Working… Menu

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 : Not yet recruiting
First Posted : December 22, 2020
Last Update Posted : April 15, 2021
Information provided by (Responsible Party):
Brain Neurotherapy Bio, Inc.

Tracking Information
First Submitted Date  ICMJE December 15, 2020
First Posted Date  ICMJE December 22, 2020
Last Update Posted Date April 15, 2021
Estimated Study Start Date  ICMJE April 2021
Estimated Primary Completion Date April 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 20, 2020)
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.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 20, 2020)
  • 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.
  • 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
  • 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.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
Descriptive Information
Brief Title  ICMJE GDNF Gene Therapy for Multiple System Atrophy
Official Title  ICMJE Randomized, Double-Blind, Placebo-controlled Safety Study of Glial Cell Line-Derived Neurotrophic Factor Gene Transfer (AAV2-GDNF) in Multiple System Atrophy
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE 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
Condition  ICMJE Multiple System Atrophy
Intervention  ICMJE
  • Biological: AAV2-GDNF gene therapy
    Bilateral image-guided infusion of AAV2-GDNF into putamen, single dose
  • Procedure: Sham (Placebo) Surgery
    Bilateral partial burr/twist holes without dural penetration
Study Arms  ICMJE
  • Experimental: Active Treatment
    Intervention: Biological: AAV2-GDNF gene therapy
  • Sham Comparator: Placebo Surgery
    Intervention: Procedure: Sham (Placebo) Surgery
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Not yet recruiting
Estimated Enrollment  ICMJE
 (submitted: December 20, 2020)
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE April 2024
Estimated Primary Completion Date April 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Male and female adults 35-75 years of age (inclusive)
  • Diagnosed with MSA with sporadic, adult-onset (>30 yo) with predominant parkinsonian symptoms
  • Less than 4 years from clinical diagnosis of MSA with expected survival > 3 years
  • Stable medication regimen
  • Ability to walk with or without an assistive device

Exclusion Criteria:

  • Presence of idiopathic Parkinson's disease or other neurological diseases
  • Myocardial sympathetic denervation inconsistent with an MSA diagnosis
  • Presence of dementia, psychosis, substance abuse or poorly controlled depression
  • Prior brain surgery (i.e. deep brain stimulator) or other brain imaging abnormalities
  • Receiving an investigational drug
  • History of cancer or poorly controlled medical conditions that would increase surgical risk
  • Inability to tolerate laying flat in an MRI or allergy to gadolinium

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 35 Years to 75 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Massimo Fiandaca, MD
Contact: Amber Van Laar, MD
Listed Location Countries  ICMJE United States
Removed Location Countries  
Administrative Information
NCT Number  ICMJE NCT04680065
Other Study ID Numbers  ICMJE MSA-101
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Brain Neurotherapy Bio, Inc.
Study Sponsor  ICMJE Brain Neurotherapy Bio, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Brain Neurotherapy Bio, Inc.
Verification Date April 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP