A Lewy body (stained brown) in a brain cell of the substantia nigra in Parkinson’s disease. Credit: Marvin 101.

Voyager Therapeutics has dosed the first patient in the Phase II RESTORE-1 trial assessing the safety and efficacy of VY-AADC for the treatment of Parkinson’s disease in patients with motor fluctuations that are refractory to medical treatment.

The randomised, double-blind, placebo-controlled trial is expected to include patients who have been suffering from Parkinson’s disease for at least four years.

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It will also enrol patients who are not responding adequately to oral medications and have at least three hours of OFF time during the day.

Enrolled patients will be randomised in 1:1 ratio to receive one-time administration of VY-AADC or placebo surgery.

“Patients with Parkinson’s disease need new therapeutic options, especially as the disease progresses.”

The trial’s primary objective is ON time without dyskinesia, or good ON time, as identified by a self-reported patient diary at 12 months.

Its secondary objectives include diary OFF time, motor function and quality of life measures as determined by the United Parkinson’s Disease Rating Scales (UPDRS-II,-III scores) and Parkinson’s Disease Questionnaire (PDQ-39).

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The study will also determine non-motor symptoms from the Non-Motor Symptom Scale (NMSS).

RESTORE-1 trial principal investigator Mark Richardson said: “Patients with Parkinson’s disease need new therapeutic options, especially as the disease progresses and there is less AADC enzyme in parts of the brain where it is needed to convert levodopa to dopamine.

“Voyager’s VY-AADC is an experimental gene therapy designed to deliver the AADC gene into brain cells where the enzyme can be produced to increase dopamine production.”

Parkinson’s is a chronic, progressive and debilitating neurodegenerative disease that is currently estimated to affect around 1,000,000 people in the US.