The Motivations for Genetic Testing study seeks to learn what people consider when deciding whether or not to learn their genetic status, especially when considering clinical trial participation. To be eligible, participants must be at least 18 years old and be able to read English. They must be a blood relative of someone who has a neurodegenerative disease, and cannot have a diagnosis themselves. Additionally, they must meet one of these criteria related to genetic testing for a neurologic condition:

• Has personally had genetic testing (no matter the result)
• Is considering having genetic testing
• Has decided not to have genetic testing

Participation includes filling out a short online survey that should take 15 to 20 minutes. The study aims to enroll 300 people: 100 at risk for Alzheimer’s Disease, 100 at risk for FTD, and 100 at risk for Huntington’s Disease. For more information, contact Laynie Dratch, ScM, CGC, at laynie.dratch@PennMedicine.upenn.edu or access the survey here.

A Phase 2a Study of TPN-101 in Patients with C9orf72 ALS/FTD (Amyotrophic Lateral Sclerosis and/or Frontotemporal Dementia)

Transposon Therapeutics, Inc., is studying TPN-101 as a new experimental drug for frontotemporal dementia (FTD) caused by mutations in the C9orf72 gene.

Brain nerve cells in patients with FTD due to a mutation in the C9orf72 gene produce harmful DNA, RNA, and proteins. These products cause the cells to become inflamed and damaged, leading to the symptoms of FTD. These effects may be blocked by inhibiting an enzyme in the cell called LINE-1 reverse transcriptase.

TPN-101 is a new inhibitor of this enzyme. The purpose of the study is to learn whether TPN-101 can reduce the damage to nerve cells and improve the symptoms of patients with FTD, when compared with a placebo.

Each patient will be evaluated to determine their eligibility. You may qualify if:

• You have a diagnosis of FTD based on symptoms such as mild cognitive impairment, mild behavioral impairment, mild cognitive/behavioral impairment, behavioral variant FTD, primary progressive aphasia, or amnestic syndrome
• You have a mutation in the C9orf72gene

Information about the study:

• You will take an oral capsule of TPN-101 or a placebo once a day for 24 weeks
• There is a 60% chance that you will get TPN-101 and a 40% chance that you will get placebo
• After 24 weeks of treatment, everyone will get 24 weeks of TPN-101
• You will need to visit the study site 12 or 13 times over the year. Each visit will last between 2 hours and half a day
• Assessments will include medical examinations, blood tests, 3 lumbar punctures for collection of CSF during the full year of the study, and completion of questionnaires

For more information about the study and research sites participating in the study, call 310-261-5312 or visit ClinicalTrials.gov.

TPN-101 has not been approved by the FDA or any other health authority.

Yale University School of Medicine and Yale New Haven Hospital are conducting a neuroimaging study. This study will help researchers better understand bvFTD and may lead to a new biomarker that could help detect and diagnosis bvFTD earlier.

WHO: People with bvFTD, ages 40-85.

WHAT: Participation involves one phone screening and at least 3 in-person visits:

• Day One: screening appointment (approx. 60 mins) with a single MRI scan (another 60 mins). Surveys and assessments (about 60 mins), both self-reported and researcher administered pen-and-paper tasks.
• Day Two: a single PET scan (approx. 120 mins). Surveys and assessments (about 60 mins), both self-reported and researcher administered pen-and-paper tasks.
• Day Three: a single PET scan (approx. 90 mins)

Study procedures will include: A screening appointment, a magnetic resonance imaging (MRI) scanning session, and up to three Positron Emission Tomography (PET) scanning sessions. During the PET scanning sessions, we will use an investigational radiotracer and an FDA-approved radiotracer. A radiotracer is a low dose of a drug that has a radioactive marker attached to it, so that it can be detected in the brain. The radiotracer gives off energy (gamma-rays) that is detected by the PET scanner and used to create brain images.

WHERE: Yale University PET Center (801 Howard Avenue, New Haven) for screening and PET scan. Anlyan Center (300 Cedar Street, New Haven) for MRI. Transportation arrangements can be made on a case-by-case basis.

WHEN: Flexible with your schedule, but most weekdays for visits. Phone screenings can occur any weekday.

