GDNF Gene Therapy for Multiple System Atrophy

Multiple System Atrophy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04680065
• Other study ID #: MSA-101
• Status: Recruiting
• Phase: Phase 1
• Start date: October 3, 2023
• Est. completion date: December 2028

Study information

• Verified date: April 2024
• Source: Brain Neurotherapy Bio, Inc.
• Contact: Akash Pandhare, MD
• Phone: 336-265-2790
• Email: askfirst[@]askbio.com
• Is FDA regulated: No
• Study type: Interventional

Clinical 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 9
• Est. completion date: December 2028
• Est. primary completion date: December 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 35 Years to 75 Years

Eligibility

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.

Related Conditions & MeSH terms

• Atrophy
• Multiple System Atrophy
• Shy-Drager Syndrome

Intervention

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

Locations

Country	Name	City	State
United States	Parkinson's Disease and Movement Disorders Center of Boca Raton	Boca Raton	Florida
United States	Massachusetts General Hospital	Boston	Massachusetts
United States	The Ohio State University Medical Center	Columbus	Ohio
United States	Quest Research Institute	Farmington Hills	Michigan
United States	University of California Irvine	Irvine	California

Sponsors (1)

Lead Sponsor	Collaborator
Brain Neurotherapy Bio, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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.	12 months
Secondary	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	12 months
Secondary	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.	12 months

External Links

•   Nurse Navigator Service for Potential Participants