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GDNF Gene Therapy for Multiple System Atrophy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT04680065
Recruitment Status : Recruiting
First Posted : December 22, 2020
Last Update Posted : October 16, 2023
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
Multiple System Atrophy Biological: AAV2-GDNF gene therapy Procedure: Sham (Placebo) Surgery Phase 1

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 9 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention 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 (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Randomized, Double-Blind, Placebo-controlled Safety Study of Glial Cell Line-Derived Neurotrophic Factor Gene Transfer (AAV2-GDNF) in Multiple System Atrophy
Actual Study Start Date : October 3, 2023
Estimated Primary Completion Date : December 2025
Estimated Study Completion Date : December 2028

Arm Intervention/treatment
Experimental: Active Treatment Biological: AAV2-GDNF gene therapy
Bilateral image-guided infusion of AAV2-GDNF into putamen, single dose

Sham Comparator: Placebo Surgery Procedure: Sham (Placebo) Surgery
Bilateral partial burr/twist holes without dural penetration

Primary Outcome Measures :
  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 Outcome Measures :
  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.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   35 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT04680065

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Contact: Akash Pandhare, MD 336-265-2790
Contact: Christian Urrea, MD 336-265-2790

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United States, California
University of California Irvine Recruiting
Irvine, California, United States, 92697
Principal Investigator: Nicolas Phielipp, MD         
United States, Massachusetts
Massachusetts General Hospital Active, not recruiting
Boston, Massachusetts, United States, 02114
United States, Michigan
Quest Research Institute Recruiting
Farmington Hills, Michigan, United States, 48334
Contact: Renee Najor   
Principal Investigator: Aaron Ellenbogen, DO         
United States, Ohio
The Ohio State University Medical Center Active, not recruiting
Columbus, Ohio, United States, 43210
Sponsors and Collaborators
Brain Neurotherapy Bio, Inc.
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Responsible Party: Brain Neurotherapy Bio, Inc. Identifier: NCT04680065    
Other Study ID Numbers: MSA-101
First Posted: December 22, 2020    Key Record Dates
Last Update Posted: October 16, 2023
Last Verified: October 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Brain Neurotherapy Bio, Inc.:
Multiple System Atrophy
Neurotrophic factor
Growth factor
Glial cell line-derived neurotrophic factor
Gene therapy
Additional relevant MeSH terms:
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Multiple System Atrophy
Shy-Drager Syndrome
Pathological Conditions, Anatomical
Primary Dysautonomias
Autonomic Nervous System Diseases
Nervous System Diseases
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Movement Disorders
Neurodegenerative Diseases
Vascular Diseases
Cardiovascular Diseases