Recruiting Studies with a Genetic ALS or FTD Focus
We strive to keep these up to date but know there will be a lag when changes are made to recruiting status. Please double check before assuming a study is still open or not open. If you have feedback contact us at info@endthelegacy.org
Looking for broader ALS and FTD studies?
Observational Studies for At Risk Genetic ALS & FTD Families - US
Study
ALS/FTD
Gene(s)
Participation Criteria
Remote
Location
Website
ALS Families Project
ALS / FTD
Known ALS/FTD-associated genes or strong familial risk
Unaffected relatives and asymptomatic carriers from families affected by genetic ALS/FTD
Columbia University, New York City
No
ALLFTD
FTD / ALS-FTD
C9orf72; GRN; MAPT; TBK1; other familial FTLD
People with FTD-spectrum disorders, people from families with genetic FTD, and eligible controls
United States and Canada
No
Pre-fALS (Pre-symptomatic Familial ALS)
ALS
Multiple familial ALS genes
People without ALS symptoms who have a family history of ALS and may be at genetic risk
University of Miami; participants may live in the U.S. or Canada
Partial
PICALS (Pathogenesis in C9ALS)
ALS / FTD
C9orf72
People with C9orf72-associated ALS, healthy blood relatives of people with C9-ALS, and selected comparison participants
Emory University, Atlanta, Georgia
No
ALS Research Collaborative (ARC)
ALS
Any ALS-associated gene; genetic status not required for diagnosed participants
People diagnosed with ALS and asymptomatic carriers of a confirmed ALS-associated mutation
Worldwide; written English required
Yes
UNICORN: UPenn Observational Research Repository on Neurodegenerative Disease
ALS / FTD and related disorders
Familial ALS/FTD; specific gene not required
People with neurodegenerative disease, people with a family history who may or may not be symptomatic or mutation carriers, and controls
Philadelphia, Pennsylvania
No
Longitudinal Assessment of the Gut Microbiome in People with ALS
ALS
Any ALS-associated gene
People with ALS, asymptomatic ALS gene carriers, and healthy volunteers
Mass General / Boston and remote participation
Yes
Clinical Manifestations and Biomarkers in ALS Type 4
ALS4 and related inherited disorders
SETX; selected RNA-processing disorders
People with genetically confirmed ALS4 or related disorders, eligible relatives, and controls
NIH Clinical Center, Bethesda, Maryland
No
EverythingALS Gene Carrier Study
ALS / FTD
ALS- or FTD-linked variants; family members may also inquire
Asymptomatic gene carriers, people living with ALS, and participating family members
Remote / worldwide participation subject to study criteria
Yes
Mayo Jacksonville Study
ALS
Unknown
Unknown
Jacksonville, Florida
Unknown
Observational Studies for At Risk Genetic ALS & FTD Families - Outside of the US
Study
ALS/FTD
Gene(s)
Participation Criteria
Remote
Location
Website
ALS Research Collaborative (ARC)
ALS
Any ALS-associated gene; genetic status not required for diagnosed participants
People diagnosed with ALS and asymptomatic carriers of a confirmed ALS-associated mutation
Worldwide; written English required
Yes
GENFI (Genetic Frontotemporal Dementia Initiative)
FTD
C9orf72; GRN; MAPT
People from families affected by genetic FTD, including presymptomatic and symptomatic participants
Research centers across Europe and Canada
No
ACORN: A C9orf72 National Study
ALS / FTD
C9orf72
People with C9orf72-associated MND/ALS or FTD, at-risk family members, and healthy comparison participants
United Kingdom
No
NeuroPrems
ALS / FTD and other neurodegenerative diseases
C9orf72; GRN among included genetic groups
Presymptomatic participants in genetic neurodegenerative-disease families
Paris Brain Institute, France
No
premodiALS
ALS
Multiple ALS-associated genes
Presymptomatic gene-mutation carriers, people with very early ALS, selected disease controls and healthy controls
Multiple European centers and Turkiye
No
Gene-Specific Treatment Opportunities
These are for individuals who are diagnosed/symptomatic
Study
ALS/FTD
Gene(s)
Study Type
Location
Website
Silence ALS
ALS
ANXA11, CHCHD10, KIF5A, PFN1, SETX, TARDBP, VAPB, VCP, more
US
Personalized ASO-development program based on exact variant
ALN-SOD
ALS
SOD1
Australia, Belgium, Canada, Germany, Japan, Poland, South Korea, Sweden and Taiwan
Phase 1/2
We especially encourage you to consider enrolling in research at centers where At Risk Care is prioritized. See those centers here.
