Clinical Trials for Frontotemporal Dementia
There are currently 4 ongoing clinical trials investigating new treatments for frontotemporal dementia, particularly for patients with specific genetic mutations in the progranulin gene. These studies are testing different approaches including gene therapy and antibody treatments across multiple European countries.
Clinical trial locations
- Belgium
- Study on AVB-101 for Patients with Frontotemporal Dementia with Progranulin Mutations
- Study on the Safety and Effects of DNL593 for Patients with Frontotemporal Dementia
- Study on the Safety and Effects of LY3884963 for Patients with Fronto-Temporal Dementia with Progranulin Mutations
- Study on the Effects of Latozinemab for Patients with Frontotemporal Dementia Due to Progranulin Gene Mutations
- Czechia
- France
- Study on the Safety and Effects of DNL593 for Patients with Frontotemporal Dementia
- Study on the Safety and Effects of LY3884963 for Patients with Fronto-Temporal Dementia with Progranulin Mutations
- Study on the Effects of Latozinemab for Patients with Frontotemporal Dementia Due to Progranulin Gene Mutations
- Germany
- Greece
- Italy
- Netherlands
- Poland
- Portugal
- Spain
- Sweden
Study on AVB-101 for Patients with Frontotemporal Dementia with Progranulin Mutations
This study is evaluating a gene therapy treatment called AVB-101 for people who have frontotemporal dementia caused by mutations in the progranulin gene. The therapy aims to increase levels of progranulin protein in the brain, which is important for brain health.
Who can participate: This trial is open to men and women between 30 and 75 years old who have a confirmed progranulin gene mutation and meet specific dementia criteria. Participants must have a study partner who can attend visits and provide information about their condition. Women who can have children must use effective birth control and have a negative pregnancy test before treatment. The thalamus, a part of the brain, must be of a certain size as measured on an MRI scan.
Who cannot participate: People without a confirmed diagnosis of this specific type of dementia cannot join. Those with certain other medical conditions or taking medications that could interfere with the study may also be excluded. Individuals who are part of vulnerable populations, such as those with severe cognitive impairments that prevent them from understanding the study requirements, may not be eligible.
What the study involves: AVB-101 is given as a one-time treatment delivered directly into a specific area of the brain through a procedure called bilateral intrathalamic infusion. After treatment, participants are closely monitored for 26 weeks, with health checks including vital signs, neurological examinations, and laboratory tests. The study then continues with follow-up visits for up to five years to monitor safety and measure changes in progranulin levels in blood and cerebrospinal fluid.
Study medication: AVB-101 is an experimental gene therapy that uses a viral vector to deliver the progranulin gene to brain cells. This is the first time this treatment is being tested in people, making it a Phase 1/2 clinical trial.
Study on the Safety and Effects of DNL593 for Patients with Frontotemporal Dementia
This trial is testing a new medication called DNL593 in people with frontotemporal dementia who have mutations in the progranulin gene. The study aims to understand how safe the medication is and how it affects the disease.
Who can participate: The study has three parts. Part A involves healthy volunteers between 18 and 55 years old. Part B is for people with the condition who are between 18 and 80 years old, have a confirmed progranulin gene mutation, and a dementia score of 0.5 or higher. Part C is an extension for people who completed Part B without serious side effects. All participants must have a body mass index between 18 and 32 and agree to use effective birth control if they can have children.
Who cannot participate: People with medical conditions other than frontotemporal dementia, serious health issues, recent participation in other trials, allergies to the study medication, history of substance abuse, or who are pregnant or breastfeeding cannot participate.
What the study involves: Part A tests a single dose of DNL593 in healthy people. Part B involves multiple doses given both orally and through an intravenous infusion to people with the condition. Part C allows participants to continue receiving the medication for up to 18 months. Throughout the study, participants undergo regular health monitoring, including laboratory tests, vital sign checks, and physical examinations.
Study medication: DNL593 is administered as an injection and targets specific proteins in the brain believed to be involved in the progression of frontotemporal dementia. It is still in the investigational phase.
