NCT06285643 · AskBio Inc
A Study of AAV2-GDNF in Adults With Moderate Parkinson's Disease (REGENERATE-PD)
(REGENERATE-PD)
What this study is about
The objective of this randomly assigned, surgically controlled, double-blinded, Phase 2 study is to evaluate the safety and effectiveness of AAV2-GDNF delivered to the putamen in subjects with moderate Parkinson's Disease.
View original scientific description
The objective of this randomized, surgically controlled, double-blinded, Phase 2 study is to evaluate the safety and efficacy of AAV2-GDNF delivered to the putamen in subjects with moderate Parkinson's Disease.
Interventions
DRUG
AAV2-GDNF gene therapy
Bilateral image-guided infusion of AAV2-GDNF into putamen, single dose
PROCEDURE
control surgery
Bilateral partial burr/twist holes without dural penetration
Primary outcome measures
Change from baseline to Month 18 on PD Motor Diary
Time frame: 18 months
Normalized Good ON time per Hauser Diary
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Age 1. Male and female adults 45-75 years of age inclusive, at the time of signing of informed consent Type of Subject and Disease Characteristics 2. Diagnosed with Parkinson's Disease in the past 4-10 years (inclusive) as defined by the following: 1. Presence of bradykinesia PLUS any of the following:
- Rest Tremor
- Postural instability 2. Presence of motor fluctuations as measured by the PD Motor Diary 3. Stable anti-parkinsonian medication regiment for \>/= 4 weeks prior to screening 4. Must demonstrate responsiveness to levodopa therapy
Exclusion criteria
- Known history or current evidence of medical, genetic, or neurological conditions that may provide an alternative to idiopathic PD diagnosis
- Presence or history of significant vascular and/or cardiovascular disease
- Presence of significant cognitive impairment, poorly controlled depression/anxiety
- Presence or history of psychosis or impulse control disorde
Where
- Phoenix, Arizona
- Irvine, California
- Loma Linda, California
- San Francisco, California
- Englewood, Colorado
- Washington D.C., District of Columbia
- Boca Raton, Florida
- Gainesville, Florida
- Miami, Florida
- Orlando, Florida
- Port Charlotte, Florida
- Atlanta, Georgia
And 17 more locations — see the full list below.
Collaborators
Bayer
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jun 29, 2026 · Source of record for eligibility and locations