Study Contacts:
David Matuskey, MD, 203-737-6316, david.matuskey@yale.edu
Yanghong Yang, PhD, 203-785-5054,  yanghong.yang@yale.edu
Arman Fesharaki MD, PhD, 203-415-9382, arman.fesharaki@yale.edu

Recruiting from January 2022 through June 2022

Emory University is conducting a small study of adolescents who live at home with a parent with young-onset dementia (YOD).

THE IDEA

• There are over 1.5 million individuals living with YOD in the U.S.
• Few studies have highlighted adolescents’ experiences of living with a parent with YOD.
• Little is known about the details of these adolescents’ lives, including their involvement in caregiving, how they navigate changes in family structure, and how their overall experiences shape who they are as individuals.

PURPOSE

• The goal of this study is to describe the experiences of adolescents living with a parent with YOD and how their experiences shape their developing identities.
• Your adolescent will be asked to complete a brief online questionnaire as well as a 60-minute interview via Zoom.
• The interview is focused on understanding your adolescent’s overall experiences, including their potential involvement in caregiving.
• Your adolescent will receive a $25 Visa gift card for their participation.

ELIGIBILITY CRITERIA

• Adolescents (ages 13-19) who have a parent with YOD
• Born in raised in two-parent household with the affected parent
• Currently lives at home with both parents
• Speaks fluent English
• Has internet access

IF INTERESTED, CONTACT:
Elizabeth Bilsborough
ebilsbo@emory.edu | 404-220-4226

Download this flyer for more information.

Recruiting through 2027

The up-LIFT-D study is investigating a gene therapy (PBFT02) that may treat individuals with frontotemporal dementia (FTD) who also have a mutation in the progranulin gene (GRN). Gene therapy aims to replace the gene that is not working properly with a version of the gene that works normally.

In the up-LIFT-D study, PBFT02 will be given once as an injection into an area at the back of the neck called the cisterna magna. This procedure will be done in a hospital under general anesthesia or sedation. The normal gene will be delivered using a carrier called a vector. A vector is a virus that has been changed so that it no longer causes disease, but instead includes a gene, in this case the GRN gene that normally makes progranulin. The virus used in this study is called an adeno-associated virus (AAV1) which has been engineered to carry the normal GRN gene to the brain.

You may be eligible for the up-LIFT-D study if:

• You are 35 to 75 years old
• You have been diagnosed with FTD and have a documented progranulin gene mutation
• You are experiencing symptoms related to FTD
• You have a reliable study partner, such as a family member or close friend, who can attend study visits with you
• You are living in the community

Information about the study:

• PBFT02 will be administered once
• Participation in this study will be for approximately 16 visits over 5 years
• Assessments will include blood tests, regular medical examinations, completion of questionnaires and assessments to evaluate disease progression, brain imaging, and lumbar punctures for CSF testing
• Travel reimbursement for study visits will be provided

Click here for more information about the study and research sites participating in the study.

PBFT02 has not been approved by the FDA or any other health authority approval around the world.

Recruiting from October 2021 through 2023

The WeCareAdvisor is an innovative online platform developed by researchers at Drexel University and the University of California, Davis to help family caregivers manage common behavioral and psychological symptoms of dementia like anger, irritability, asking repeated questions, pacing, or refusing needed help. WeCareAdvisor can be accessed on any web-enabled device.

The WeCareAdvisor walks caregivers through a step-by-step approach, referred to as The DICE Approach™, to describe a behavior he/she seeks to address, investigate factors that may contribute to the behavior, create a plan or set of strategies (DICE), and evaluate if strategies worked or not and if additional strategies are needed to manage the behavior. The WeCareAdvisor also provides helpful daily tips and education about dementia.

We are testing whether use of the WeCareAdvisor helps family caregivers better manage challenging behaviors, reduces feelings of stress and upset with behavioral symptoms, and enhances confidence. This research is led by Dr. Laura N. Gitlin of Drexel University and Dr. Helen C. Kales of University of California, Davis and their research teams.

You may be eligible to participate if you are:

• 21 years of age or older
• Able to read, speak, and understand English
• The primary caregiver of a person living with dementia for at least 6 months
• Currently managing challenging dementia-related behaviors or psychological symptoms
• Own and are comfortable using a computer, smartphone, or tablet
• Have internet access

If this study is right for you, you will be asked to participate in an initial telephone interview with a trained member of the research team to learn about your health and the wellbeing of the person you care for. All caregivers are then assigned by chance (randomly, like a flip of a coin) to either a group that receives the WeCareAdvisor immediately or to a group that waits three months prior to starting its use. Regardless of group assignment, all participants in this study will have an opportunity to use the WeCareAdvisor. All caregivers are then interviewed by telephone again at one (1), three (3) and six (6) months after starting the study.

Upon completion of each interview, caregivers will receive an Amazon gift card for $15 (up to $60 total for study).

To see if this study is right for you and to learn more about the study, please email us at WeCare@drexel.edu, call us at 267-359-1111, or visit WeCareAdvisorStudy.com and a WeCareAdvisor study team member will reach out to you.

Recruiting through 2022

Researchers at the University of California, San Francisco are inviting caregivers nationwide who live in rural or farming areas and care for someone living with frontotemporal degeneration or related dementias to participate in the Rural Dementia Caregiver Project.

In this project, we are offering a free online workshop called Building Better Caregivers that lasts six weeks. Thanks to a grant from the National Institute on Aging, there is no cost to caregivers for attending.

The workshops teach caregivers how to reduce their stress, take better care of themselves, and manage the changes in memory, behavior, thinking, or mood of the family member or friend they are caring for. Caregivers can access the workshop anytime (day or night), and can participate using a smartphone, tablet, or computer. Zoom and video capabilities are not required to participate. During the workshop, participants communicate with each other through discussion boards, rather than having everyone online at the same time. Caregivers can also remain anonymous, and participants can choose a screen name to protect their privacy.

You may be eligible to participate if you:

• Live in a rural or farming area of the United States
• Give care at least 10 hours a week for a family member or friend with frontotemporal degeneration or a related diagnosis (e.g., give help with dressing, meals, transportation, medications, appointments, or similar support)
• Are 18 years of age or older
• Read and write English
• Have internet access

Because this is a study, caregivers will be randomly assigned to one of two groups by a computer. Half of the caregivers will receive the Building Better Caregivers workshop right away. The other half will have to wait 12 months. Caregivers who have to wait will receive a free caregiver handbook on tips and resources, and support phone calls from our trained staff. All caregivers will be asked to complete four surveys on their caregiving experiences and will receive up to $80 for doing so.

For more information, visit our website: caregiverproject.ucsf.edu. Want to see if you are eligible? Complete an eligibility survey!

Project Contact Information:
Rural Dementia Caregiver Project | 1-833-634-0603 (toll-free) | caregiverproject@ucsf.edu

Researchers at Oregon Health & Science University are seeking volunteers to help design an education and support program (STELLA-FTD) that addresses the specific needs of family caregivers for those with FTD. Participants will attend one 60-90–minute focus group, review printed material, and complete a brief survey. All study activities can be done from your home — no travel is needed — and gift cards up to $25 may be provided for completing all study activities.

You may be eligible to participate if:

• You currently care for a family member with FTD
• You previously cared for a family member with FTD who has recently passed away
• You have a computer and can participate in an online focus group

For more information about the study, contact Dr. Allison Lindauer at telestella@ohsu.edu or complete this survey.

Researchers at Nipissing University are inviting persons with lived experience of rare or young-onset forms of dementia to participate in individual or group interviews, completion of questionnaires, or engagement using arts-based methods.

These learning activities will help us document the experience of living with or caring for someone living with a rare or young-onset dementia, contributing to better understanding of the unique needs associated with atypical dementias and development of tailored support services to meet these needs.

Your participation is completely voluntary, and you can choose to participate in one or all aspects of the study. Each of these activities will last about two hours, will occur once a year for three years (until December 2023), and will be conducted virtually using GoToMeeting video conferencing.

You can participate in the study if you are:

• Living with FTD or other rare forms of dementia (e.g., posterior cortical atrophy, young-onset Alzheimer’s disease)
• A current or former care partner to someone living with FTD or other rare forms of dementia
• Working with or have a professional interest in FTD or other rare forms of dementia
• Residing in Canada
• Can understand and communicate in English

The study is a five-year (2020 to 2024) cross-national collaboration between Nipissing University; University College London (UCL), England; and Bangor University, Wales led by Prof Sebastian Crutch of the UCL Dementia Research Centre. Ethics approval has been obtained from Nipissing University Research Ethics Board.

