Frontotemporal dementia (FTD) is a progressive neurodegenerative disease that affects the front or certain areas of the brain, leading to behavioural changes and potential language and functional problems. Symptoms usually occur in a person’s fifties or sixties, and they often vary from one patient to another. However, one of the most common signs of FTD is behavioural changes in a person’s judgment and personality, which often makes it difficult for persons with FTD to interact appropriately with other people. Some people also develop problems with speech and language, called aphasia.
Familial FTD is very common and different types of mutations are associated with it. Especially mutation in the GRN gene is known to be associated with the development of this type of dementia. The presence of this mutation is associated with increased nerve cell death in the front and sides of the brain, causing the typical symptoms of FTD. A family history is seen in up to 40% of all FTD cases. Therefore, if a person is diagnosed with FTD and has a GRN mutation, relatives are at a higher risk of developing FTD as well. As no treatment currently exists that effectively slows down the progression of FTD patients who have the GRN mutation, new care options are being investigated.
A new potential treatment is being evaluated to decrease the progression of FTD in patients with a GRN mutation. The aim of this study is to assess the risk and benefit of a new treatment with the potential to slow down the progression of FTD in patients with a GRN gene mutation. If you or any of your loved ones are suffering from FTD or have a GRN mutation you might qualify for the study. If you qualify for participation in the study, you might receive a placebo instead of the study medication. A placebo is provided instead of the study medication and contains no active ingredients. The use of a placebo is standard practice in research studies investigating the effect of study medications and is used as a measure of comparison. Your chance of receiving a placebo will be explained by the study staff in advance of your participation.
Who can participate?
- Male and female between 25-85 years old.
- If you are diagnosed with frontotemporal dementia OR
- If you are carrier of the GRN mutation or have a relative who has FTD
Other criteria are also required before you may pre-qualify for participation in the study. To find out, please click on the link below and answer the digital questionnaire.
- New York
- La Jolla
- Chapel Hill
- San Antonio
- Box Hill
There will be no costs to you if you choose to participate in this research study. The study drug and any tests required by the study are provided by the study team, and you may receive reimbursement for travel related expenses incurred by taking part in the research study until your participation is complete.
The research study will assess the risk and benefit of the study drug in patients with FTD and GRN gene mutation. You will be provided with a further detailed overview of the study procedures by the study team before participation in the research study. There will be opportunities and time to discuss any questions before starting any study procedures.
The duration of study participation is 96 weeks in the four study periods described below:
A screening assessment period
During the screening visit at the study site, you will be told about the study and get answers to questions about the study. If you agree to participate, you will be asked to sign the informed consent document. If you agree to participate, you will undergo clinical and laboratory assessments that will determine if you are eligible for the study. This will include blood sampling, MRI scans, collection of vital parameters as blood pressure, body temperature etc.
A washout period
To participate in the study, some drugs used to treat frontotemporal dementia or other diseases will have to be terminated before you can receive the study drug. This process is called a washout period. The period can vary according to the type of drug you are currently using. The study team will provide you with detailed information about the length of your possible washout period.
If you are eligible to participate in the study, you will be randomly assigned to either study drug or placebo for 96 weeks. The study treatment will be administrated as an intravenous injection at the clinic. After 96 weeks, patients receiving the study drug can choose to continue on the treatment for further 96 weeks. The maximum length of exposure to the study drug in this study is up to 192 weeks. During this period, you will be in contact with the study team and have regular clinic visits to monitor your health.
After termination of the study treatment, you will enter a 10-week post-treatment follow-up period. During this period, you will visit the site 10 weeks after you have received your last study treatment. Your health will be closely monitored by study staff throughout your participation in the research study. You can withdraw your participation at any time without providing any reason.
In the figure below, you can see the maximum duration of the different periods of the research study.