NCT04680065

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.

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
9

participants targeted

Target at below P25 for phase_1

Timeline
27mo left

Started Oct 2023

Longer than P75 for phase_1

Geographic Reach
1 country

7 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress54%
Oct 2023Aug 2028

First Submitted

Initial submission to the registry

December 15, 2020

Completed
7 days until next milestone

First Posted

Study publicly available on registry

December 22, 2020

Completed
2.8 years until next milestone

Study Start

First participant enrolled

October 3, 2023

Completed
2.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2026

Expected
2 years until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2028

Last Updated

January 30, 2026

Status Verified

January 1, 2026

Enrollment Period

2.8 years

First QC Date

December 15, 2020

Last Update Submit

January 28, 2026

Conditions

Multiple System Atrophy

Keywords

MSAMultiple System AtrophyNeurotrophic factorGrowth factorGlial cell line-derived neurotrophic factorGDNFAAVGene therapy

Outcome Measures

Primary Outcomes (1)

  • 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.

    3 years

Secondary Outcomes (3)

  • MSA symptoms/signs as assessed by the Unified Multiple System Atrophy Rating Scale (UMSARS)

    12 months

  • Change in striatal dopamine transporter binding as measured by [123-I] Ioflupane

    12 months

  • Change in the quality of life as measured by Multiple System Atrophy Quality of Life (MSA-QoL)

    12 months

Study Arms (2)

Active Treatment

EXPERIMENTAL
Biological: AAV2-GDNF gene therapy

Placebo Surgery

SHAM COMPARATOR
Procedure: Sham (Placebo) Surgery

Interventions

AAV2-GDNF gene therapyBIOLOGICAL

Bilateral image-guided infusion of AAV2-GDNF into putamen, single dose

Active Treatment
Sham (Placebo) SurgeryPROCEDURE

Bilateral partial burr/twist holes without dural penetration

Placebo Surgery

Eligibility Criteria

Age35 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (7)

University of California Irvine

Irvine, California, 92697, United States

Location

Parkinson's Disease and Movement Disorders Center of Boca Raton

Boca Raton, Florida, 33486, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Quest Research Institute

Farmington Hills, Michigan, 48334, United States

Location

NYU Langone Health

New York, New York, 10016, United States

Location

The Ohio State University Medical Center

Columbus, Ohio, 43210, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

Related Links

MeSH Terms

Conditions

Multiple System Atrophy

Interventions

salicylhydroxamic acidSurgical Procedures, Operative

Condition Hierarchy (Ancestors)

Primary DysautonomiasAutonomic Nervous System DiseasesNervous System DiseasesBasal Ganglia DiseasesBrain DiseasesCentral Nervous System DiseasesMovement DisordersSynucleinopathiesNeurodegenerative Diseases

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Model Details: 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.
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 15, 2020

First Posted

December 22, 2020

Study Start

October 3, 2023

Primary Completion (Estimated)

August 1, 2026

Study Completion (Estimated)

August 1, 2028

Last Updated

January 30, 2026

Record last verified: 2026-01

Data Sharing

IPD Sharing
Will not share

Locations

US(7)