For Healthcare Professionals

GDNF Gene Therapy for Multiple System Atrophy

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About the study

The objective of this randomized, double-blinded, placebo-controlled Phase 1 investigation is to evaluate the safety and potential clinical effect of AAV2-GDNF delivered to the putamen in subjects with either a possible or probable diagnosis of Multiple System Atrophy.
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Who can take part

You may be eligible to participate in the study if you meet the following criteria:

INCLUSION CRITERIA

Inclusion Criteria:

  1. Male and female 35-75 years of age (inclusive)
  2. Clinical diagnosis of MSA, parkinsonian type with symptoms onset sporadic, progressive and > 30 years of age
  3. Less than 5 years from MSA parkinsonian diagnosis with expected survival more than 3 years
  4. Stable anti-parkinsonian medication regimen
  5. Ability to walk a distance of 25 feet with or without an assistive device

EXCLUSION CRITERIA

Exclusion Criteria:

  1. Presence of idiopathic Parkinson's disease (PD) or any PD-related mutation or other neurological diseases
  2. Presence of dementia, psychosis, substance abuse or poorly controlled depression
  3. Prior brain surgery (i.e., deep brain stimulator implantation) or other brain imaging abnormalities
  4. History of cancer or poorly controlled medical conditions that would increase surgical risk
  5. Received investigational agent within 12 weeks
  6. Inability to tolerate laying flat in an MRI and/or allergy to gadolinium

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

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Study Locations

Enter your ZIP code/Postal code/PIN code to locate study sites near you:

How to Apply


Contact the study center to learn if this study is a good match for you.
Phone iconCall 336-265-2790Email iconEmail Study Center

Study Details


Contition

Multiple System Atrophy

Age

35 - 75

Phase

PHASE1

Participants Needed

9

Est. Completion Date

Dec 31, 2028

Treatment Type

INTERVENTIONAL


Sponsor

Brain Neurotherapy Bio, Inc.

ClinicalTrials.gov NCT Identifier

NCT04680065

Study Number

MSA-101

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