Summary:
AL001 is an experimental treatment designed to slow the progression of Frontotemporal Dementia by elevating progranulin levels in the brain. This phase 3 trial is open to both symptomatic and asymptomatic individuals with progranulin (GRN) genetic variants. Participants in this study will be randomly assigned to receive either the experimental treatment or a placebo (something that looks like the experimental treatment but has no experimental medicine in it). It is designed to be ‘double blind’ which means that participants will not know if they received AL001 or a placebo and the study doctor will not know either. Only the researchers analyzing the data will have that information. The study will collect data to evaluate how safe AL001 is, as well as how effective it is at slowing disease progression. This study is closed to enrollment after meeting its target enrollment.
Who is eligible?
- Men and women aged 25 to 85 years
- Carriers of a progranulin (GRN) pathogenic (disease causing) genetic variant
- At risk of developing FTD symptoms OR symptomatic and meeting one or more of the criteria for the diagnosis of possible behavioral variant FTD, or a diagnosis of Primary Progressive Aphasia
- Has a study partner who is with the participant every day for a total of at least 5 hours per week
- Does not have dementia due to a condition other than FTD
- Does not have a history of severe allergic reaction to chimeric, human, or humanized antibodies or fusion proteins
- Does not have uncontrolled chronic medical condition such as high blood pressure, diabetes, thyroid disease, heart disease, liver disease or kidney disease.
- Is not currently pregnant, breastfeeding or planning to become pregnant
- Does not currently use blood thinners
- Does not have a history of cancer (within the last 5 years)
- Does not live in a nursing home or require continuous nursing care
- Has not participated in other experimental vaccine or gene therapy
Interested individuals meeting the above requirements should contact the study coordinator for additional requirements and exclusions if interested in participating.
What will happen during the study?
This study will randomly assign participants to receive the experimental treatment or the placebo group where participants will receive a placebo (something that looks like the experimental treatment but has no medicine in it). Both the experimental treatment and placebo are IV medications, which are given through a needle in the arm every 4 weeks.
What are they measuring to evaluate the experimental treatment?
- Changes in a clinical rating scale called the CDR plus NACC FTLD, which stands for “Clinical Dementia Rating (CDR)+ National Alzheimer's Coordinating Center (NACC) frontotemporal lobar degeneration (FTLD)”
- Changes in additional cognitive, behavioral and activities of daily living assessments
- Changes biomarkers measured through blood samples and spinal fluid
- Any undesired and/or unexpected effects of the experimental treatment (adverse events)
How long will participants be involved in the study?
Study participants will be followed for up to 96 weeks.
