--- title: Gene therapy reduces movement fluctuations nct_id: NCT06285643 phase: PHASE2 status: RECRUITING sponsor: AskBio Inc study_type: INTERVENTIONAL canonical_url: "https://parkinsonspathways.com/trial/NCT06285643" clinicaltrials_gov: "https://clinicaltrials.gov/study/NCT06285643" last_fetched: "2026-05-10T14:07:19.517Z" source: "Parkinson's Pathways (curated)" --- # Gene therapy reduces movement fluctuations **Goal (in five words):** Gene therapy reduces movement fluctuations **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 **Trial ID:** [NCT06285643](https://clinicaltrials.gov/study/NCT06285643) ## Key Facts - **Phase:** PHASE2 - **Status:** RECRUITING - **Study Type:** INTERVENTIONAL - **Sponsor:** AskBio Inc - **Target Enrollment:** 127 participants - **Start Date:** 2024-06-11 - **Completion Date:** 2028-08-31 - **Conditions:** Parkinson Disease - **Interventions:** AAV2-GDNF gene therapy, control surgery - **Intervention Types:** DRUG, PROCEDURE ## 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. ## Eligibility - **Minimum age:** 45 Years - **Maximum age:** 75 Years - **Sex:** ALL ### Full Criteria ``` 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: * Rigidity * Rest tremor * Postural instability 2. Presence of motor fluctuations as measured by the PD Motor Diary 3. Stable anti-parkinsonian medication regimen 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 disorder * History of malignancy other than treated cutaneous squamous or basal cell carcinomas * Presence of clinically relevant conditions that could compromise surgical suitability and/or subject safety * Contraindication to magnetic resonance imaging and/or use gadolinium-based contrast agents * Prior history of brain surgery including, but no limited: DBS, pallidotomy focused ultrasound thalamotomy, or other experimental neurosurgical procedure * Chronic immunosuppressive therapy ``` ## Locations (39) - UCI Alpha Clinic (Neurology), Irvine, California, United States _(33.6695, -117.8231)_ - (CONTACT) — 949-824-3990 — alphaclinic@uci.edu - Loma Linda University (Neurology), Loma Linda, California, United States _(34.0483, -117.2612)_ - Julie Lacanlale — (CONTACT) — jlacanlale@llu.edu - Loma Linda University (Surgical), Loma Linda, California, United States _(34.0483, -117.2612)_ - University of California San Francisco (Neurology), San Francisco, California, United States _(37.7749, -122.4194)_ - Aaron Daley — (CONTACT) — Aaron.Daley@ucsf.edu - University of San Francisco (Surgical), San Francisco, California, United States _(37.7749, -122.4194)_ - CenExel Rocky Mountain Clinical Research (Neurology), Englewood, Colorado, United States _(39.6478, -104.9878)_ - (CONTACT) — 720-776-0092 - Georgetown University (Neurology), Washington D.C., District of Columbia, United States _(38.8951, -77.0364)_ - Luke Lovelace — (CONTACT) — LL928@georgetown.edu - Parkinson's Disease and Movement Disorders Center of Boca Raton (Neurology), Boca Raton, Florida, United States _(26.3587, -80.0831)_ - Lauren Salmon — (CONTACT) — 561-392-1818 — lsalmon@parkinsonscenter.org - Parkinson's Disease Treatment Center of Southwest Florida (Neurology), Port Charlotte, Florida, United States _(26.9762, -82.0906)_ - Emory University (Surgical), Atlanta, Georgia, United States _(33.7490, -84.3880)_ - Rush University Medical Center (Neurology), Chicago, Illinois, United States _(41.8500, -87.6500)_ - Neepa J. Patel, MD — (CONTACT) — 312-563-2900 - University of Kansas Medical Center (Neurology), Kansas City, Kansas, United States _(39.1142, -94.6275)_ - Sarina Fay — (CONTACT) — sfay2@kumc.edu - Massachusetts General Hospital (Neurology), Boston, Massachusetts, United States _(42.3584, -71.0598)_ - Nisha Kabir — (CONTACT) — nkabir@mgh.harvard.edu - Massachusetts General Hospital (Surgical), Boston, Massachusetts, United States _(42.3584, -71.0598)_ - Michigan State University (Neurology), East Lansing, Michigan, United States _(42.7370, -84.4839)_ - Leigh Whittet — (CONTACT) — 517-884-2275 — kmetleig@msu.edu, - Quest Research Institute (Neurology), Farmington Hills, Michigan, United States _(42.4853, -83.3772)_ - Denise Morgott — (CONTACT) — denise.morgott@questri.com - Cleveland Clinic Lou Ruvo (Neurology), Las Vegas, Nevada, United States _(36.1750, -115.1372)_ - Liliana Dumitrescu — (CONTACT) — 702-483-6016 — DUMITRL@ccf.org - University of North Carolina (Neurology), Chapel Hill, North Carolina, United States _(35.9132, -79.0558)_ - Hans Nettesheim — (CONTACT) — Hans_Nettesheim@med.unc.edu - Duke Neurosurgery (Surgical), Durham, North Carolina, United States _(35.9940, -78.8986)_ - Duke University (Neurology), Durham, North Carolina, United States _(35.9940, -78.8986)_ - Karen White-Tong — (CONTACT) — 919-668-2905 — karen.white@duke.edu - Ohio State University (Surgical), Columbus, Ohio, United States _(39.9612, -82.9988)_ - University of Pennsylvania (Neurology), Philadelphia, Pennsylvania, United States _(39.9524, -75.1636)_ - Neda Almassi — (CONTACT) — 215-829-7289 — neda.almassi@pennmedicine.upenn.edu - Vanderbilt University (Neurology), Nashville, Tennessee, United States _(36.1659, -86.7844)_ - Haley Kling — (CONTACT) — 615-208-3974 — haley.kling@vumc.org - Houston Methodist (Neurology), Houston, Texas, United States _(29.7633, -95.3633)_ - Daniel Arnaud Dominguez — (CONTACT) — darnauddominguez@houstonmethodist.org - Houston Methodist (Surgical), Houston, Texas, United States _(29.7633, -95.3633)_ - EvergreenHealth Research Department (Neurology), Kirkland, Washington, United States _(47.6815, -122.2087)_ - Gowri Rajendran — (CONTACT) — grajendran@evergreenhealthcare.org - University of Wisconsin (Neurology), Madison, Wisconsin, United States _(43.0731, -89.4012)_ - Amy Nagel — (CONTACT) — anagel@neurology.wisc.edu - Charité - Universitätsmedizin Berlin (Surgical), Berlin, Germany _(52.5244, 13.4105)_ - Philipps-Universität Marburg (Neurology), Marburg, Germany _(50.8090, 8.7707)_ - Stefanie Spriewald — (CONTACT) — Stefanie.Spriewald@uk-gm.de - Universitätsklinikum Tübingen (Neurology), Tübingen, Germany _(48.5227, 9.0522)_ - Gabriele König — (CONTACT) — gabriele.koenig@med.uni-tuebingen.de - Universitätsklinikum Tübingen (Surgical), Tübingen, Germany _(48.5227, 9.0522)_ - Universitätsklinikum Würzburg (Neurology), Würzburg, Germany _(49.7939, 9.9512)_ - Christine Leypold — (CONTACT) — leypold_c@ukw.de - Copernicus Podmiot Leczniczy, Szpital św. Wojciecha (Neurology), Gdansk, Poland _(54.3523, 18.6491)_ - Anna Roszmann — (CONTACT) — aniar@gumed.edu.pl - Specjalistyczna Praktyka Lekarska Prof. Grzegorz Opala (Neurology), Katowice, Poland _(50.2597, 19.0217)_ - Magdalena Łyniewska — (CONTACT) — opala.praktyka@gmail.com - Mazowiecki Szpital Bródnowski (Neurology), Warsaw, Poland _(52.2298, 21.0118)_ - Joanna Kralka — (CONTACT) — kralkajoanna@gmail.com - Mazowiecki Szpital Bródnowski (Surgical), Warsaw, Poland _(52.2298, 21.0118)_ - Samodzielny Publiczny Szpital Kliniczny im. Prof. W. Orłowskiego CMKP (Neurology), Warsaw, Poland _(52.2298, 21.0118)_ - Marta Piaścik-Gromada — (CONTACT) — kl.neurologii@szpital-orlowskiego.pl - North Bristol NHS Trust - Southmead Hospital (Neurology), Bristol, United Kingdom _(51.4552, -2.5966)_ - Dominika Kruszynska — (CONTACT) — Dominika.Kruszynska@nbt.nhs.uk - University Hospital of Wales (Surgical), Cardiff, United Kingdom _(51.4800, -3.1800)_ ## Central Contacts - Nisha Chhabria, MD — (CONTACT) — 919-388-1040 — askfirst@askbio.com - Amber Van Laar, MD — (CONTACT) — 919-388-1040 — askfirst@askbio.com --- *Canonical: https://parkinsonspathways.com/trial/NCT06285643* *HTML version: https://parkinsonspathways.com/trial/NCT06285643* *Source data: https://clinicaltrials.gov/study/NCT06285643*