GDNF Gene Therapy for Multiple System Atrophy
- Conditions
- Multiple System Atrophy
- Interventions
- Biological: AAV2-GDNF gene therapyProcedure: Sham (Placebo) Surgery
- Registration Number
- NCT04680065
- Lead Sponsor
- Brain Neurotherapy Bio, Inc.
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 9
- 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
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Active Treatment AAV2-GDNF gene therapy - Placebo Surgery Sham (Placebo) Surgery -
- Primary Outcome Measures
Name Time Method The incidence of Treatment-Emergent Adverse Events (TEAE) and Serious Adverse Events (SAE) assessed clinically by physical and neurological examinations 3 years Number of TEAE and SAE's reported post-treatment.
- Secondary Outcome Measures
Name Time Method MSA symptoms/signs as assessed by the Unified Multiple System Atrophy Rating Scale (UMSARS) 12 months Change from baseline in the Unified Multiple System Atrophy Rating Scale (UMSARS) and compared to placebo. UMSARS total scores range from 0-104 points with higher scores indicating greater severity of impairment.
Change in the quality of life as measured by Multiple System Atrophy Quality of Life (MSA-QoL) 12 months Change from baseline and compared to placebo in the Multiple System Atrophy Quality of Life (MSA-QoL) scale. MSA-QoL is a self-reported questionnaire that measures MSA impact in day to day activities. Scale consists of 40 items with a five response option format (0 - no problem to 4 extreme problem) and a "not applicable" response option.
Change in striatal dopamine transporter binding as measured by [123-I] Ioflupane 12 months Percent and absolute change in ratio of specific to non-specific binding of 123I FP-CIT to DaT from baseline and compared to placebo by Single Photon Emission Computed Tomography (SPECT) dopamine transporter (DaT) imaging
Trial Locations
- Locations (7)
University of California Irvine
🇺🇸Irvine, California, United States
Parkinson's Disease and Movement Disorders Center of Boca Raton
🇺🇸Boca Raton, Florida, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
Quest Research Institute
🇺🇸Farmington Hills, Michigan, United States
NYU Langone Health
🇺🇸New York, New York, United States
The Ohio State University Medical Center
🇺🇸Columbus, Ohio, United States
Vanderbilt University Medical Center
🇺🇸Nashville, Tennessee, United States