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Gene Therapy

GDNF Gene Therapy for Parkinson's Disease

Phase 1
Waitlist Available
Research Sponsored by Brain Neurotherapy Bio, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
OR: At least 4 years since clinical diagnosis of PD and moderate to severe UPDRS III OFF score
Male and female adults 35-75 years of age (inclusive)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 5 years
Awards & highlights

Study Summary

This trial is testing whether a gene therapy called AAV2-GDNF is safe and effective in treating people with Parkinson's disease.

Who is the study for?
This trial is for adults aged 35-75 with Parkinson's Disease (PD), diagnosed within the last 5 years with mild to moderate symptoms or at least 4 years ago with moderate to severe symptoms. Participants must respond to levodopa treatment, not have atypical parkinsonism, severe dyskinesia, dementia, psychosis, substance abuse issues or severe depression. They can't have had prior brain surgery or be on investigational drugs.Check my eligibility
What is being tested?
The study tests AAV2-GDNF gene therapy delivered to a part of the brain called putamen in people with PD. It aims to assess safety and potential benefits of this treatment for those newly diagnosed or living long-term with PD.See study design
What are the potential side effects?
As it's an early-stage trial focusing on safety, specific side effects are being investigated but may include typical gene therapy-related reactions such as immune response against the vector, inflammation in the brain area where it's injected and general discomfort.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I was diagnosed with Parkinson's disease over 4 years ago and have moderate to severe symptoms.
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I am between 35 and 75 years old.
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I have been diagnosed with Parkinson's disease.
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My Parkinson's disease is in the early to mid-stages without medication.
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I was diagnosed with Parkinson's disease less than 5 years ago with mild to moderate symptoms or at least 4 years ago with moderate to severe symptoms.
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My condition improves with levodopa.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and 5 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
The incidence of Treatment-Emergent Adverse Events (TEAE) assessed clinically by physical and neurological examinations
Secondary outcome measures
Brain dopaminergic cell integrity as measured by DaTscan
Motor symptoms as assessed by the Movement Disorder Society's Unified Parkinson's Disease Rating Scale (MDS-UPDRS)
Non-motor symptoms of Parkinson's disease as assessed by the Non-Motor Symptom Scale (NMSS)

Trial Design

2Treatment groups
Experimental Treatment
Group I: Later stage PDExperimental Treatment1 Intervention
Group II: Earlier stage PDExperimental Treatment1 Intervention

Find a Location

Who is running the clinical trial?

California Institute for Regenerative Medicine (CIRM)OTHER
63 Previous Clinical Trials
3,137 Total Patients Enrolled
Brain Neurotherapy Bio, Inc.Lead Sponsor
1 Previous Clinical Trials
9 Total Patients Enrolled
Asklepios Biopharmaceutical, Inc.Industry Sponsor
7 Previous Clinical Trials
422 Total Patients Enrolled

Media Library

AAV2-GDNF (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04167540 — Phase 1
Parkinson's Disease Research Study Groups: Later stage PD, Earlier stage PD
Parkinson's Disease Clinical Trial 2023: AAV2-GDNF Highlights & Side Effects. Trial Name: NCT04167540 — Phase 1
AAV2-GDNF (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04167540 — Phase 1

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Has AAV2-GDNF received therapeutic license from the FDA?

"AAV2-GDNF is still in early development, as reflected by its Phase 1 status. This means that there is limited safety and efficacy data available."

Answered by AI

Are there any stipulations for who can join this research project?

"For this parkinson disease study, we need 12 men and women aged 35-75 who meet the following profile: Modified Hoehn and Yahr stage I-III OFF medication, Male and female adults 35-75 years of age (inclusive), EITHER: Less than 5 years since clinical diagnosis of PD and mild to moderate UPDRS III OFF score, OR: At least 4 years since clinical diagnosis of PD and moderate to severe UPDRS III OFF score."

Answered by AI

Are patients who are over 30 years old eligible for this clinical trial?

"According to the eligibility requirements published for this clinical trial, applicants must be aged 35 to 75. Out of the 534 similar medical studies, this is one of 29 that is for patients under 18 and one of 505 for patients over 65."

Answered by AI

Who else is applying?

What site did they apply to?
University of California San Francisco
What portion of applicants met pre-screening criteria?
Met criteria
How many prior treatments have patients received?
2
Recent research and studies
clinicaltrials.govStudy
GDNF Gene Therapy for Parkinson's Disease
2020. "GDNF Gene Therapy for Parkinson's Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04167540.
~2 spots leftby Apr 2025
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