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Active, Not Recruiting

NCT Number: NCT04680065

GDNF Gene Therapy for 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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Multiple System Atrophy Autonomic Nervous System Diseases Basal Ganglia Diseases Brain Diseases Central Nervous System Diseases Movement Disorders Nervous System Diseases Neurodegenerative Diseases Primary Dysautonomias Synucleinopathies

Age range

35 year–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

University of California Irvine, Irvine, California, United States

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

AAV2-GDNF gene therapy

Biological

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

Sham (Placebo) Surgery

Procedure

Bilateral partial burr/twist holes without dural penetration

Primary outcomes

  1. 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.

Secondary outcomes

  1. 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.

  2. 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

  3. 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.

Sponsors and collaborators

Lead sponsor

Brain Neurotherapy Bio, Inc.

Industry

Registry information

Official study title

Randomized, Double-Blind, Placebo-controlled Safety Study of Glial Cell Line-Derived Neurotrophic Factor Gene Transfer (AAV2-GDNF) in Multiple System Atrophy

Important dates

Study start
2023
Primary completion
2026
Study completion
2028
First posted
Dec 22, 2020
Registry last updated
Jan 30, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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