Parkinson’s Disease Phase 2 Clinical Trial Currently Enrolling

A new Phase 2 Parkinson’s disease trial is now enrolling 42 patients who have lived with the condition four years or more. Ultimately, the trial aims to evaluate a new therapy for patients who have not had an adequate response to the typical medication used for Parkinson’s – levodopa, an oral medication. The trial will recruit across 7 states.

This trial is being conducted by Voyager Therapeutics. They have worked to investigate VY-AADC as a form of gene therapy in Parkinson’s.

The trial is called RESTORE-1 (NCT03562494).


VY-AADC has already received regenerative medicine advanced therapy (RMAT) designation by the FDA.

The therapy works to deliver the DDC gene to brain cells located in the putman region. The DDC gene tells the cells how to make the AADC enzyme which is necessary for converting levodopa into dopamine. Dopamine is the molecule which Parkinson’s disease patients lack.

The disease causes dopamine neurons to degenerate within the substantia nigra (a section of the midbrain). However, researchers believe they may be able to improve the symptoms of the condition by increasing dopamine within the putamen region of the brain.

Previous Research

So far, VY-AADC has had positive results in a Phase 1b clinical trial. This trial showed an improvement in motor skills for patients up to 3 years following treatment. Additionally, the treatment showed to increase AADC enzyme activity, which means that patients didn’t need as high of a dose of levodopa.

Most importantly, patients in this Phase 1b trial indicated a significantly improved quality of life.

These results stemmed from just one single administration of the therapy.

You can read more about the Phase 1b trial here.

Breakdown of the Phase 2 trial

The Phase 2 trial will evaluate the efficacy of the treatment by measuring AADC enzyme levels and activity. This will be examined using position emission tomography (PET). Additionally, any changes in participants’ dose of levodopa will be evaluated.

Patients enrolling in the trial must be 40 to 75 years of age and be experiencing a minimum of three hours each day where levodopa is not working effectively. This measurement will be self-reported by the patient.

Each of the 42 participants will either receive one infusion of placebo or one infusion of VY-AADC. The treatment will be administered surgically into the brain. The patients will be monitored carefully for one full year. Researchers will examine any differences in the patients’ motor fluctuations and their ability to complete daily tasks. Additionally, any changes in their quality of life will be evaluated.

It is beyond evident that Parkinson’s disease patients need better therapeutic options. This Phase 2 trial is a positive step forward in the evaluation of a new therapy for those living with this condition.

For more information on the trial, including enrollment information click here.

You can also read a comprehensive overview about the trial here.

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