Clinical trial NCT04167540: GDNF Gene Therapy for Parkinson's Disease
Official title: Open-Label Safety Study of Glial Cell Line-Derived Neurotrophic Factor Gene Transfer (AAV2- GDNF) in Parkinson's Disease
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE1
Conditions: Parkinson's Disease
Keywords: PD
Parkinson's disease
Neurotrophic factor
Growth factor
Glial cell line-derived neurotrophic factor
GDNF
AAV
Gene therapy
Lead sponsor: Brain Neurotherapy Bio, Inc.
Sponsor class: INDUSTRY
Collaborators: name: AskBio Inc
name: California Institute for Regenerative Medicine (CIRM)
First posted: 2019-11-19
Enrollment: 11
Sex: ALL
Minimum age (years): 35
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Intervention model description: 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.
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: The incidence of Treatment-Emergent Adverse Events (TEAE) assessed clinically by physical and neurological examinations
timeFrame: 5 years
Secondary outcomes:
measure: Motor symptoms as assessed by the Movement Disorder Society's Unified Parkinson's Disease Rating Scale (MDS-UPDRS)
timeFrame: 18 months
measure: Non-motor symptoms of Parkinson's disease as assessed by the Non-Motor Symptom Scale (NMSS)
timeFrame: 18 months
measure: Brain dopaminergic cell integrity as measured by DaTscan
timeFrame: 18 months
Other outcomes:
Locations:
city: Irvine
country: United States
facility: University of California Irvine
state: California
city: San Francisco
country: United States
facility: University of California San Francisco
state: California
city: Columbus
country: United States
facility: The Ohio State University Medical Center
state: Ohio
Study officials:
References: