Study of OXB-102 (AXO-Lenti-PD) in Patients With Bilateral, Idiopathic Parkinson's Disease
- Conditions
- Parkinson Disease
- Interventions
- Other: Imitation Surgical Procedure (ISP)
- Registration Number
- NCT03720418
- Lead Sponsor
- Sio Gene Therapies
- Brief Summary
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.
- Detailed Description
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).
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 6
- Diagnosed with bilateral idiopathic PD
- Males/females between 30 and 70 years at the time of surgery
- Unified Parkinson's Disease Rating Scale (UPDRS) (Part III) score of between 30 and 60 in the "OFF" medication state
- Presence of motor fluctuations and/or dyskinetic movement
- Candidate for surgical intervention
- Hoehn and Yahr (H&Y) Stage 3 or 4 in the "OFF" medication state
- Stable dosing of PD medication, including L-DOPA, for four weeks prior to screening with Levodopa equivalent daily dose (LEDD) of at least 900 mg
Key
- History of psychosis or current treatment with dopamine blocking agents and prior regular exposure to antipsychotic agents
- History of stereotactic or other surgery for the treatment of PD, including Deep Brain Stimulation (DBS)
- Participation in a prior cell or gene transfer therapy study
- Contraindications to use of anaesthesia
- Current or anticipated treatment with anticoagulant therapy or the use of anticoagulation therapy that cannot be temporarily stopped around the time of surgery
- Diagnosis of multiple system atrophy
- Abnormal MRI findings such as mega cisterna, septum pellucidum, signs of severe cortical or subcortical atrophy, brain tumours, vascular diseases, trauma or arteriovenous malformations
- Presence of dementia
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description OXB-102 Dose Level 2 OXB-102 OXB-102 Dose Level 2 Single Administration (Part A: open-label) OXB-102 Dose Level 3 OXB-102 OXB-102 Dose Level 3 Single Administration (Part A: open-label) Imitation Surgical Procedure Imitation Surgical Procedure (ISP) General anesthesia with bilateral skin incisions (Part B: double-blind) OXB-102 Selected Dose OXB-102 Selected Dose of OXB-102 Single Administration (Part B: double-blind) OXB-102 Dose Level 1 OXB-102 OXB-102 Dose Level 1 Single Administration (Part A: open-label)
- Primary Outcome Measures
Name Time Method Safety of OXB-102 as measured by changes in vital signs 3 months timepoint Number of clinically significant changes in vital signs
Safety of OXB-102 as measured by changes in clinical laboratory analysis 3 months timepoint Number of clinically significant changes in clinical laboratory analysis
Safety of OXB-102 as measured by changes in brain MRI findings 3 months timepoint Number of clinically significant changes in brain MRI findings
Safety of OXB-102 as measured by incidence of treatment emergent adverse events and serious adverse events 3 months timepoint 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
Safety of OXB-102 as measured by changes in physical examination 3 months timepoint Number of clinically significant changes in physical examination
- Secondary Outcome Measures
Name Time Method Change in Unified Parkinson's Disease Rating Scale (UPDRS) scores defined in "OFF" and "ON" medication states Baseline to 6 months Change in dyskinesia rating scale score Baseline to 6 months Change in "OFF" time during waking day compared to baseline as assessed by participant diaries Baseline to 6 months
Trial Locations
- Locations (3)
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