Study of OXB-102 (AXO-Lenti-PD) in Patients With Bilateral, Idiopathic Parkinson's Disease

Parkinson Disease Clinical Trial

— SUNRISE-PD  

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)

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT03720418
• Other study ID #: OXB-102-01
• Status: Terminated
• Phase: Phase 1/Phase 2
• Start date: October 17, 2018
• Est. completion date: April 12, 2022

Study information

• Verified date: April 2022
• Source: Sio Gene Therapies
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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 information / eligibility

• Status: Terminated
• Enrollment: 6
• Est. completion date: April 12, 2022
• Est. primary completion date: April 12, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 30 Years to 70 Years

Eligibility

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

Related Conditions & MeSH terms

• Parkinson Disease

Intervention

Drug:
• OXB-102
Neurosurgical delivery of OXB-102 (gene therapy) to the putamen

Other:
• Imitation Surgical Procedure (ISP)
Participants randomized to the control group in Part B will receive an ISP

Locations

Country	Name	City	State
France	Service de Neurochirurgie, Hôpital Henri Mondor	Créteil
United Kingdom	University of Cambridge, Centre for Brain Repair	Cambridge	Cambridgeshire
United Kingdom	The National Hospital for Neurology and Neurosurgery	London

Sponsors (1)

Lead Sponsor	Collaborator
Sio Gene Therapies

Countries where clinical trial is conducted

France,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	3 months timepoint
Primary	Safety of OXB-102 as measured by changes in clinical laboratory analysis	Number of clinically significant changes in clinical laboratory analysis	3 months timepoint
Primary	Safety of OXB-102 as measured by changes in vital signs	Number of clinically significant changes in vital signs	3 months timepoint
Primary	Safety of OXB-102 as measured by changes in brain MRI findings	Number of clinically significant changes in brain MRI findings	3 months timepoint
Primary	Safety of OXB-102 as measured by changes in physical examination	Number of clinically significant changes in physical examination	3 months timepoint
Secondary	Change in Unified Parkinson's Disease Rating Scale (UPDRS) scores defined in "OFF" and "ON" medication states		Baseline to 6 months
Secondary	Change in "OFF" time during waking day compared to baseline as assessed by participant diaries		Baseline to 6 months
Secondary	Change in dyskinesia rating scale score		Baseline to 6 months