A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 1b/2a Study of WVE-004 Administered Intrathecally to Patients with C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD)

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.

From clinicaltrials.gov:

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.

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From genfi.org.uk/:

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.

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Improving Neurological Health in Aging Via Neuroplasticity-based Computerized Exercise

Researchers from McGill University will ask you to complete a computerized brain training program at home for 30 mins a day for 10 weeks. You will also be asked to visit our office in Montreal, Quebec to receive a complete overview of the study, cognitive assessments and brain imaging (PET, MRI) before and after your training program. You can be reimbursed up to $211 CAD for your participation.

Who do we need?

1. Participants who are 65 or older without dementia
2. Speak fluent French or English
3. Can access the internet
4. Can receive MRI and PET scans
5. Can use a brain training program on a study provided tablet