Our expert researchers collaborate with teams throughout the institution, country, and world to study a range of neurodegenerative and genetic conditions such as Parkinson’s Disease, Alzheimer’s, and Huntington’s Disease. These clinical trials have been built in part on the work of researchers within the Gene Therapy Institute to begin testing these therapies in patients suffering from these conditions. The work of our physicians, research support staff, and ancillary groups brings this cutting edge technology once step closer to providing patients with improved treatment options.
Clinical Trials
The Clinical Research Division of the Department of Neurological Surgery at Ohio State is engaged in a number of industry sponsored and investigator initiated clinical trials within gene therapy.
Learn More
If you are interested in any of our active trials please visit the ClinicalTrials.gov website linked below to learn more about study design and eligibility criteria. You may also reach out to our research staff to enquire about specific studies at osugenetherapyresearch@osumc.edu.
List of Trials
A Phase I Study to Assess the Safety, Tolerability and Preliminary Efficacy of AAV2-BDNF [Adeno-Associated Virus (AAV)-based, Vector-Mediated Delivery of Human Brain Derived Neurotrophic Factor] in Subjects with Early Alzheimer’s Disease and Mild Cognitive Impairment – actively recruiting
A Single-Stage, Adaptive, Open-Label, Dose Escalation Safety Study of Adeno-Associated Virus Encoding Human Aromatic L-Amino Acid Decarboxylase (AAV2-hAADC) Administered by MR-Guided Convective Infusion into the Midbrain in Pediatric Patients with AADC Deficiency (AAV2-hAADC) – actively recruiting
A Phase 1/2 Open-Label, Ascending Dose, Multicenter Study to Evaluate the Safety and Preliminary Efficacy of AVB-101 Administered by Bilateral Intrathalamic Infusion in Subjects With Frontotemporal Dementia With Progranulin Mutations (FTD-GRN) – actively recruiting
A Phase I/II, Randomized, Double-blind, Sham Control Multiple Ascending Doses of Striatally-Administered rAAV5-miHTT Total Huntingtin Gene (HTT) Lowering Therapy (AMT-130) in Early Manifest Huntington Disease – actively recruiting
Randomized, double-blind, placebo-controlled safety study of glial cell line-derived neurotrophic factor gene transfer (AAV2-GDNF) in multiple system atrophy (MSA-101 AAV2-GDNF) - surgical site only
Open-Label Safety Study of Glial Cell Line-Derived Neurotrophic Factor Gene Transfer (AAV2-GDNF) in Parkinson's Disease – enrollment complete
A Randomized, Placebo Surgery Controlled, Double-blinded, Multi-center, Phase 2 Clinical Trial, Evaluating the Efficacy and Safety of VY-AADC02 in Advanced Parkinson's Disease with Motor Fluctuations; PD-1105 – enrollment complete