A Randomized, Placebo Surgery Controlled, Double-Blinded, Multi-Center, Phase 2 Clinical Trial, Evaluating the Efficacy and Safety of VY-AADC02 in Advanced Parkinson's Disease with Motor Fluctuations (RESTORE-1)
Anna Morenkova, MD, PhD
UCI Main Campus Irvine, CA; UCSF Medical Center San Francisco, CA; San Francisco Veteran's Affairs Medical Center, San Francisco, CA
The purpose of this research study is to test the safety and efficacy (how well it works) of an experimental intervention called VY-AADC02 on the severity of PD. VY-AADC02 involves placing a gene in the brain that programs brain cells to produce an enzyme (protein) called AADC (aromatic amino acid decarboxylase).
You can participate in this study if you
- Are male or female, between 40 and 75 years of age, inclusive.
- Have been diagnosed with Parkinson's disease for at least 4 years.
- Are responsive to dopaminergic therapy (i.e. carbidopa/levodopa).
- On stable doses of Parkinson's disease medication regimens for at least 4 weeks.
- Have an average of at least 3 hours of "OFF" time per day over the course of 3 consecutive days.
You cannot participate in this study if you
- Have been diagnosed with atypical or secondary parkinsonism.
- Have a history of psychosis or moderate depression.
- Have a medical condition that can lead to disability and interfere with the study (i.e. stroke, orthopedic condition, spinal disorders, neuropathy, myelopathy, severe pulmonary or cardiovascular disease, cognitive decline).
- Have a history of substance abuse (i.e. alcohol, drugs) in the last 2 years.
- Have had any prior complicated brain surgery or infusion therapy.
- Plan to receive any vaccination within 30 days of the surgery.
- Have any ongoing or planned treatments that might interfere with the study (i.e. deep brain stimulation).
- Are pregnant or lactating.
Your participation in this study will last approximately 12 months. While you are in the study, you will have approximately 17 visits to the study center. Your PD medications have to remain unchanged for at least 4 weeks prior to your participation in the study. The study will involve a total of approximately 10 visits. 9 of the visits will be at the study center, and 1 visit (the surgery visit) will be done at the surgical center.
Upon completion of the study, participants who meet eligibility requirements will be invited to enroll in a long-term extension study where they will be followed for up to an additional 8 years. Participation in the follow-up study is not required for participation in this study.
Taking part in this study may or may not make your health better. Your condition may get better, it may get worse, or it may stay the same. While researchers hope VY-AADC02 will be more effective than the standard (usual) treatment, there is no proof of this yet.
If you are in the group that receives VY-AADC02 and it proves to treat your condition more effectively/with fewer side effects than standard therapy/placebo, you may benefit from participating in the study, but this cannot be guaranteed.
You will be paid $150 for the surgical, Day 180 and Day 360 visits, $100 for the screening and baseline visits, and $50 for all other visits that you complete. Total compensation for participation in the entire study is $900. If you decide to withdraw from the study or are withdrawn by the research team, you will receive compensation for the visits and/or procedures that you have completed.
Breana Chew, CCR-Neurology