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

Sio Gene Therapies

Status and phase: Terminated; Phase 2; Phase 1

Conditions: Parkinson Disease

Treatments: Drug: OXB-102; Other: Imitation Surgical Procedure (ISP)

Study type: Interventional

Funder types: Industry

Identifiers

NCT03720418

OXB-102-01

Details and patient eligibility

About

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.

Full 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).

Enrollment: 6 patients

Sex: All

Ages: 30 to 70 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: Quadruple Blind

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6 participants in 5 patient groups

OXB-102 Dose Level 1

Experimental group

Description:

OXB-102 Dose Level 1 Single Administration (Part A: open-label)

Treatment:

Drug: OXB-102

OXB-102 Dose Level 2

Experimental group

Description:

OXB-102 Dose Level 2 Single Administration (Part A: open-label)

Treatment:

Drug: OXB-102

OXB-102 Dose Level 3

Experimental group

Description:

OXB-102 Dose Level 3 Single Administration (Part A: open-label)

Treatment:

Drug: OXB-102

OXB-102 Selected Dose

Experimental group

Description:

Selected Dose of OXB-102 Single Administration (Part B: double-blind)

Treatment:

Drug: OXB-102

Imitation Surgical Procedure

Sham Comparator group

Description:

General anesthesia with bilateral skin incisions (Part B: double-blind)

Treatment:

Other: Imitation Surgical Procedure (ISP)