Trials / Completed
CompletedNCT03065192
Safety and Efficacy Study of VY-AADC01 for Advanced Parkinson's Disease
An Open-label Safety and Efficacy Study of VY-AADC01 Administered by MRI-Guided Convective Infusion Using a Posterior Trajectory Into the Putamen of Participants With Parkinson's Disease With Fluctuating Responses to Levodopa
- Status
- Completed
- Phase
- Phase 1
- Study type
- Interventional
- Enrollment
- 16 (actual)
- Sponsor
- Neurocrine Biosciences · Industry
- Sex
- All
- Age
- 40 Years – 75 Years
- Healthy volunteers
- Not accepted
Summary
Safety and efficacy of AADC gene transfer in participants with Parkinson's disease.
Detailed description
Parkinson's disease (PD) is a neurodegenerative disorder involving loss of dopamine producing neurons located in the striatum. Levodopa is the primary treatment used to treat Parkinson's disease, which converts to dopamine by the enzyme (protein) Aromatic L-Amino Acid Decarboxylase (AADC). As PD progresses, the amount of AADC levels in the brain decreases, and in turn, reduces the amount of dopamine that is produced with each dose of levodopa. The primary objective of this study is to evaluate the safety of increasing AADC levels, via gene delivery. The investigational drug, termed VY-AADC-01, will be injected directly into the striatum during a neurosurgical procedure that is performed with real-time MRI imaging to monitor delivery. Participants will continue to take their Parkinson medications, including levodopa while participating in this study. The safety and potential clinical responses to VY-AADC-01 will be assessed by repeated clinical evaluations of Parkinson's disease, treatment review phone calls, cognitive tests, laboratory blood tests, patient reported outcomes scales, patient diaries, collection of adverse events, and neuro-imaging. Clinical evaluations will be performed over a 3 year follow-up period.
Conditions
- Idiopathic Parkinson's Disease
- Parkinson's Disease
- Basal Ganglia Disease
- Brain Diseases
- Central Nervous System Diseases
- Movement Disorders
- Nervous System Diseases
- Neurodegenerative Diseases
- Parkinsonian Disorders
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | VY-AADC01 | Single dose, neurosurgically infused, bilaterally into the striatum. |
Timeline
- Start date
- 2017-05-11
- Primary completion
- 2021-08-10
- Completion
- 2021-08-10
- First posted
- 2017-02-27
- Last updated
- 2022-03-21
Locations
4 sites across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT03065192. Inclusion in this directory is not an endorsement.