Clinical trial NCT03720418: Study of OXB-102 (AXO-Lenti-PD) in Patients With Bilateral, Idiopathic Parkinson's Disease
Official title: A Phase I/II Safety and Dose Evaluation Study of OXB-102 (AXO-Lenti-PD) in Patients With Bilateral Idiopathic Parkinson's Disease (SUNRISE-PD)
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE1
PHASE2
Conditions: Parkinson Disease
Keywords: Parkinson's disease
Gene therapy
OXB-102
AXO-Lenti-PD
Lead sponsor: Sio Gene Therapies
Sponsor class: INDUSTRY
First posted: 2018-10-25
Enrollment: 6
Sex: ALL
Minimum age (years): 30
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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).

Eligibility criteria: Key Inclusion Criteria:

1. Diagnosed with bilateral idiopathic PD
2. Males/females between 30 and 70 years at the time of surgery
3. Unified Parkinson's Disease Rating Scale (UPDRS) (Part III) score of between 30 and 60 in the "OFF" medication state
4. Presence of motor fluctuations and/or dyskinetic movement
5. Candidate for surgical intervention
6. Hoehn and Yahr (H\&Y) Stage 3 or 4 in the "OFF" medication state
7. 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 Exclusion Criteria:

1. History of psychosis or current treatment with dopamine blocking agents and prior regular exposure to antipsychotic agents
2. History of stereotactic or other surgery for the treatment of PD, including Deep Brain Stimulation (DBS)
3. Participation in a prior cell or gene transfer therapy study
4. Contraindications to use of anaesthesia
5. Current or anticipated treatment with anticoagulant therapy or the use of anticoagulation therapy that cannot be temporarily stopped around the time of surgery
6. Diagnosis of multiple system atrophy
7. Abnormal MRI findings such as mega cisterna, septum pellucidum, signs of severe cortical or subcortical atrophy, brain tumours, vascular diseases, trauma or arteriovenous malformations
8. Presence of dementia

Primary outcomes:
measure: Safety of OXB-102 as measured by incidence of treatment emergent adverse events and serious adverse events
timeFrame: 3 months timepoint
measure: Safety of OXB-102 as measured by changes in clinical laboratory analysis
timeFrame: 3 months timepoint
measure: Safety of OXB-102 as measured by changes in vital signs
timeFrame: 3 months timepoint
measure: Safety of OXB-102 as measured by changes in brain MRI findings
timeFrame: 3 months timepoint
measure: Safety of OXB-102 as measured by changes in physical examination
timeFrame: 3 months timepoint
Secondary outcomes:
measure: Change in Unified Parkinson's Disease Rating Scale (UPDRS) scores defined in "OFF" and "ON" medication states
timeFrame: Baseline to 6 months
measure: Change in "OFF" time during waking day compared to baseline as assessed by participant diaries
timeFrame: Baseline to 6 months
measure: Change in dyskinesia rating scale score
timeFrame: Baseline to 6 months
Other outcomes:
Locations:
city: Créteil
country: France
facility: Service de Neurochirurgie, Hôpital Henri Mondor
city: Cambridge
country: United Kingdom
facility: University of Cambridge, Centre for Brain Repair
state: Cambridgeshire
city: London
country: United Kingdom
facility: The National Hospital for Neurology and Neurosurgery
Study officials:
affiliation: Sio Gene Therapies
name: Erika De Boever, DDS, PhD
role: STUDY_DIRECTOR
References:
citation: McFarthing K, Prakash N, Simuni T. CLINICAL TRIAL HIGHLIGHTS: 1. GENE THERAPY FOR PARKINSON'S, 2. PHASE 3 STUDY IN FOCUS - INTEC PHARMA'S ACCORDION PILL, 3. CLINICAL TRIALS RESOURCES. J Parkinsons Dis. 2019;9(2):251-264. doi: 10.3233/JPD-199001. No abstract available.