Clinical trial NCT04680065: GDNF Gene Therapy for Multiple System Atrophy
Official title: Randomized, Double-Blind, Placebo-controlled Safety Study of Glial Cell Line-Derived Neurotrophic Factor Gene Transfer (AAV2-GDNF) in Multiple System Atrophy
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
Conditions: Multiple System Atrophy
Keywords: MSA
Multiple System Atrophy
Neurotrophic factor
Growth factor
Glial cell line-derived neurotrophic factor
GDNF
AAV
Gene therapy
Lead sponsor: Brain Neurotherapy Bio, Inc.
Sponsor class: INDUSTRY
First posted: 2020-12-22
Enrollment: 9
Sex: ALL
Minimum age (years): 35
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Up to 9 study participants meeting eligibility criteria will be randomized in a 2:1 fashion to receive either the investigational medicinal product or sham surgery in this Phase 1 trial.
Primary purpose: TREATMENT

Brief summary: The objective of this randomized, double-blinded, placebo-controlled Phase 1 investigation is to evaluate the safety and potential clinical effect of AAV2-GDNF delivered to the putamen in subjects with either a possible or probable diagnosis of Multiple System Atrophy.


Eligibility criteria: Inclusion Criteria:

* Male and female 35-75 years of age (inclusive)
* Clinical diagnosis of MSA, parkinsonian type with symptoms onset sporadic, progressive and \> 30 years of age
* Less than 5 years from MSA parkinsonian diagnosis with expected survival more than 3 years
* Stable anti-parkinsonian medication regimen
* Ability to walk a distance of 25 feet with or without an assistive device

Exclusion Criteria:

* Presence of idiopathic Parkinson's disease (PD) or any PD-related mutation or other neurological diseases
* Presence of dementia, psychosis, substance abuse or poorly controlled depression
* Prior brain surgery (i.e., deep brain stimulator implantation) or other brain imaging abnormalities
* History of cancer or poorly controlled medical conditions that would increase surgical risk
* Received investigational agent within 12 weeks
* Inability to tolerate laying flat in an MRI and/or allergy to gadolinium

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Primary outcomes:
measure: The incidence of Treatment-Emergent Adverse Events (TEAE) and Serious Adverse Events (SAE) assessed clinically by physical and neurological examinations
timeFrame: 3 years
Secondary outcomes:
measure: MSA symptoms/signs as assessed by the Unified Multiple System Atrophy Rating Scale (UMSARS)
timeFrame: 12 months
measure: Change in striatal dopamine transporter binding as measured by [123-I] Ioflupane
timeFrame: 12 months
measure: Change in the quality of life as measured by Multiple System Atrophy Quality of Life (MSA-QoL)
timeFrame: 12 months
Other outcomes:
Locations:
city: Irvine
country: United States
facility: University of California Irvine
state: California
city: Boca Raton
country: United States
facility: Parkinson's Disease and Movement Disorders Center of Boca Raton
state: Florida
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Farmington Hills
country: United States
facility: Quest Research Institute
state: Michigan
city: New York
country: United States
facility: NYU Langone Health
state: New York
city: Columbus
country: United States
facility: The Ohio State University Medical Center
state: Ohio
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
Study officials:
References: