About this research
In order to participate in this study, you must be able to speak and read Dutch fluently.
A multicenter, open-label phase II study to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of AL001 in heterozygous carriers of C9orf72 mutations leading to frontotemporal dementia.
The aim of this study is to find out how safe the new substance AL001 is and how well the substance is tolerated (i.e. looking for possible side effects) when given to subjects who have the genetically confirmed mutation in the granulin or C9orf72. gene. The study drug is administered by IV.
The research is carried out in the Brain Research Center Amsterdam.
This research is commissioned by Alector.
This text has been directly translated from the Dutch trial description as approved by the Ethical Committee.
Screening - Selection
During a maximum period of six weeks, it will be determined whether you meet the study criteria and whether your general health allows participation in the study. If so, you can participate. These examinations take place in Amsterdam and include blood tests, physical examinations, memory tests, ECG questionnaires, MRI, and a spinal tap.
Each participant will receive AL001 (not a placebo/fake drug in this study). An IV treatment takes place every 4 weeks. In addition, appointments will be made with you when you should come to our center for check-ups (inquiries about side effects and medication use) and there will be physical and neurological examinations and neuropsychological examinations, blood tests, and ECGs and MRI scans will be made. The duration of treatment is 96 weeks.
After completion of the treatment phase, another visit is scheduled. During these visits, questionnaires and tests are taken for your memory, among other things. Furthermore, blood tests, physical examinations, an MRI scan, and/or a spinal tap will be performed.
Who can participate?
- You have a genetically confirmed C9orf72 mutation
- You are between 18 and 85 years old.
- You have someone close to you who can accompany you during the visits to the research center and answer questions about your health and functioning, among other things.