- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04680065
GDNF Gene Therapy for Multiple System Atrophy
April 8, 2024 updated by: Brain Neurotherapy Bio, Inc.
Randomized, Double-Blind, Placebo-controlled Safety Study of Glial Cell Line-Derived Neurotrophic Factor Gene Transfer (AAV2-GDNF) in Multiple System Atrophy
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.
Study Overview
Status
Recruiting
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Estimated)
9
Phase
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Christian Urrea, MD
- Phone Number: 336-265-2790
- Email: askfirst@askbio.com
Study Contact Backup
- Name: Akash Pandhare, MD
- Phone Number: 336-265-2790
- Email: askfirst@askbio.com
Study Locations
-
-
California
-
Irvine, California, United States, 92697
- Recruiting
- University of California Irvine
-
Principal Investigator:
- Nicolas Phielipp, MD
-
Contact:
- Sally Alcocer
- Phone Number: 949-824-3990
- Email: GDNFMSA@hs.uci.edu
-
-
Florida
-
Boca Raton, Florida, United States, 33486
- Recruiting
- Parkinson's Disease and Movement Disorders Center of Boca Raton
-
Contact:
- Lauren Salmon
- Email: info@parkinsonscenter.org
-
Principal Investigator:
- Stuart H. Isaacson, M.D.
-
-
Massachusetts
-
Boston, Massachusetts, United States, 02114
- Active, not recruiting
- Massachusetts General Hospital
-
-
Michigan
-
Farmington Hills, Michigan, United States, 48334
- Recruiting
- Quest Research Institute
-
Principal Investigator:
- Aaron Ellenbogen, DO
-
Contact:
- Renee Najor
- Email: Renee.Najor@questri.com
-
-
Ohio
-
Columbus, Ohio, United States, 43210
- Active, not recruiting
- The Ohio State University Medical Center
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
35 years to 75 years (Adult, Older Adult)
Accepts Healthy Volunteers
No
Description
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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Active Treatment
|
Bilateral image-guided infusion of AAV2-GDNF into putamen, single dose
|
Sham Comparator: Placebo Surgery
|
Bilateral partial burr/twist holes without dural penetration
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
The incidence of Treatment-Emergent Adverse Events (TEAE) and Serious Adverse Events (SAE) assessed clinically by physical and neurological examinations
Time Frame: 3 years
|
Number of TEAE and SAE's reported post-treatment.
|
3 years
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
MSA symptoms/signs as assessed by the Unified Multiple System Atrophy Rating Scale (UMSARS)
Time Frame: 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.
|
12 months
|
Change in striatal dopamine transporter binding as measured by [123-I] Ioflupane
Time Frame: 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
|
12 months
|
Change in the quality of life as measured by Multiple System Atrophy Quality of Life (MSA-QoL)
Time Frame: 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.
|
12 months
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Helpful Links
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
October 3, 2023
Primary Completion (Estimated)
December 1, 2025
Study Completion (Estimated)
December 1, 2028
Study Registration Dates
First Submitted
December 15, 2020
First Submitted That Met QC Criteria
December 20, 2020
First Posted (Actual)
December 22, 2020
Study Record Updates
Last Update Posted (Actual)
April 9, 2024
Last Update Submitted That Met QC Criteria
April 8, 2024
Last Verified
April 1, 2024
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Cardiovascular Diseases
- Vascular Diseases
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Basal Ganglia Diseases
- Movement Disorders
- Synucleinopathies
- Neurodegenerative Diseases
- Pathological Conditions, Anatomical
- Autonomic Nervous System Diseases
- Primary Dysautonomias
- Hypotension
- Atrophy
- Multiple System Atrophy
- Shy-Drager Syndrome
Other Study ID Numbers
- MSA-101
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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-
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