changes in clinical laboratory analysis
Number of clinically significant changes in clinical laboratory analysis
Time frame: 3 months timepoint
Safety of OXB-102 as measured by changes in vital signs
Number of clinically significant changes in vital signs
Time frame: 3 months timepoint
Safety of OXB-102 as measured by changes in brain MRI findings
Number of clinically significant changes in brain MRI findings
Time frame: 3 months timepoint
Safety of OXB-102 as measured by changes in physical examination
Number of clinically significant changes in physical examination
Time frame: 3 months timepoint
Change in Unified Parkinson's Disease Rating Scale (UPDRS) scores defined in "OFF" and "ON" medication states
Time frame: Baseline to 6 months
Change in "OFF" time during waking day compared to baseline as assessed by participant diaries
Time frame: Baseline to 6 months
Change in dyskinesia rating scale score
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Time frame: Baseline to 6 months