For more information about the study, please contact Dr. Adetola Grillo (adetolag@nipissingu.ca) or the Principal Investigator, Dr. Mary Pat Sullivan (maryps@nipissingu.ca).

A Phase 3 clinical study investigating the efficacy of AL001 in slowing the progression of FTD in progranulin gene mutation carriers

Alector, Inc. is studying AL001 as a new experimental drug for frontotemporal dementia (FTD) caused by mutations in the progranulin gene. These mutations reduce progranulin levels in the body and may lead to symptoms of FTD.

The purpose of the phase 3 study is to learn if increasing progranulin levels with treatment of AL001 will delay onset of symptoms or slow disease progression, when compared to a placebo (a solution that contains no active AL001 drug).

Each individual will be evaluated to determine their eligibility. You may qualify if:

• You have a progranulin gene mutation and are at risk of developing FTD symptoms as evidenced by a biomarker, or
• You are diagnosed with FTD and have a progranulin gene mutation.

Information about the study:

• AL001 or a placebo will be administered every 4 weeks by an intravenous (IV) infusion.
• Assessments will include regular medical examinations, blood tests, brain imaging, and completion of questionnaires.
• For people who do not have FTD symptoms:
  • You will be in the study about 2 years
  • You will need to visit the study site at least 1 time per month for 2 years
• For people who are diagnosed with FTD:
  • You will be in the study a little over 1 year
  • You will need to visit the study site at least 1 time per month for 1 year

For more information about the study and research sites participating in the study, call 1-833-FindFTD (1-833-346-3383) or visit https://clinicaltrials.gov/ct2/show/NCT04374136.

AL001 has not been approved by the FDA or any other health authority approval around the world.

Researchers at University College London are conducting a study of Ignite, an iPad-based cognitive assessment app designed to detect early signs of FTD. Unlike other computerized neuropsychology test batteries, Ignite tests cognitive functions known to be affected early in FTD, including social skills, attention, and problem-solving abilities, rather than being focused on memory.

Ignite includes a set of short game-like tasks that measure these different brain functions. Completing all of the tasks will take approximately 20-30 minutes. The researchers aim to generate a baseline for performance on the tests from a large population of healthy individuals. The results will allow them to create a benchmark that can be compared to individuals at risk of developing FTD in future studies.

You can participate in this study if:

• You are a healthy individual between 20 and 80 years of age
• You own an iPad (The Ignite app is compatible with all versions of iPad but not with iPhones or any other devices, i.e. Androids or other smartphones)
• You can understand and comply with instructions in English

Ignite is an online study. Participants will download Ignite for free from the App Store onto their personal iPad, by searching “IGNITE App”, and complete the assessment. No personal data are collected and therefore the results of this study are completely anonymous.

The PROCLAIM study is researching the safety and effectiveness of an investigational one-time gene therapy (PR006) that is designed to potentially treat patients diagnosed with a form of FTD caused by a mutation, or change, in the gene progranulin (GRN). This gene provides instructions to cells to make a protein called progranulin.

In the PROCLAIM study, a carrier called a vector is genetically engineered to deliver a normal copy of the progranulin gene. The vector is called adeno-associated virus 9 (AAV9). These viruses are specially modified so they cannot cause disease in people, and the body’s immune system clears the virus after the gene enters the cell nucleus and the virus is no longer needed. Gene delivery with an AAV9 vector has a track record of efficacy and safety. While this particular gene therapy has never been used before in humans, AAV-based gene therapies have been successfully used in treatments for other serious illnesses, including spinal muscular atrophy and retinal dystrophy.

The investigational drug will be injected into the back of the head near the top of the neck, into an area near the brain called the cisterna magna. This procedure will be done in the hospital under either general anesthesia or deep sedation. The study will examine if the investigational drug travels to the brain cells that contain the GRN mutation and successfully corrects or modifies the effects of the mutation.

