Frontotemporal Dementia Research Program

The goal of the ALLFTD study is to understand the changes in brain function that occur as a result of disease progression, and how changes differ from normal aging. By tracking changes in clinical features (symptoms, neurologic examination) and biomarkers (neuropsychological tests, blood proteins, MRI, CSF proteins) over several years, the investigators predict to see changes in biomarkers that precede clinical changes by years or even decades.

Study Details

Participants enrolled in ALLFTD are carefully assessed through interviews, physical examinations, cognitive testing and blood and/or CSF testing, and in many cases brain imaging, in order to describe how each person is affected, or not affected, by FTLD. Please see the ALLFTD website for more information about the study.

ALLFTD is currently enrolling participants with the conditions below:

• Behavioral variant of frontotemporal dementia (bvFTD)
• Behavioral variant of frontotemporal dementia with motor neuron disease (bvFTD-MND)
• Semantic variant of primary progressive aphasia (svPPA)
• Nonfluent variant of primary progressive aphasia (nfvPPA)
• Progressive supranuclear palsy (PSP)
• Corticobasal syndrome (CBS)
• Any member of a family with a history that suggests FTLD, including patients with symptoms of any form of FTLD and people who are part of these families but have either no symptoms or questionable symptoms

What can you expect?

ALLFTD currently targets mainly in-person assessment of participants with specific diagnoses in the FTLD spectrum, with the expectation that all patients will come for repeat assessments each year. For those who are unable or unwilling to return for annual evaluations, a more focused assessment of participants is performed. Also, every participant is required to identify an “informant” (usually a close friend or family member) who can provide their perspective on how the participants are doing.

ALLFTD participants have a comprehensive evaluation. This assessment usually takes about two full days to complete, often more, and so participants must return to the center from home multiple times within a couple of weeks or stay overnight nearby in order to complete them. The assessment includes:

• Interview and examination by a physician
• Cognitive testing using traditional “pencil and paper” tests as well as computerized cognitive testing
• Interview of an informant by a nurse or study coordinator
• Brain imaging using MRI
• Blood collection
• Lumbar puncture (often called a spinal tap) optional
• Participants and their informant also fill out questionnaires about various aspects of their life, including habits such as exercising, eating, occupation, hobbies, and use of cigarettes and drugs or alcohol.

The Principal Investigator for the study is Dr. Richard Ryan Darby, MD

This study is currently enrolling select participant. There is a waitlist for the study, so please reach out.  

For more detailed information on this study please contact:

Lead: Jerica Reeder, B.S.
Senior Program Manager
Phone: (615) 875-2987
Email: jerica.reeder@vumc.org

Lindsey Keener, B.A.
Clinical Translational Research Coordinator II
Phone: (615-875-8731)
Email: lindsey.keener@vumc.org

This study aims to understand social decision-making deficits in dementia patients as it pertains to legal complications and connect this with neurodegeneration patterns in brain imaging.

What can you expect?

Computer Tasks

When you participate in a computer study, you will be asked answer questions about how you would make moral and social decisions in certain situations. No computer experience is necessary to perform these tasks and rest breaks are provided frequently.

MRI

For the Magnetic resonance imaging (MRI) study, you will be asked to lie still while being scanned in the magnet for about 50 minutes for 1 session.

Questionnaires

A series of questionnaires will be given to the patient and their caregiver. The questionnaires will ask questions pertaining to social and moral decision-making skills and memory assessments. 

Eligibility:

• Males and Females
• Age 18 to 85 for symptomatic participants/genetic carriers
• Have a diagnosis of Behavioral Variant Frontotemporal Dementia, Alzheimer's, or Mild Cognitive Impairment. 
• Enrolling healthy controls (age 45-85)

Exclusion Criteria:

Unstable/untreated medical condition, history of mental illness, hearing/visual impairments; standard MRI exclusion criteria, including: claustrophobia, any trauma or surgery which may have left ferromagnetic material in the body, ferromagnetic implants or pacemakers, inability to lie still.

Participants will be compensated for their time. Travel expenses are reimbursed for participants who have to travel more than 15 miles.

The Principal Investigator for the study is Dr. Richard Ryan Darby, MD

This study is currently enrolling.

For more detailed information on this study please contact:

Levi Pettit, B.S.
Clinical Translational Research Coordinator I
Email: Levi.f.pettit@vumc.org

This is a phase 3, multicenter, randomize, double-blind, placebo-controlled clinical trial to evaluate the safety and efficacy of AMX0035 in patients with Progressive Supranuclear Palsy (PSP).

