Participating in research can bring new ways to help families today, and build hope for a future without this disease. You may choose to find out more about the following featured studies:
A Study of a Gene Therapy for Frontotemporal Degeneration Due to Progranulin Mutations (PROCLAIM)
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 anaesthesia 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.
Prevail Therapeutics │ 917-336-9310 │ [email protected]
Study site contact:
Jessica Garaycoa │ 689-216-3100 │ [email protected]
A Study Collecting Speech Samples for a Research Dataset
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.
A Study of In-Home Technology for Dementia Caregivers
Recruiting from February 2019 to February 2021
UC Berkeley researchers are evaluating an in-home technology system designed to reduce worry and loneliness in dementia caregivers. Eligible caregivers must enroll by end of October 2019, live in Los Angeles, Orange County, the Bay Area or Las Vegas, live with a person diagnosed with dementia, be fluent/literate in English, own an iPhone, and have in-home wireless internet. All technology is provided and installed for free, including various sensors and an iPad. The system alerts caregivers to potential risks like falls, episodes of wandering, and home alerts like water leaks or forgotten stove-tops. The system also includes a “Social Connector” app keeping caregivers connected to their friends and family. Participation includes completing four online questionnaires throughout the nine-month study. Caregivers will be paid $175 for completing all questionnaires and are able to keep the technology.
A Phase 2 Clinical Trial of Intranasal Oxytocin for Frontotemporal Dementia
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 ([email protected]).
Rehabilitation of Speech and Language in Primary Progressive Aphasia (PPA)
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.
Recruiting from June 2018 to December 2020
The TANGLES research study is being conducted at Washington University School of Medicine in St. Louis. The purpose of this study is to examine how the tau protein is made, transported and cleared in the human body. Tau forms tangles in the brain found in several neurological conditions.
Understanding of these processes is important in designing treatments and diagnostic tools to target this protein.
You may be eligible to participate if you are:
- Diagnosed with progressive supranuclear palsy (PSP), corticobasal degeneration (CBD), or have a family history of FTD with the MAPT gene mutation
- Not taking anticoagulant medications
- Are at least 18 years old
- Able to take food and drink by mouth safely
Study participation lasts for 4 months and includes:
- 6 study visits (including 1 overnight visit)
- Intravenous (IV) infusion and blood draws
- Lumbar punctures (spinal taps)
Participants will be reimbursed up to $1,200 for time and effort and travel expenses will be covered for the participant and family member.
Additional information from AFTD:
Each participant, accompanied by a care partner, will make six visits to the study site (Washington University in St. Louis) over the four-month study period. The first visit will require an overnight stay and will involve an intravenous infusion and blood draws. During each of the five subsequent visits, participants will undergo a lumbar puncture and have blood drawn.
Travel expenses for all visits, including flights, hotels, and ground transportation, will be covered for both the participant and care partner. The study team can assist participants with travel arrangements or transport needs such as wheelchairs.
Contact: Melody Li | 314-273-6062, [email protected]
Language in Primary Progressive Aphasia
Recruiting from September 2007 to April 2022
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, [email protected]
Communication Bridge: A Person-Centered Internet-Based Intervention for Individuals With Primary Progressive Aphasia
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.
Learn more at clinicaltrials.gov.
Other recent studies of note
Low-Dose Lithium for the Treatment of Behavioral Symptoms of FTD
Recruiting from August 2016 to September 2020
A study sponsored by Columbia University in collaboration with the Alzheimer’s Drug Discovery Foundation is seeking persons diagnosed with behavioral variant FTD, semantic variant primary progressive aphasia (PPA), or agrammatic, non-fluent PPA with behavioral symptoms, for a study to test the use of a drug to treat behavioral symptoms in FTD. The study, which will run over approximately 12 weeks, is recruiting volunteer participants at the Irving Institute at Columbia University Medical Center. A care partner (family member) will be required to provide information about the patient during interviews. The study will cover transportation costs as well as an additional stipend per participant. For more information, contact Hannah Silverman at: [email protected] or by calling 212-305-6284.
Clinical trials and studies aren’t the only way to participate in research! You may also wish to consider:
- The FTD Disorders Registry features ways to participate in research, to spur innovation that will lead to effective treatments and therapies.
- ALLFTD Study – ARTFL-LEFFTDS Longitudinal Frontotemporal Lobar Degeneration (ALLFTD) is a comprehensive natural history study that targets the progression and treatment of FTD, open to anyone with an FTD disorder and to any member of a family with a history that suggests FTD. Funded by the National Institutes of Health (NIH), ALLFTD combines the research efforts of the existing Advancing Research and Treatment of Frontotemporal Lobar Degeneration (ARTFL) and Longitudinal Evaluation of Familial Frontotemporal Dementia Subjects (LEFFTDS) studies to form a multi-side consortium that allows researchers to work collaboratively to advance FTD knowledge for future clinical trials. ALLFTD is now actively recruiting participants at certain study sites, depending on local conditions. To ensure safety during the COVID-19 pandemic, the project is recruiting and seeing participants using a mix of in-person and virtual telemedicine appointments. You can visit the ALLFTD website to find the study site closet to you and contact the site coordinator for more information.
PLEASE NOTE: AFTD is under no obligation to publicize any study, and may turn down such a request at any time. AFTD at no time “endorses” or “approves” any study, whether or not it chooses to publicize it.