The aim of the study is to understand more about genetic FTD, particularly in those who have mutations in the progranulin (GRN), microtubule-associated protein tau (MAPT) and chromosome 9 open reading frame 72 (C9ORF72) genes. There are now promising avenues for treatment of these disorders but we still do not know when drugs should be started or how we should measure the response to treatment.
GENFI investigates both people who have developed symptoms and also people who have a risk of developing symptoms in the future because they carry an abnormal genetic mutation. By studying these individuals who are destined to develop the disease later in life we can understand the development from the very earliest changes. The key objectives of GENFI are therefore to develop markers which help identify the disease at its earliest stage as well as markers that allow the progression of the disease to be tracked.
The eventual aim will be to use these markers in future clinical trials of drugs in genetic FTD.
The purpose of this study is to assess the safety, tolerability and effects on behaviour of Syntocinon given intranasally (by a spray into the nostrils) compared to placebo (an inactive saline substance that contains no medication) in participants with frontotemporal dementia/Pick’s disease. This study will take place in approximately 15 centres across Canada and the United States. Approximately 112 patients in total will be enrolled in this study. In the first phase we will examine which of three different dosing schedules of oxytocin may be more effective. In the second phase of the study, patients entering the study will be randomized to the oxytocin dosing schedule that appeared most effective in the first phase.
You (the research participant) are being asked to participate in a research study designed to assess individuals with different sorts of cognitive and movement changes seen in older adults. We (the study sponsor) will look at the usefulness of imaging studies, clinical assessments and biomarker tests (blood, saliva, urine, cerebrospinal fluid (optional), and feces (optional)) together with measurements of memory, thinking and daily functioning, for distinguishing these changes from each other and from healthy aging. You have been asked to participate because there are concerns about your memory, thinking or speaking ability, your behaviour, abnormal movements or problems walking, or because you have none of these concerns or problems and can be part of a healthy aging comparison group.
This is a clinical trial of WVE-004, a new drug under development for individuals with either ALS or FTD associated with an expansion in the C9orf72 gene. Clinical trials gather evidence about the safety and effectiveness of new drugs. Right now, WVE-004 can only be used in clinical trials, because it has not been proven safe or effective enough to be used in regular medical practice.
This study is what we call a “first-in-human” study. In a first-in-human study, the intervention has never been tested on humans before. This study is allowed to be conducted because the information that has been obtained through conducting tests in laboratories and animals suggests that the intervention could be safe for humans. However, there is no proof yet that it is safe for humans to receive this intervention. A “first-in-human” study aims at proving that humans can be exposed to the intervention without suffering from side effects that are too severe; a first-in-human study does not aim at demonstrating effectiveness of the intervention.
The purpose of this study is to determine if WVE-004 is safe and well tolerated in patients with ALS or FTD when compared with a placebo control.
Active Trials (not recruiting)
The purpose is to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of IONIS-MAPTRx in patients with Mild Alzheimer’s Disease
This is a clinical trial of aducanumab, a drug under development for Alzheimer’s disease. Clinical trials gather evidence about the safety and effectiveness of new drugs. Right now, aducanumab can only be used in clinical trials, because it has not been proven safe or effective enough to be used in regular medical practice. However, it has already been tested in humans to see how safe it is and whether it works. Health Canada does not object to its use in clinical trials.
The purpose of this study is to evaluate the safety and tolerability of high dose aducanumab for a longer period.