What can you expect?

Enrolled subjects will participate in a 52-week double-blind treatment period. During the year, patients and their study partner will come in to VUMC for 7 in-person clinic visits. Between the in-person visits will be 9 remote phone calls to check for study drug compliance and adverse events. After the 1 year-long double-blind treatment phase, patients can consent to participating in the Open Label Extension phase, where they will receive the study drug for the following 1 year.

Key Eligibility Criteria:

· Age 40 to 80

· Presence of PSP symptoms for less than 5 years

· Able to walk 5 steps with minimal assistance (stabilization of one arm)

· No evidence of any clinically significant neurological disorder other than PSP

· Willing to undergo 2 brain MRIs and 2 Lumbar Punctures during the year of treatment

The Principal Investigator for the study is Dr. Ryan Darby, MD

This study is currently enrolling.

For more detailed information on this study please contact:

Lead: Lindsey Keener, B.A.
Clinical Translational Research Coordinator II
Phone: (615) 875-8731
Email: lindsey.keener@vumc.org

This is a phase 2, multicenter, randomized, double-blind, placebo-controlled clinical trial to evaluate the safety and efficacy of FNP-223 in patients with Progressive Supranuclear Palsy (PSP).

What can you expect?

Enrolled subjects will participate in a 52-week double-blind treatment period. During the year, patients and their study partner will come in to VUMC for 7 in-person clinic visits. Between the in-person visits will be 9 remote phone calls to check for study drug compliance and adverse events

Key Eligibility Criteria:

· Age 50 – 80

· Presence of PSP symptoms for less than 3 years

· Able to walk 10 steps with minimal assistance

· No evidence of any clinically significant neurological disorder other than PSP

· 1 brain MRI required for screening

· 1 additional MRI and 2 lumbar punctures are optional

The Principal Investigator for the study is Dr. Ryan Darby, MD

This study is currently enrolling.

For more detailed information on this study please contact:

Lead: Lindsey Keener, B.A.
Clinical Translational Research Coordinator II
Phone: (615) 875-8731
Email: lindsey.keener@vumc.org

This Baylor-Vanderbilt collaborative research project is designed to study the effect of art therapy for people with Progressive Supranuclear Palsy (PSP).  

This study is funded by CurePSP.

What can you expect?

This study is fully remote, aside from an in-person consenting process that can occur at an already scheduled clinic appointment.

Visit 1: A remote visit with the study doctor, including a neurological examination and questionnaires

8 weeks of remote art therapy, with sessions lasting for 60 minutes once a week

Visit 2: A remote visit with the study doctor halfway through the art therapy course (at Week 4)

Visit 3: A remote visit with the study doctor after the 8 weeks of therapy have concluded

Eligibility:

• Diagnosis of Progressive Supranuclear Palsy (PSP)
• Having the cognitive ability to consent to participation 

The Principal Investigator for the study is Dr. Amy Brown, MD

This study is currently enrolling.

For more detailed information on this study please contact:

Lead: Lindsey Keener, B.A.
Clinical Translational Research Coordinator II
Phone: 615-875-8731
Email: lindsey.keener@vumc.org

More information coming soon. 

The primary purpose of this phase 3, multicenter, randomized, double-Blind, placebo-controlled study is to evaluate the efficacy and safety of AL001 in individuals at risk for or with Frontotemporal Dementia due to heterozygous mutations in the progranulin gene

What can you expect?

This study is enrolling symptomatic and pre-symptomatic patients who have a known progranulin genetic mutation. If genetic status is unknown, please contact the coordinator below to discuss available options for genetic testing, insurance information, and counseling. Eligible participants will be involved in monthly infusion visits that may also include blood sampling, MRI imaging, optional lumbar puncture, neuropsychological assessments, and other various clinical procedures.

Subjects enrolled in this placebo-controlled study will receive monthly infusions over the course of 1 to 2 years, after which they will be eligible for an open-label extension, on which every subject receives the drug. 

The Principal investigator for the study is Dr. Lealani Acosta MD, MPH.

Enrollment is closed.

For more detailed information on this study please contact:

Lead: Jerica Reeder, B.S.
Senior Program Manager
Phone: (615) 875-2987
Email: jerica.reeder@vumc.org