Gene therapy reduces movement fluctuations
- Trial ID
- NCT06285643
- Official Title
- A Phase 2, Randomized, Double-blind, Sham Surgery-controlled Study of the Efficacy and Safety of Intraputaminal AAV2-GDNF in the Treatment of Adults With Moderate Stage Parkinson's Disease
- Goal
- Gene therapy reduces movement fluctuations
- Phase
- PHASE2
- Status
- RECRUITING
- Sponsor
- AskBio Inc
- Study Type
- INTERVENTIONAL
- Enrollment
- 127 participants
- Conditions
- Parkinson Disease
- Interventions
- AAV2-GDNF gene therapy, control surgery
Summary For Families
The goal is to see whether boosting production of GDNF in the putamen can protect or restore dopamine neurons and improve motor symptoms and troublesome on/off fluctuations in people with moderate Parkinson's. The approach uses an AAV2 viral vector injected into the putamen so local brain cells make GDNF, the procedure involves stereotactic surgery with a sham surgery control to maintain blinding, and the treatment is meant to complement levodopa rather than replace it since participants must be levodopa responsive. The trial is looking for adults 45 to 75 years old diagnosed 4 to 10 years ago who have motor fluctuations, a stable Parkinson medication regimen, and clear levodopa responsiveness, while excluding those with prior brain surgery, significant vascular or cognitive/psychiatric issues, MRI contraindications, chronic immunosuppression, or other conditions that could mimic Parkinson's.
Locations
- UCI Alpha Clinic (Neurology), Irvine, California, United States
- Loma Linda University (Neurology), Loma Linda, California, United States
- Loma Linda University (Surgical), Loma Linda, California, United States
- University of California San Francisco (Neurology), San Francisco, California, United States
- University of San Francisco (Surgical), San Francisco, California, United States
- CenExel Rocky Mountain Clinical Research (Neurology), Englewood, Colorado, United States
- Georgetown University (Neurology), Washington D.C., District of Columbia, United States
- Parkinson's Disease and Movement Disorders Center of Boca Raton (Neurology), Boca Raton, Florida, United States
- Parkinson's Disease Treatment Center of Southwest Florida (Neurology), Port Charlotte, Florida, United States
- Emory University (Surgical), Atlanta, Georgia, United States
- Rush University Medical Center (Neurology), Chicago, Illinois, United States
- University of Kansas Medical Center (Neurology), Kansas City, Kansas, United States
- Massachusetts General Hospital (Neurology), Boston, Massachusetts, United States
- Massachusetts General Hospital (Surgical), Boston, Massachusetts, United States
- Michigan State University (Neurology), East Lansing, Michigan, United States
- Quest Research Institute (Neurology), Farmington Hills, Michigan, United States
- Cleveland Clinic Lou Ruvo (Neurology), Las Vegas, Nevada, United States
- University of North Carolina (Neurology), Chapel Hill, North Carolina, United States
- Duke Neurosurgery (Surgical), Durham, North Carolina, United States
- Duke University (Neurology), Durham, North Carolina, United States
And 19 more locations.
Frequently Asked Questions
- What is this trial testing?
- This trial is studying AAV2-GDNF gene therapy. The goal is to see whether boosting production of GDNF in the putamen can protect or restore dopamine neurons and improve motor symptoms and troublesome on/off fluctuations in people with moderate Parkinson's. The approach uses an AAV2 viral vector injected into the putamen so local brain cells make GDNF, the procedure involves stereotactic surgery with a sham surgery control to maintain blinding, and the treatment is meant to complement levodopa rather than replace it since participants must be levodopa responsive. The trial is looking for adults 45 to 75 years old diagnosed 4 to 10 years ago who have motor fluctuations, a stable Parkinson medication regimen, and clear levodopa responsiveness, while excluding those with prior brain surgery, significant vascular or cognitive/psychiatric issues, MRI contraindications, chronic immunosuppression, or other conditions that could mimic Parkinson's.
- Who can participate?
- Participants must be between 45 Years and 75 Years.
- Where is this trial located?
- This trial is recruiting at 39 locations.
- Does it cost anything to join?
- No. There is no cost to participate. Study-related care and treatment are provided at no charge.
- How long does the trial last?
- This Phase 2 trial is estimated to last approximately 4 years and 3 months.
- Will I get the real drug or a placebo?
- It depends on the trial design. Some Parkinson's trials are placebo controlled, meaning a portion of participants get an inactive comparison while others get the experimental treatment. Some use crossover designs so everyone eventually receives the active treatment at some point. Observational trials do not use a placebo at all because they are not testing a new treatment. The consent form for any specific trial spells out the design and your odds of being assigned to placebo before you enroll.
- How often will I need to visit the study site?
- Visit frequency varies by trial. Many Parkinson's studies require an in person visit every 4 to 12 weeks during the active treatment phase, with shorter or longer gaps depending on the design. The site coordinator can give you the full visit schedule before you sign anything, so you know what the time commitment looks like.
- Can I leave the trial if I change my mind?
- Yes. You can withdraw from any clinical trial at any time, for any reason, without affecting your standard medical care. Trials are voluntary by law. The team may ask if you are willing to do a brief exit visit so they can collect safety information, but you are not obligated.
- Will travel or parking be reimbursed?
- Many trials reimburse for parking, mileage, and sometimes lodging if the site is far from your home. Reimbursement policies vary by sponsor and site. When you contact the trial team, ask specifically what is covered and how reimbursement is processed.
- Can my spouse or care partner come with me to visits?
- In most cases yes, and it is often encouraged. Care partners can help with notes, questions, and getting home safely after a long visit. Some study assessments do need to happen one on one, but care partners are usually welcome for the rest of the appointment.