Multiple System AtrophyPhase 1NCT04680065
GDNF Gene Therapy for Multiple System Atrophy
A Phase 1 study testing AAV2-GDNF gene therapy in people with Multiple System Atrophy, run by Brain Neurotherapy Bio, Inc. at 7 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 35 to 75 years
- Conditions the study is for
- Multiple System Atrophy
- What is being tested?
- AAV2-GDNF gene therapy
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 9 participants
- Where
- 7 sitesUnited States
- Study dates
- 3 Oct 2023to Aug 2028
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 35 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
United States7 sites
Parkinson's Disease and Movement Disorders Center of Boca Raton
Boca Raton, Florida, 33486
Massachusetts General Hospital
Boston, Massachusetts, 02114
The Ohio State University Medical Center
Columbus, Ohio, 43210
Quest Research Institute
Farmington Hills, Michigan, 48334
University of California Irvine
Irvine, California, 92697
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
NYU Langone Health
New York, New York, 10016
About this study
Official title: 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.
- Conditions
- Multiple System Atrophy
- Being tested
- AAV2-GDNF gene therapy, Sham (Placebo) Surgery
- Keywords
- MSA, Multiple System Atrophy, Neurotrophic factor, Growth factor, Glial cell line-derived neurotrophic factor, GDNF, AAV, Gene therapy
- Medical subject headings
- Multiple System Atrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AAV2-GDNF gene therapyBiological
Bilateral image-guided infusion of AAV2-GDNF into putamen, single dose
Sham (Placebo) SurgeryProcedure or surgery
Bilateral partial burr/twist holes without dural penetration
| Study group | Role | What they receive |
|---|---|---|
| Active Treatment | Experimental | Not described Receives: Biological: AAV2-GDNF gene therapy |
| Placebo Surgery | Sham comparator | Not described Receives: Procedure: Sham (Placebo) Surgery |
What is being measured
The primary outcome is the main question the study is designed to answer.
The incidence of Treatment-Emergent Adverse Events (TEAE) and Serious Adverse Events (SAE) assessed clinically by physical and neurological examinations
Number of TEAE and SAE's reported post-treatment.
Measured: 3 years
Secondary outcomes (3)
MSA symptoms/signs as assessed by the Unified Multiple System Atrophy Rating Scale (UMSARS)
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.
Measured: 12 months
Change in striatal dopamine transporter binding as measured by [123-I] Ioflupane
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
Measured: 12 months
Change in the quality of life as measured by Multiple System Atrophy Quality of Life (MSA-QoL)
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.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Dec 2020
- Study start3 Oct 2023
- Primary completionAug 2026 (estimated)
- Study completionAug 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Multiple System AtrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- 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.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 9 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Brain Neurotherapy Bio, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04680065
- Sponsor's protocol ID
- MSA-101
- Status last verified
- Jan 2026
- First submitted
- 15 Dec 2020
- Last updated
- 30 Jan 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized, Double-Blind, Placebo-controlled Safety Study of Glial Cell Line-Derived Neurotrophic Factor Gene Transfer (AAV2-GDNF) in Multiple System Atrophy
- ClinicalTrials.gov ID
- NCT04680065
- Acronym / sponsor ID
- MSA-101
- Conditions
- Multiple System Atrophy
- Keywords
- MSA, Multiple System Atrophy, Neurotrophic factor, Growth factor, Glial cell line-derived neurotrophic factor, GDNF, AAV, Gene therapy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 9 participants (estimated)
- Lead sponsor
- Brain Neurotherapy Bio, Inc.Industry (pharma, biotech, devices)
- Study start
- 3 Oct 2023 (actual)
- Primary completion
- Aug 2026 (estimated)
- Study completion
- Aug 2028 (estimated)
- First posted
- 22 Dec 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- 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.
| Arm | Type | Receives |
|---|---|---|
| Active Treatment | Experimental | Biological: AAV2-GDNF gene therapy |
| Placebo Surgery | Sham comparator | Procedure: Sham (Placebo) Surgery |
Who may qualify
- Ages
- 35 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AAV2-GDNF gene therapyBilateral image-guided infusion of AAV2-GDNF into putamen, single dose | Biological | Active Treatment |
| Sham (Placebo) SurgeryBilateral partial burr/twist holes without dural penetration | Procedure or surgery | Placebo Surgery |
Primary outcome
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 secondary outcomes
MSA symptoms/signs as assessed by the Unified Multiple System Atrophy Rating Scale (UMSARS)
Time frame: 12 months
Change in striatal dopamine transporter binding as measured by [123-I] Ioflupane
Time frame: 12 months
Change in the quality of life as measured by Multiple System Atrophy Quality of Life (MSA-QoL)
Time frame: 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Jan 2026.
- Nurse Navigator Service for Potential ParticipantsLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing AAV2-GDNF gene therapy in people with Multiple System Atrophy, run by Brain Neurotherapy Bio, Inc. at 7 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 30 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The incidence of Treatment-Emergent Adverse Events (TEAE) and Serious Adverse Events (SAE) assessed clinically by physical and neurological examinations (time frame: 3 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Jan 2026.