GDNF Gene Therapy for Multiple System Atrophy

Phase 1Active Not RecruitingNCT04680065

Brain Neurotherapy Bio, Inc.9 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Multiple System Atrophy

Interventions

AAV2-GDNF gene therapyBIOLOGICAL

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

Sham (Placebo) SurgeryPROCEDURE

Bilateral partial burr/twist holes without dural penetration

Eligibility

Sex: ALLMin age: 35 YearsMax age: 75 Years

Medical Language ↔ Plain English

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.

Locations (7)

University of California Irvine

Irvine, California, United States

Parkinson's Disease and Movement Disorders Center of Boca Raton

Boca Raton, Florida, United States

Massachusetts General Hospital

Boston, Massachusetts, United States

Quest Research Institute

Farmington Hills, Michigan, United States

Outcomes

Primary Outcomes

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.

Time frame: 3 years

Secondary Outcomes

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.

Time frame: 12 months

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

Time frame: 12 months

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.

Time frame: 12 months

Data from ClinicalTrials.gov

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