AAV2-hAADC for Parkinson's Disease (PDCS-01)

Part of paid clinical trials in Columbus, Ohio.

Sponsor
Krzysztof Bankiewicz
Study ID
NCT07267065
Phase
PHASE1
Status
Not Yet Recruiting

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Conditions

  • Early Onset Parkinson Disease
  • Parkinson's Disease (PD)

Eligibility Criteria

Sex
ALL
Age
18 Years - 75 Years
Healthy Volunteers
Not accepted

Interventions

  • AAV2-hAADC — DRUG
    The purpose of this study is to investigate AAV2-hAADC gene therapy (the study drug) for the treatment of moderately-advanced PD with motor fluctuations. AAV2-hAADC gene therapy works by using a modified virus called adeno-associated virus 2 (AAV2). This virus is naturally-occurring and not associated with any disease. The virus' DNA has been changed so that it can deliver the human enzyme called Aromatic L-Amino Acid Decarboxylase (AADC). This enzyme converts levodopa into dopamine. AAV2-hAADC can enter brain cells and transfer the copy of the AADC gene directly into these cells. Once the gene is inside the brain cells, those cells will make AADC. By increasing the levels of AADC, we aim to increase the levels of dopamine and improve the symptoms of PD. AAV2-hAADC will be delivered through a brain surgery to two parts of the brain called the putamen and the caudate. These parts of the brain are involved in PD. The goal of this study is to assess whether infusing AAV2-h

Study Details

This study is looking at whether a gene therapy called AAV2-hAADC is safe and may help people with Parkinson's Disease. AAV2-hAADC is intended to increase the levels of dopamine in your brain. It contains a virus called adeno-associated virus 2 (AAV2) that has been modified to carry the genetic code for an enzyme called Aromatic L-Amino Acid Decarboxylase, or AADC for short. In this study, AAV2-hAADC is delivered to two parts of the brain called the putamen and the caudate. Increasing the amount of AADC gene in these parts of the brain converts more levodopa into dopamine, the chemical that is lacking in PD.

Key Dates

Start date
Feb 1, 2026
Status verified
Nov 2025
Primary completion
Aug 1, 2028
Completion
Dec 1, 2028

Study Design

Enrollment
9 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Dose 1 (Low Dose)
    Total Dose (vg): up to 4.2E12
  • Experimental: Dose 2 (High Dose)
    Total Dose (vg): up to 1.3E13

Primary Outcome Measure

Adverse Events [ Time Frame: Through 3 year Follow-up ]

Locations (1)

FacilityCityStateZIPSite coordinators
The Ohio State UniversityColumbusOhio43210
Andrea Davis, MS
[email protected]

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By research site
The Ohio State University· Columbus, OH

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