Randomized Double-Blind Placebo-controlled Safety Study of Glial Cell Line-Derived Neurotrophic Factor Gene Transfer (AAV2-GDNF) in Multiple System Atrophy

Are you eligible to participate in this study?

You may be eligible for this study if you meet the following criteria:

  • Conditions:
    Multiple System Atrophy
  • Age: Between 35 Year(s) - 75 Year(s)
  • Gender: Male or Female
  • Other Inclusion Criteria:
    1. Clinical diagnosis of MSA with clinical features of primarily Parkinsonism (MSA-P).
    2. Parkinsonian diagnosis ≤ 5 years at the time of screening.
    3. Ability to walk a distance of 25 feet (ft) with or without an assistive device.

You may not be eligible for this study if the following are true:

    1. Movement disorder due to known cerebrovascular disease, brain tumor, trauma, exposure to parkinsonian-linked toxicants or other neurological diseases.
    2. Presence of any Parkinson's disease (PD)-related mutation.
    3. Presence of dementia.
    4. Presence or history of psychosis.
    5. Presence of substance (i.e., drug, alcohol) abuse.
    6. History of stroke or poorly controlled cardiovascular disease.
    7. Uncontrolled hypertension or diabetes.
    8. History of malignancy (cerebral or systemic) other than treated cutaneous squamous or basal cell carcinomas.
    9. Clinically active infection.
    10. Prior brain surgery (i.e., deep brain stimulator implantation) or neuromodulatory intervention.
    11. Contraindication to MRI and/or use of gadolinium
    12. Pregnancy or lactation.
    13. Male or female with reproductive capacity that is unwilling to use barrier contraception for 3 months post-dosing.
    14. Chronic immunosuppressive therapy (e.g., chronic steroids, tumor necrosis factor (TNF) antagonists, chemotherapy).
    15. Unwilling to defer any vaccination within 1 month before or after treatment.
    16. Received investigational agent within 12 weeks prior to screening.



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