CompletedPhase 1

AAV2-GDNF for Advanced Parkinson s Disease

United States25 participantsStarted 2013-03-13

Plain-language summary

Background: \- Glial cell line-derived neurotrophic factor (GDNF) is a chemical that may help protect and strengthen brain cells that produce dopamine. Dopamine is a chemical that affects brain function. People with Parkinson's disease (PD) have problems producing dopamine in the brain. Researchers want to see if gene transfer can help deliver GDNF into the area of the brain that is damaged by PD. The gene transferred in this study, called AAV2-GDNF, may help produce GDNF to protect the damaged brain cells. Objectives: \- To test the safety and effectiveness of AAV2-GDNF gene transfer for advanced PD. Eligibility: \- Individuals at least 18 years of age who have advanced PD that is not well controlled by medications. Design: * Participants will be in the study for about 5 years. There will be 18 outpatient study visits and a 3-day stay in the hospital. There may also be overnight stays for followup visits. * Participants will be screened with a physical exam and medical history. Blood samples will be collected. Tests of PD symptoms and mood and memory will be given. Imaging studies will be used to find the right part of the brain to infuse the gene. The screening visit will take place up to 60 days before surgery. * Participants will have a baseline visit about a month before the surgery. For 1 week before the baseline visit, participants will keep a diary on any motor problems. The visit will involve movement tests given before and after taking a regular dose of levodopa. * Participants will have surgery to infuse AAV2-GDNF into the brain. The surgery will also include a lumbar puncture (spinal tap) to collect cerebrospinal fluid. After surgery, participants will recover in the hospital for at least 2 days. * Participants will have another lumbar puncture 6 and 18 months after surgery. This will be an outpatient visit. * Participants will have regular followup visits after the surgery. These visits will include neurological tests and movement studies. Visits with a neurosurgeon will take place 1, 2, and 4 weeks after surgery. Additional visits will take place every 3 months for the first 3 years, and then at longer intervals for up to 5 years.

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

* INCLUSION CRITERIA: Greater than 18 years of age. Diagnosed with Idiopathic PD. * Bradykinesia and * At least 3 of the following clinical features: resting tremor, cogwheel rigidity, bradykinesia, postural reflex impairment. The above clinical features must not be due to trauma, brain tumor, infection, cerebrovascular disease, other known neurological disease (e.g., hereditary form of PD, multiple system atrophy, progressive supranuclear palsy, striatonigral degeneration, Huntington s disease, Wilson s disease), or to known drugs, chemicals or toxicants. Disease duration of \> 5 years. Hoehn and Yahr Stage III or IV off medication. Disability present despite optimal antiparkinsonian medication therapy. Disability will be operationally defined as a Modified Schwab and England Activities of Daily Living Scale score of 80% or lower (during the off state). Unified PD Rating Scale (UPDRS) (Fahn et al., 1987) total motor score greater than or equal to 30 in the defined off state. Unequivocal responsiveness to levodopa, based on the single-dose levodopa test (as described in the CAPIT and CAPSIT guidelines). A 30% or greater improvement in the UPDRS total motor score will be required to establish unequivocal responsiveness to levodopa. Able to provide proper Informed Consent. Laboratory values at screening visit (unless other visit specified): * Platelets \>100,000/mm3 (transfusion independent) * PT/PTT in normal range and INR less than or equal to 1.3 (on day prior to…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1This is a Phase 1 trial testing four different dose levels of a gene therapy called AAV2-GDNF — since Phase 1 studies are primarily designed to check safety rather than prove the treatment works, what does that mean for what we actually know about whether it helped people with Parkinson's?
2The trial is now completed — has the safety and tolerability data from this study been published, and are there any follow-up or Phase 2 trials building on these results that might be worth looking into?
3Since this involves delivering a gene therapy directly to the brain, what are the specific surgical and long-term risks I should understand before considering any similar approach in the future?
4Given that this was tested in people with advanced Parkinson's disease, how does my current stage of the disease compare to the patients who were enrolled, and does that affect whether this line of research is even relevant to my situation?
5Are there currently available standard treatments or other ongoing trials that my care team would recommend I consider alongside or instead of pursuing gene therapy options like this one?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Assess the safety and tolerability of 4 different dose levels of AAV2-GDNF

Timeframe: Day 7, Day 14, 1 Month, 2 Month, 3 Month, 6 Month, 9 Month, 12 Month, 15 Month, 18 Month, 24 Month, 30 Month, 36 Month, 48 Month, 60 Month

Trial details

NCT IDNCT01621581

SponsorNational Institute of Neurological Disorders and Stroke (NINDS)

Sponsor typeNIH

Study typeINTERVENTIONAL

Primary completion2022-02-04

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