You may be eligible for the PROCLAIM study if:

• You are between 30 and 80 years of age
• You have been diagnosed with FTD with a disease-causing GRN mutation
• You are experiencing symptoms related to FTD (examples include personality changes or changes in language skills)
• You have a study partner — i.e., someone who is close to you, such as a family member or close friend — who can attend study visits with you

Study participation will last for up to five years and will involve 20 visits to the study center, including one three-day visit, two or three days of which will be inpatient.

Additional information from AFTD:
PR006, the investigational drug being tested in the PROCLAIM study, is intended to treat the underlying cause of FTD-GRN at the genetic level. The study drug will be administered via an injection through the base of the skull near the back of the neck, and performed by a neurosurgeon or an interventional radiologist. The neurosurgeon or interventional radiologist will use computed tomography scans to safely guide the injection needle into the appropriate area. All participants taking part in this study will also receive the steroids prednisone and methylprednisolone as well as the immunosuppressant sirolimus. These medications are to reduce the risk of experiencing a reaction or other side effects during and after the injection.

As a part of baseline and follow-up visits, participants will undergo blood draws, electrocardiograms, MRI scans, MRA scans, DEXA scans, and lumbar punctures for cerebrospinal fluid (CSF) testing. Travel reimbursement for study visits will be provided.

Contact:
Prevail Therapeutics │ 917-336-9310 │ patients@prevailtherapeutics.com

Study site contact:
Florida
Jessica Garaycoa │ 689-216-3100 │ Jessica.Garaycoa@ppdi.com

The Frontotemporal Degeneration Center and the Linguistic Data Consortium at the University of Pennsylvania are working to develop simple, easy, and effective ways to track neurocognitive health through short interactions with a web app. You can help us create a large open dataset, which researchers all over the world will use to create and test automated methods to track neurocognitive health.

Today, diagnosing neurodegenerative disorders like Alzheimer’s Disease or Frontotemporal Degeneration depends on someone scoring below a low threshold on clinically administered tests, which often means that they’ve been suffering from the disease for some time. Often the time to diagnosis can be a decade or more if you live remotely or the condition has an uncommon clinical presentation. Easy and reliable tracking over time, including remote monitoring, will let us identify early changes and allow more timely diagnosis, and to see how neurocognitive health changes over time. The same methods will support large-scale evaluation of interventions, whether those are medications or lifestyle changes.

This project is not just aimed at participants with cognitive impairment, or even at elderly people — we need all ages and backgrounds and conditions. The tasks involve oral descriptions of two pictured scenes, and naming as many words as you can in a minute that are from a target category. We will publish an anonymized version of the data for use by research groups worldwide.

To participate in the first step of this project, which should take less than five minutes of your time, click here.

Recruiting from August 2017 to September 2023

The “FOXY” study will use a medication called oxytocin, a hormone normally found in the brain which is related to social behavior. The oxytocin will be given as a nasal spray twice daily with the assistance of the care partner. To participate in this clinical trial, you must have a diagnosis of behavioral variant FTD, or semantic dementia (also called semantic type aphasia; non-fluent aphasia; or non-fluent, agrammatic type aphasia). Participation in this study is voluntary.

You may be eligible to participate if:

• You have a diagnosis of probable bvFTD, semantic dementia or non-fluent type aphasia
• You currently have symptoms of apathy/indifference as measured on the NPI scale
• You have a care partner or family member who can assist with administration of the nasal spray
• You are between 30-80 years of age and have no history of stroke or neurologic disorder other than FTD

The study will last for 22 weeks, which will include five visits to the clinic site. A care partner/family member who lives with the person with FTD, or sees them for at least three hours per day, is required to administer the nasal spray. The care partner/family member must be able to accompany the person with FTD to all study clinic visits. Each study site will provide reimbursement for travel expenses to the clinic according to their site guidelines.

There are six participating sites in the U.S., located in Los Angeles, San Francisco, Seattle, Baltimore, New York City and Boston. There are four participating sites in Canada, located in Vancouver, London, Toronto and Montreal.

For more details on participant eligibility, please visit ClinicalTrials.gov.

If you are interested in participating in this study or would like more information, please contact Kristy Coleman (CognitiveNeurology@sjhc.london.on.ca).

