Genomic Translation for Amyotrophic Lateral Sclerosis Care
GTAC
1 other identifier
observational
254
2 countries
14
Brief Summary
The purpose of this study is to look for abnormal genes and gene expression profiles that help determine why a person develops amyotrophic lateral sclerosis (ALS) and related motor neuron diseases (MND) and why their symptoms present and progress with a particular pattern.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Jun 2016
Longer than P75 for all trials
14 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 7, 2016
CompletedStudy Start
First participant enrolled
June 8, 2016
CompletedFirst Posted
Study publicly available on registry
June 10, 2016
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 27, 2021
CompletedStudy Completion
Last participant's last visit for all outcomes
June 29, 2022
CompletedAugust 19, 2022
August 1, 2022
5.1 years
June 7, 2016
August 18, 2022
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Correlation of DNA genotype with ALS phenotypes
Because subjects are followed over their entire disease course and undergo whole genome sequencing of their DNA, this project will study the distinct features (progression and particular symptoms) of subjects with and without mutations in already known ALS genes.
36 months
Correlation of gene expression in blood with ALS phenotypes
Because subjects are followed over their entire disease course and undergo gene expression profiling on their blood sample, this project will study the distinct features (progression and particular symptoms) of subjects with different types of gene expression profiles.
36 months
Eligibility Criteria
Patients with ALS (meeting El Escorial criteria for definite, probable or possible ALS), or primary lateral sclerosis or progressive bulbar/muscular atrophy.
You may qualify if:
- Study participants meeting all of the following criteria will be eligible for enrollment in GTAC:
- Men or women of any race or ethnicity aged 18 or older
- Diagnosis of familial or sporadic ALS (definite, probable, or possible according to El Escorial Criteria, Appendix 1), or those with primary lateral sclerosis or progressive bulbar/muscular atrophy forms of motor neuron disease. All-comers with ALS/MND should be enrolled without regard to familial vs sporadic or gene mutation status (i.e. participants with known gene mutations should still be enrolled), or phenotype.
- Capable of providing informed consent and following study procedures (in the case that a subject lacks the ability to provide informed consent, informed consent will be sought from the subject's surrogate representative).
- Willing to return to clinic site (or another participating center) for follow-up care.
You may not qualify if:
- Study participants meeting any of the following criteria during screening evaluation will be excluded from enrolling in GTAC:
- Invasive ventilation (i.e. tracheostomy) in place.
- Non-invasive ventilation dependent (defined as \>22 hours per day)
- Pregnancy.
- Known Human Immunodeficiency Virus (HIV) , chronic Hepatitis B, or Hepatitis C (because cells will be frozen down for future cell line generation).
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Columbia Universitylead
- ALS Associationcollaborator
- Biogencollaborator
Study Sites (14)
Cedar Sinai Medical Center
Los Angeles, California, 90048, United States
University of Colorado School of Medicine
Aurora, Colorado, 80045, United States
Univeristy of Michigan
Ann Arbor, Michigan, 48104, United States
University of Minnesota
Minneapolis, Minnesota, 55455, United States
Washington University
St Louis, Missouri, 63110, United States
Columbia University
New York, New York, 10032, United States
Duke University
Durham, North Carolina, 27705, United States
Oregon Health & Sciences University
Portland, Oregon, 97239, United States
Penn State College of Medicine
Hershey, Pennsylvania, 17033, United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15213, United States
Houston Methodist Neurological Institute
Houston, Texas, 77030, United States
University of Utah
Salt Lake City, Utah, 84112, United States
University of Washington
Seattle, Washington, 98195, United States
The University of Edinburgh
Edinburgh, United Kingdom
Biospecimen
DNA, RNA and Peripheral blood mononuclear cells (PBMCs)
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Matthew Harms, MD
Columbia University
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 7, 2016
First Posted
June 10, 2016
Study Start
June 8, 2016
Primary Completion
July 27, 2021
Study Completion
June 29, 2022
Last Updated
August 19, 2022
Record last verified: 2022-08
Data Sharing
- IPD Sharing
- Will share
- Time Frame
- At the end of the study.
- Access Criteria
- The investigators intend to share DNA, sequencing data, and phenotypic data with the NEALS repository and Biogen Idec. Coded genomic and clinical data will be shared with dbGaP and ASLGEN.
The investigators intend to share DNA, sequencing data, and phenotypic data broadly. Participants will be consented for sharing of GTAC data and samples with the Northeast ALS Consortium (NEALS) repository, with others carrying out ALS/MND research (including other consortia), for biomedical research in general (including use as controls and use by for-profit companies), and with Biogen Idec. Additionally, GTAC has plans to share coded genomic and clinical data with Database of Genotypes and Phenotypes (dbGaP) and a ALS/MND consortium for genomic discovery (ASLGEN). This includes sharing of genetic and phenotypic data with Biogen Idec, a pharmaceutical company that is partnering with GTAC to fund the sequencing of samples.