Hackensack Participates in Gene Therapy Clinical Trial for Parkinsons Patients

What you need to know

The Division of Movement Disorders at the Departments of Neurology and Neurosurgery at Hackensack Meridian Health University Medical Center have been selected as one of multiple medical centers in the United States to participate in the RESTORE-1 Phase 2 Clinical Study of VY-AADC, an investigational gene therapy for Parkinson’s disease. The randomized, double-blinded, placebo-surgery controlled trial, RESTORE-1, will evaluate the safety and efficacy of the surgically delivered VY-AADC for people with advancing Parkinson’s disease.

Recruiting participants

The hospital is recruiting participants ages 40-75, male and female, who have been diagnosed with Parkinson’s disease for four years or more and who have been experiencing unpredictable motor functions despite oral medications.  The study will involve two months of pre-treatment assessment and 12 months of follow-up visits.

VY-AADC gene therapy

VY-AADC is an investigational gene therapy designed to put the AADC enzyme into brain cells where it can convert levodopa to dopamine. To do this, the AADC gene is delivered inside a transporter called "adeno-associated viral vector" (AAV). The investigational one-time therapy is delivered directly to the putamen, a part of the brain that helps control motor function, during a neurosurgical procedure monitored with real-time MRI imaging.

For more information

For more information about the RESTORE-1 clinical trial, including eligibility criteria, please visit restore1study.com or clinicaltrials.gov.