NCT04167540

Brief Summary

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.

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
11

participants targeted

Target at below P25 for phase_1

Timeline
13mo left

Started Apr 2020

Longer than P75 for phase_1

Geographic Reach
1 country

3 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress84%
Apr 2020Jun 2027

First Submitted

Initial submission to the registry

November 7, 2019

Completed
12 days until next milestone

First Posted

Study publicly available on registry

November 19, 2019

Completed
4 months until next milestone

Study Start

First participant enrolled

April 1, 2020

Completed
3.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 17, 2023

Completed
3.6 years until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2027

Expected
Last Updated

September 19, 2025

Status Verified

September 1, 2025

Enrollment Period

3.5 years

First QC Date

November 7, 2019

Last Update Submit

September 16, 2025

Conditions

Parkinson's Disease

Keywords

PDParkinson's diseaseNeurotrophic factorGrowth factorGlial cell line-derived neurotrophic factorGDNFAAVGene therapy

Outcome Measures

Primary Outcomes (1)

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

    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.

    5 years

Secondary Outcomes (3)

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

    18 months

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

    18 months

  • Brain dopaminergic cell integrity as measured by DaTscan

    18 months

Study Arms (2)

Earlier stage PD

EXPERIMENTAL
Biological: AAV2-GDNF

Later stage PD

EXPERIMENTAL
Biological: AAV2-GDNF

Interventions

AAV2-GDNFBIOLOGICAL

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

Earlier stage PDLater stage PD

Eligibility Criteria

Age35 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

University of California Irvine

Irvine, California, 92697, United States

Location

University of California San Francisco

San Francisco, California, 94103, United States

Location

The Ohio State University Medical Center

Columbus, Ohio, 43210, United States

Location

MeSH Terms

Conditions

Parkinson Disease

Condition Hierarchy (Ancestors)

Parkinsonian DisordersBasal Ganglia DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesMovement DisordersSynucleinopathiesNeurodegenerative Diseases

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Model Details: A total of 12 study participants will be administered the investigational product in this Phase 1b trial. Participants will be enrolled into cohorts, based upon the duration and stage of their PD. Six (6) participants will be dosed in each cohort.
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 7, 2019

First Posted

November 19, 2019

Study Start

April 1, 2020

Primary Completion

October 17, 2023

Study Completion (Estimated)

June 1, 2027

Last Updated

September 19, 2025

Record last verified: 2025-09

Data Sharing

IPD Sharing
Will not share

Locations

US(3)