GDNF Gene Therapy for Multiple System Atrophy

Launched by BRAIN NEUROTHERAPY BIO, INC. · Dec 20, 2020

Keywords

ClinConnect Summary

This clinical trial is studying a potential new treatment for Multiple System Atrophy (MSA), a rare and progressive neurological condition. The researchers are testing a gene therapy called AAV2-GDNF, which is delivered directly to a specific part of the brain (the putamen) to see if it can improve safety and possibly help with symptoms. This study is in its early phase (Phase 1), which means it is primarily focused on understanding if the treatment is safe for patients.

To participate in this trial, individuals need to be between 35 and 75 years old and have a confirmed diagnosis of MSA with symptoms that began after age 30. Eligible participants should also be able to walk at least 25 feet, with or without help, and must have been diagnosed with MSA within the last five years. Participants will be randomly assigned to receive either the gene therapy or a placebo (a treatment that looks the same but has no active ingredients) and will be monitored closely throughout the study. This trial is currently recruiting participants, and being part of it could help advance knowledge and treatment options for MSA in the future.

Gender

ALL

Eligibility criteria

• 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.