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Active, Not Recruiting

NCT Number: NCT04167540

GDNF Gene Therapy for Parkinson's Disease

The objective of this Phase 1b investigation is to evaluate the safety and potential clinical effect of AAV2-GDNF delivered to the putamen in subjects with either a recent or a long-standing diagnosis of PD.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Parkinson's Disease Basal Ganglia Diseases Brain Diseases Central Nervous System Diseases Movement Disorders Nervous System Diseases Neurodegenerative Diseases Parkinson Disease Parkinsonian Disorders Synucleinopathies

Age range

35 year–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

University of California Irvine, Irvine, California, United States

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Key Inclusion Criteria:

  • Male and female adults 35-75 years of age (inclusive)
  • Diagnosed with Parkinson's disease
  • Modified Hoehn and Yahr stage I-III OFF medication
  • Time since receiving a clinical diagnosis of PD and disease severity consistent with one of the following:
  • 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
  • Responsiveness to levodopa

Key Exclusion Criteria:

  • Atypical parkinsonism
  • Severe dyskinesia
  • Presence of dementia, psychosis, substance abuse or qualify as "severe depression"
  • Prior brain surgery (i.e. deep brain stimulator or DBS implantation) or other brain imaging abnormalities
  • Receiving an investigational drug
  • History of cancer or poorly controlled medical conditions that would increase surgical risk
  • Inability to tolerate laying flat in an MRI or allergy to gadolinium

Treatment and study plan

AAV2-GDNF

Biological

Bilateral image-guided infusion of AAV2-GDNF into putamen, single dose

Primary outcomes

  1. The incidence of Treatment-Emergent Adverse Events (TEAE) assessed clinically by physical and neurological examinations

    Time frame: 5 years

    Evaluation of the safety and tolerability through the assessment of incidence of TEAE, identified by MedDRA preferred term and grouped by MedDRA System Organ Class, as well as clinically meaningful changes in clinical exams or laboratory assays.

Secondary outcomes

  1. Motor symptoms as assessed by the Movement Disorder Society's Unified Parkinson's Disease Rating Scale (MDS-UPDRS)

    Time frame: 18 months

    Change from baseline in the MDS-UPDRS. The MDS-UPDRS contains 4 subscales: Part I, non-motor aspects of experiences of daily living (13 items); Part II, motor aspects of experiences of daily living (13 items); Part III, motor examination (33 scores based on 18 items); Part IV, motor complications (6 items). The rating for each item, or sub-item, is from 0 (normal) to 4 (severe). The total score for each Part is obtained from the sum of the corresponding item scores.

  2. Non-motor symptoms of Parkinson's disease as assessed by the Non-Motor Symptom Scale (NMSS)

    Time frame: 18 months

    Change from baseline in the NMSS. The NMSS evaluates 9 domains of non-motor of severity and frequency of PD symptoms associated with cardiovascular health, sleep and fatigue, mood and cognition, perceptual problems and hallucinations, attention and memory, gastrointestinal tract, urinary, sexual function, and a miscellaneous domain for other common non-motor conditions. Severity x frequency scores range 0-108, with 0 being less severe and less frequent.

  3. Brain dopaminergic cell integrity as measured by DaTscan

    Time frame: 18 months

    Percentage and absolute changes in Ioflupane retention as a marker for dopamine transporter protein expressed by dopamine producing cells within the brain. Measured by quantitative analysis of DaTscan SPECT imaging.

Sponsors and collaborators

Lead sponsor

Brain Neurotherapy Bio, Inc.

Industry

Collaborators

  • AskBio Inc
  • California Institute for Regenerative Medicine (CIRM)

Registry information

Official study title

Open-Label Safety Study of Glial Cell Line-Derived Neurotrophic Factor Gene Transfer (AAV2- GDNF) in Parkinson's Disease

Important dates

Study start
2020
Primary completion
2023
Study completion
2027
First posted
Nov 19, 2019
Registry last updated
Sep 19, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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