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Gene Therapy
GDNF Gene Therapy for Multiple System Atrophy
Phase 1
Recruiting
Research Sponsored by Brain Neurotherapy Bio, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Stable anti-parkinsonian medication regimen
Male and female 35-75 years of age (inclusive)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 3 years
Awards & highlights
Study Summary
This trial is testing a gene therapy for safety and potential clinical effect in people with Multiple System Atrophy.
Who is the study for?
Adults aged 35-75 with Multiple System Atrophy (MSA) and parkinsonian symptoms, who can walk independently or with help. They must be diagnosed within the last 4 years, have a life expectancy over 3 years, and be on stable medication for MSA. Excluded are those with Parkinson's disease, dementia, psychosis, substance abuse issues, prior brain surgery or cancer.Check my eligibility
What is being tested?
This study tests AAV2-GDNF gene therapy versus a sham surgery to see if it's safe and effective for treating MSA. Participants will randomly receive either the real treatment directly into their brains or a placebo procedure without knowing which one they got.See study design
What are the potential side effects?
Potential side effects of AAV2-GDNF gene therapy may include reactions at the injection site in the brain, headache, nausea or other neurological changes due to its novel nature; however specific risks will be monitored closely given this is an early-phase trial.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
My Parkinson's medication dose has been stable.
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I am between 35 and 75 years old.
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I was diagnosed with MSA-Parkinsonian type after turning 30, and my symptoms have been getting worse.
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I can walk 25 feet with or without help.
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I was diagnosed with MSA after 30, showing mainly Parkinson-like symptoms.
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I am between 35 and 75 years old.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 3 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~3 years
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
The incidence of Treatment-Emergent Adverse Events (TEAE) and Serious Adverse Events (SAE) assessed clinically by physical and neurological examinations
Secondary outcome measures
Change in striatal dopamine transporter binding as measured by [123-I] Ioflupane
Change in the quality of life as measured by Multiple System Atrophy Quality of Life (MSA-QoL)
MSA symptoms/signs as assessed by the Unified Multiple System Atrophy Rating Scale (UMSARS)
Trial Design
2Treatment groups
Experimental Treatment
Placebo Group
Group I: Active TreatmentExperimental Treatment1 Intervention
Group II: Placebo SurgeryPlacebo Group1 Intervention
Find a Location
Who is running the clinical trial?
Brain Neurotherapy Bio, Inc.Lead Sponsor
1 Previous Clinical Trials
11 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- You have severe memory problems, hallucinations, drug addiction, or uncontrolled sadness.I was diagnosed with MSA parkinsonism less than 5 years ago and expect to live more than 3 years.My Parkinson's medication dose has been stable.I am between 35 and 75 years old.I can walk by myself or with help from a device.I was diagnosed with MSA less than 4 years ago and expected to live more than 3 years.I was diagnosed with MSA-Parkinsonian type after turning 30, and my symptoms have been getting worse.I can walk 25 feet with or without help.I was diagnosed with MSA after 30, showing mainly Parkinson-like symptoms.I have a history of cancer or medical conditions that make surgery risky.I have Parkinson's disease or a related genetic condition.I have had brain surgery or have abnormalities in brain scans.I cannot lay flat in an MRI or am allergic to gadolinium.I am between 35 and 75 years old.
Research Study Groups:
This trial has the following groups:- Group 1: Active Treatment
- Group 2: Placebo Surgery
Awards:
This trial has 0 awards, including:Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Multiple System Atrophy Patient Testimony for trial: Trial Name: NCT04680065 — Phase 1
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Has the FDA sanctioned AAV2-GDNF gene therapy?
"Safety assessments of AAV2-GDNF gene therapy produced a score of 1 since this is an experimental Phase 1 trial, meaning evidence for safety and efficacy is sparse."
Answered by AI
Is enrollment for this clinical research still available?
"According to clinicaltrials.gov, this investigation is actively seeking participants. The trial's announcement was made on August 1st 2022 and the post has been updated most recently on July 22nd 2022."
Answered by AI
Is this clinical trial open to individuals aged 45 or older?
"The prerequisites for participation in this study necessitate patients to be between the ages of 35 and 75. In contrast, there are 42 trials recruiting those under 18 years old and an additional 146 seeking individuals aged 65 or more."
Answered by AI
Who is the ideal participant for this clinical investigation?
"Only those who have multiple system atrophy and fall within the age range of 35 to 75 will be accepted into this trial, which is recruiting up to 9 patients."
Answered by AI
How many participants are there in the current clinical trial?
"Affirmative. Clinicaltrials.gov records demonstrate that this study is actively recruiting, with the original posting taking place on August 1st 2022 and later updates made on July 22nd of the same year. A total of nine patients have to be enrolled across four various sites."
Answered by AI
Who else is applying?
What state do they live in?
New York
Virginia
What portion of applicants met pre-screening criteria?
Met criteria
What site did they apply to?
University of California Irvine
Ohio Health
How many prior treatments have patients received?
0
Why did patients apply to this trial?
I have MSA and I'm looking for anything to help improve my condition. I am.also willing to help those that come after me.
PatientReceived 2+ prior treatments
DX PD ion 2018, but then diagnosed MSA May 1, 2023. Met with movement disorder specialist June 1 and dx confirmed there. I am willing to see what trials may be out there that might be beneficial to me. MSA has a short window, as we know, so being accepted into a trial soon is important.
PatientReceived 2+ prior treatments
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