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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)
Must not have
Severe dyskinesia
History of cancer or poorly controlled medical conditions that would increase surgical risk
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 5 years
Awards & highlights
No Placebo-Only Group

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.
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.
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.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I experience severe involuntary movements.
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I have a history of cancer or medical conditions that could make surgery risky.
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I have had brain surgery or have abnormalities in brain scans.
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I have been diagnosed with atypical parkinsonism.

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

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
The incidence of Treatment-Emergent Adverse Events (TEAE) assessed clinically by physical and neurological examinations
Secondary study objectives
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)

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

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
69 Previous Clinical Trials
3,347 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
~2 spots leftby Dec 2025
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