Study on the Safety and Effects of LY3884963 for Patients with Fronto-Temporal Dementia with Progranulin Mutations
This study is testing an investigational gene therapy called LY3884963 in people with frontotemporal dementia caused by progranulin gene mutations. The treatment aims to increase progranulin protein levels in the brain.
Who can participate: Men and women between 30 and 85 years old with a confirmed progranulin gene mutation can participate. They must have a reliable study partner who spends at least five hours per week with them and can provide information about their behavior and abilities. Participants should be able to walk without needing a walker or wheelchair and live in the community rather than a nursing home. They must have received vaccines for pneumococcal pneumonia and shingles within the past 10 years, weigh between 88 and 242 pounds, and have a BMI between 18 and 34. Both participants and their partners must agree to use effective birth control if they can have children.
Who cannot participate: People who do not have this specific type of dementia, those with other health conditions that could interfere with the study, individuals who are pregnant or breastfeeding, or those who are part of vulnerable populations unable to consent cannot participate.
What the study involves: LY3884963 is delivered through a single injection into a specific area at the base of the skull called the cisterna magna. The study evaluates three different dose levels. Some participants may also receive additional medications like prednisone, sirolimus, or methylprednisolone to help manage their condition. Participants are monitored regularly with blood tests, cerebrospinal fluid samples, and imaging studies to track progranulin levels and any changes in their health.
Study medication: LY3884963 is an investigational gene therapy that uses a viral vector to deliver the progranulin gene. It is still in the research phase and aims to modify the underlying genetic cause of the disease.
Study on the Effects of Latozinemab for Patients with Frontotemporal Dementia Due to Progranulin Gene Mutations
This trial is evaluating latozinemab, also known as AL001, for people who are at risk of or already have frontotemporal dementia caused by mutations in the progranulin gene. The study aims to determine if this treatment can improve symptoms or slow disease progression.
Who can participate: Men and women between 25 and 85 years old with a confirmed progranulin gene mutation can join. Participants must have a study partner who knows them well, spends at least five hours per week with them, and can attend certain visits to provide information about their condition. Women must not be pregnant or breastfeeding and must use effective birth control during the study. Men must also use birth control and agree not to donate sperm. All participants must agree not to donate blood during the study and for one year after the final dose.
Who cannot participate: People with other types of dementia, those who have had recent major surgery or are planning surgery during the study, individuals with severe allergic reactions to medications, those currently in other clinical trials, people with unstable or serious medical conditions, pregnant or breastfeeding women, individuals with recent drug or alcohol abuse, and those with mental health conditions that could interfere with the study cannot participate.
What the study involves: This is a double-blind study, meaning neither participants nor researchers know who receives the actual medication versus a placebo. Latozinemab is given through an intravenous infusion directly into the vein. Participants undergo regular assessments throughout the study to monitor their cognitive and functional abilities. The main goal is to measure changes from the start of the study at weeks 48, 72, and 96. The study is expected to continue until 2026.
Study medication: Latozinemab (AL001) is currently being studied in a Phase 3 clinical trial. It is designed to target and modify certain biological pathways associated with frontotemporal dementia. The medication is still investigational and not yet approved for general medical use.
Summary
All four clinical trials are focused on frontotemporal dementia related to progranulin gene mutations, highlighting the importance of genetic factors in this form of dementia. Three of the studies test different approaches to directly increase progranulin levels through gene therapy (AVB-101, LY3884963) or antibody treatment (latozinemab), while DNL593 takes a different molecular approach.
Belgium, the Netherlands, Spain, and Italy have the most trials available, with participants in these countries having access to all or most of the ongoing studies. France also hosts three of the four trials. This concentration of research in Western Europe reflects the availability of specialized medical centers and expertise in treating rare forms of dementia.
All trials require participants to have a study partner who can provide information and attend visits, recognizing the progressive nature of the condition and the need for reliable support throughout the research process. The age ranges vary slightly between studies, but most accept participants between their 30s and 80s, reflecting the typical age of onset for this genetic form of dementia.
These studies represent important steps in developing treatments for a condition that currently has no cure. Each uses a different strategy to address the underlying genetic cause, potentially offering hope for better treatment options in the future.
