Gene therapy using GDNF for treating Multiple System Atrophy
Randomized, Double-Blind, Placebo-controlled Safety Study of Glial Cell Line-Derived Neurotrophic Factor Gene Transfer (AAV2-GDNF) in Multiple System Atrophy
This study is testing a new gene therapy to see if it can help people with Multiple System Atrophy feel better and slow down their symptoms.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 9 (estimated) |
| Ages | 35 Years to 75 Years |
| Sex | All |
| Sponsor | Brain Neurotherapy Bio, Inc. Industry-sponsored |
| Locations | 7 sites (Irvine, California and 6 other locations) |
| Trial ID | NCT04680065 on ClinicalTrials.gov |
What this trial studies
This Phase 1 clinical trial aims to evaluate the safety and potential clinical effects of AAV2-GDNF gene therapy delivered to the putamen in patients diagnosed with Multiple System Atrophy (MSA). The study is randomized, double-blinded, and placebo-controlled, meaning participants will receive either the gene therapy or a sham surgery without knowing which one they received. The trial focuses on individuals aged 35-75 with a recent diagnosis of MSA and stable medication regimens. The goal is to assess how this innovative treatment may impact the progression of MSA symptoms.
Who should consider this trial
Good fit: Ideal candidates are males and females aged 35-75 with a clinical diagnosis of MSA, parkinsonian type, who have had symptoms for less than five years.
Not a fit: Patients with idiopathic Parkinson's disease, other neurological diseases, or significant comorbidities may not benefit from this study.
Why it matters
Potential benefit: If successful, this gene therapy could provide a new treatment option that may slow the progression of Multiple System Atrophy.
How similar studies have performed: While gene therapy approaches are being explored for various neurological conditions, this specific application for MSA is novel and has not been extensively tested in prior studies.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Male and female 35-75 years of age (inclusive) * Clinical diagnosis of MSA, parkinsonian type with symptoms onset sporadic, progressive and \> 30 years of age * Less than 5 years from MSA parkinsonian diagnosis with expected survival more than 3 years * Stable anti-parkinsonian medication regimen * Ability to walk a distance of 25 feet with or without an assistive device Exclusion Criteria: * Presence of idiopathic Parkinson's disease (PD) or any PD-related mutation or other neurological diseases * Presence of dementia, psychosis, substance abuse or poorly controlled depression * Prior brain surgery (i.e., deep brain stimulator implantation) or other brain imaging abnormalities * History of cancer or poorly controlled medical conditions that would increase surgical risk * Received investigational agent within 12 weeks * Inability to tolerate laying flat in an MRI and/or allergy to gadolinium NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Where this trial is running
Irvine, California and 6 other locations
- University of California Irvine — Irvine, California, United States (Recruiting)
- Parkinson's Disease and Movement Disorders Center of Boca Raton — Boca Raton, Florida, United States (Recruiting)
- Massachusetts General Hospital — Boston, Massachusetts, United States (Active_not_recruiting)
- Quest Research Institute — Farmington Hills, Michigan, United States (Recruiting)
- NYU Langone Health — New York, New York, United States (Recruiting)
- The Ohio State University Medical Center — Columbus, Ohio, United States (Active_not_recruiting)
- Vanderbilt University Medical Center — Nashville, Tennessee, United States (Recruiting)
Study contacts
- Study coordinator: Akash Pandhare, MD
- Email: askfirst@askbio.com
- Phone: 919-388-1040
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.