Recruiting through June 2022

Researchers at the University of Texas at Austin’s Aphasia Research Treatment Lab and the UCSF Memory and Aging Center are conducting a study to develop effective speech and language treatment options for individuals with primary progressive aphasia (PPA). They are investigating treatment approaches that can be delivered in person or via the computer, and hope to learn about both immediate benefits of therapy, as well as long-term outcomes. In addition, eligible individuals may undergo MRI scanning in order to inform researchers about how brain structure and function affect response to therapy.

You may be eligible to participate if you:

• Have a diagnosis of primary progressive aphasia (PPA)
• Do not have any other neurological or psychiatric diagnoses
• Do not have uncorrected visual impairments
• Speak English, Spanish, or are bilingual (i.e., English + any other language)

Participation in this speech and language treatment study can occur via teletherapy or in person (for those who live locally to one of our research sites: Austin, Texas or San Francisco, California).

Note that other eligibility criteria may apply. An initial screening will take place with a research coordinator in order to determine eligibility for participation.

Participation in the study will include pre-treatment assessment, tailored speech and language treatment and post-treatment assessment. Testing and treatment are completed over the course of 3-4 months. During the assessment phases, participants will meet with a clinician 2-3 times weekly for testing sessions (about 1-2 hours per session). During the treatment phase, participants will meet with a clinician twice weekly for therapy sessions (about one hour per session). Independent daily home practice will occur over the computer.

The participant and a care partner may be asked to travel to one of the research sites during the pre-treatment and/or post-treatment assessment phases. Airfare, lodging and a fixed stipend will be provided.

Follow-up assessment sessions will take place at 3-, 6-, and 12-months after treatment ends. Participation in the study is completely free of charge.

Contact:
Lisa Wauters, M.A., CCC-SLP| 512-471-3420| lisa.wauters@austin.utexas.edu
Carly Miller, M.S., CCC-SLP| Carly.Miller@ucsf.edu

For more information, click here.

Recruiting from September 2007 to April 2027

The Language in Primary Progressive Aphasia (PPA) study aims to understand the behavioral, anatomical and physiological changes in people with PPA throughout the course of the illness. The researchers in this study want to increase awareness of PPA, educate others about this unique disorder, and to encourage more research to eventually develop therapies.

During the three-day research program, participants will be asked to undergo neuropsychological testing (paper and pencil tests that evaluate cognition), advanced imaging (MRI and PET), eyetracking experiments, and other computer and language testing batteries. Participants may be asked to return every two years to complete the same measures.

For participants not living near Chicago, IL, flights and accommodations (for both the person with PPA and their companion) will be covered by the study.

Additional information from AFTD:
During this three-day research program, participants will be asked to undergo neuropsychological testing (paper and pencil tests that evaluate cognition), an MRI (a non-invasive brain-imaging procedure), eyetracking experiments and other computer and language testing batteries. Participants may be asked to return every two years to complete the same measures.

The study will recruit approximately 15 participants with PPA per year. For participants not living near Chicago, IL, flights and accommodations (for both the person with PPA and their companion) will be covered by the study.

Contact: Christina Coventry, MS, RN | 312-908-9681, christina.coventry@northwestern.edu

Recruiting from December 2017 to February 2023

The Communication Bridge study is an internet-based, speech therapy intervention for individuals with Primary Progressive Aphasia and their Communication Partner. A Communication Partner can be a spouse, relative, or close friend that will participate in the study along with the individual with PPA. The goal of the study is to understand how speech-language therapy affects communication abilities in people living with PPA. All study visits take place over the Internet in your home. We will provide you with a computer for the length of the study. We will connect through a video-chat program on the computer. The study lasts about one year. You will complete 5 evaluations with a licensed speech-language therapist, 15 one-hour speech therapy sessions with a licensed speech-language therapist, and home exercises on the computer we provide you. This study accepts persons all over the country, but they do have to be in the mild impairment stage of PPA, have a communication partner willing to participate, and sufficient internet connection.

Additional information from AFTD:
Participants with a diagnosis of PPA and their actively engaged care partners will be involved in the study for 12 months. There are no costs to participate in this study and compensation will be provided.

Contact: Libby Rogers, M.A. | 312-503-4012, libbyrogers@northwestern.edu
Contact: Emily Rogalski, Ph.D. | 312-503-1155, e-rogalski@northwestern.edu

Learn more at clinicaltrials.gov.