A Study of AAV2-GDNF in Adults with Moderate Parkinson’s Disease (REGENERATE-PD)
Research type
Research Study
Full title
A Phase 2, Randomized, Double-blind, Sham Surgery-controlled Study of the Efficacy and Safety of Intraputaminal AAV2-GDNF in the Treatment of Adults with Moderate Stage Parkinson’s Disease
IRAS ID
1009652
Contact name
Albana Dalipi
Contact email
Sponsor organisation
Asklepios BioPharmaceutical, Inc.
Clinicaltrials.gov Identifier
Research summary
REGENERATE-PD is a trial investigating the clinical benefits and safety of a new gene therapy for Parkinson’s (PD). Gene therapy treats a disease at the cell level by changing or fixing how the cell functions. In this case, researchers are delivering instructions to the cell to produce more of the so-called GDNF (glial cell line-derived neurotrophic factor). We think that increasing the amount of GDNF in the brain of people with PD may help slow down or stop the loss of brain cells, which causes PD.
We will test participants to make sure it is safe for them to take part in the trial. Those who are able to take part will be assigned, at random, to receive the GDNF gene therapy (67% of participants) or be in the control group (33% of participants).
Those assigned to receive the GDNF gene therapy will undergo an operation on their brain. A needle will be inserted into the area of the brain important in the development of PD and the gene therapy will be infused into the brain. This operation can be between 6 – 10 hours. Once the gene therapy is infused it cannot be removed.
Participants in the control group will undergo a control surgery. All the same steps as the treatment group are followed, as far as drilling partial holes in the skull (i.e. the holes do not go all the way through the skull).
All surgeries will happen at the surgical study site (in Cardiff).
All study participants will need to visit the study site (Bristol Brain Centre) for assessments before (over approximately 4 months) and after the surgery. There will be up to 20 visits, some of which can be conducted remotely by video/telephone calls. Assessments will include brain imaging, questionnaires, blood tests, medical assessment of the participants general health and PD symptoms.
The study will end when the last participant completes month 18 visit. Therefore, the minimum study duration will be 18 months, but could be longer (e.g., 4 years).REC name
South Central - Oxford A Research Ethics Committee
REC reference
24/SC/0125
Date of REC Opinion
24 Jun 2024
REC opinion
Further Information Favourable Opinion