A Study of OXB-102, a Gene Therapy, for Parkinsons Disease
Research type
Research Study
Full 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).
IRAS ID
202977
Contact name
Roger Barker
Contact email
Sponsor organisation
Axovant Sciences GmbH
Eudract number
2015-002717-29
Duration of Study in the UK
15 years, 0 months, 0 days
Research summary
OXB-102 is a second generation gene therapy product in development at Axovant Sciences GmbH for the treatment of patients with Parkinson’s disease (PD), a progressive neurodegenerative disorder of the central nervous system. The rationale for treatment is to provide dopamine replacement to the dopamine-depleted putamen in the brain of PD patients by gene transfer of 3 critical enzymes involved in dopamine production. OXB-102 will be administered into the brain by infusion during surgery and is hoped to provide a constant replacement of dopamine. This has the potential for patients to reduce their L-DOPA and dopamine agonist therapy, restore their movement control and have shorter “OFF” medication periods.
Study OXB-102-01 is a first in man, safety and dose evaluation study in patients with bilateral advanced PD who are not adequately controlled by medication. The study is in two parts: Part A is a non-randomised dose escalation which will evaluate safety, tolerability and signs of biological effect in up to three doses of OXB-102 (minimum of 2 patients in cohort 1, minimum of 4 patients in cohort 2 & 3, with the option to expand to 6 patients) in order to determine an optimal dose in Part B, a randomised, double blind controlled part. Twelve patients will be randomised in Part B, (2:1) to receive active treatment (OXB-102) or an imitation surgical procedure as a control group. Progression through the dose levels and from Part A to Part B will occur only when safety and tolerability data are assessed by an independent data monitoring committee. In accordance with gene therapy guidelines patients will be monitored in this study for 15 years.
Two NHS hospital site's in the UK and one site in France will take part, two of those sites, previously undertook a similar study with the first-generation gene therapy product, ProSavin®.REC name
South Central - Oxford A Research Ethics Committee
REC reference
16/SC/0201
Date of REC Opinion
14 Jul 2016
REC opinion
Further Information Favourable Opinion