GDNF Gene Therapy for Parkinson's Disease

Parkinson's Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04167540
• Other study ID #: GDNF-102
• Status: Active, not recruiting
• Phase: Phase 1
• Start date: April 1, 2020
• Est. completion date: June 2027

Study information

• Verified date: January 2023
• Source: Brain Neurotherapy Bio, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 11
• Est. completion date: June 2027
• Est. primary completion date: December 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 35 Years to 75 Years

Eligibility

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:

1. EITHER: Less than 5 years since clinical diagnosis of PD and mild to moderate UPDRS III OFF score

2. 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

Related Conditions & MeSH terms

• Parkinson Disease
• Parkinson's Disease

Intervention

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

Locations

Country	Name	City	State
United States	The Ohio State University Medical Center	Columbus	Ohio
United States	University of California Irvine	Irvine	California
United States	University of California San Francisco	San Francisco	California

Sponsors (3)

Lead Sponsor	Collaborator
Brain Neurotherapy Bio, Inc.	Asklepios Biopharmaceutical, Inc., California Institute for Regenerative Medicine (CIRM)

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Motor symptoms as assessed by the Movement Disorder Society's Unified Parkinson's Disease Rating Scale (MDS-UPDRS)	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.	18 months
Secondary	Non-motor symptoms of Parkinson's disease as assessed by the Non-Motor Symptom Scale (NMSS)	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.	18 months
Secondary	Brain dopaminergic cell integrity as measured by DaTscan	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.	18 months