Clinical Trial

Multiple System Atrophy (MSA)

MSA-101 is a randomized, Phase 1 clinical trial evaluating the safety and potential effect of AAV2-GDNF, a glial cell line-derived neurotrophic factor gene therapy, in people with MSA-parkinsonian type (MSA-P).

What is Multiple System Atrophy (MSA)?

MSA is a rare, progressive neurodegenerative disease characterized by a combination of motor and autonomic dysfunctions with limited treatment options and can be misdiagnosed as Parkinson’s disease. 

MSA is categorized into two subtypes: parkinsonian type (MSA-P) and cerebellar type (MSA-C). This study focuses on MSA-P.

Patients with MSA may experience a range of debilitating symptoms, including:

  • Slow movements
  • Stiffness
  • Clumsiness or coordination problem
  • Speech difficulties
  • Orthostatic hypotension (when blood pressure drops 
  • upon standing from a seated or lying down position)
  • Bladder control issues

MSA is a life-threatening disease that severely impacts quality of life; if you think you have MSA, please talk to a medical professional.

About the MSA-101 Trial

MSA-101 is a randomized, Phase 1 clinical trial evaluating the safety and potential effects of AAV2-GDNF in people with MSA. AAV2-GDNF is a one-time gene therapy delivered surgically directly into the brain to provide a continuous expression of the GDNF protein, which may aid to restore or protect the dying nerve cells (neurons) and improve symptoms of MSA. Eligible participants have a 2 out of 3 chance to receive active treatment versus control surgery. Participants randomized to control surgery may be offered the gene therapy product after the main part of the study.

Learn more about the MSA-101 study at (NCT04680065).

AAV2-GDNF is an investigational therapy and has not been approved by the U.S. Food & Drug Administration (FDA) or any other healthcare authority for commercial use. 

Clinical trial activities are outlined below:

Screening Period
Used to determine eligibility

Day of Surgery
Neurosurgical procedure where one-time gene therapy dose is administered or control procedure is performed.

Health Assessments
Occur twice within the first month post-surgery, then about every 3 months for the first year. 

Long-Term Follow-Up Period
After the first year of participation post-surgery, follow up visits will occur every 6 months for 2 additional years.

Are You Eligible?

All clinical trials have specific eligibility criteria that patients must meet in order to participate. These are called
inclusion and exclusion criteria and are related to a person’s health when they enter a clinical trial.

You may be eligible if:

  • Male and female adults 35-75 years of age (inclusive)
  • Diagnosed with MSA with mostly parkinsonian symptoms
    (i.e. MSA-P)
  • Parkinsonian diagnosis less or equal to 5 years at the time of screening
  • Stable anti-parkinsonian medication regimen
  • Ability to walk with or without an assistive device

You are not eligible if:

  • Presence of idiopathic Parkinson’s disease or other neurological diseases
  • Presence of dementia, psychosis, substance abuse, or poorly controlled depression
  • Prior brain surgery (i.e., deep brain stimulator) or other brain imaging abnormalities
  • Participating in another investigational drug study
  • History of cancer or poorly controlled medical conditions that would increase surgical risk
  • Inability to tolerate lying flat in an MRI or allergy to gadolinium

The MSA-101 Study

To find out if you are eligible, please speak with your doctor or visit to learn more about the MSA-101 study (NCT04680065).