Phenotype, Genotype & Biomarkers in ALS and Related Disorders
The goals of this study are: (1) to better understand the relationship between the phenotype and genotype of amyotrophic lateral sclerosis (ALS) and related diseases, including primary lateral sclerosis (PLS), hereditary spastic paraplegia (HSP), progressive muscular atrophy (PMA), and frontotemporal dementia (FTD); and (2) to develop biomarkers that might be useful in aiding therapy development for this group of disorders.
Disease:Amyotrophic Lateral Sclerosis (ALS), Familial ALS, Sporadic ALS, Primary Lateral Sclerosis (PLS), Hereditary Spastic Paraplegia (HSP), Healthy Volunteer, Healthy Volunteer with a Family History of ALS
Study Type:Observational Study
Study Category:Diseases, Study Type, Study Status, Phase, Gender
Study Status:Active, currently recruiting
Study Chair(s)/Principal Investigator(s):
Michael Benatar, University of Miami
Clinicaltrials.gov ID (11 digit #):NCT02327845
Coordinating Center Contact Information
Full Study Summary:
This study will recruit patients with ALS, ALS-FTD, PLS, HSP, and PMA, with a focus on incident cases. Patients with both familial and sporadic forms of these diseases will be enrolled and followed longitudinally using a standardized set of evaluations. Biological samples (blood, urine, CSF) will be collected from all study participants, and will be used for biomarker discovery and validation. Family members of affected individuals may also be enrolled and asked to contribute DNA and biological samples to aid genetic and biomarker discovery.
Study Sponsor:University of Miami National Institute of Neurological Disorders and Stroke (NINDS) National Center for Advancing Translational Science (NCATS) St. Jude Children's Research Hospital
Estimated Study Start Date:03/31/2015
Estimated Study Completion Date:07/31/2019
Posting Last Modified Date:06/30/2016
Date Study Added to alsconsortium.org:08/18/2015
- More Information
Time since Symptom Onset:
Time since Diagnosis:
Can participants use Riluzole?
Patients with ALS or a related neurodegenerative disorder, including FTD, HSP, PLS, PMA and MSP. Select family members of affected participants.
-Member of at least one of the following categories:
a. Individuals with a clinical diagnosis of ALS or a related disorder, including FTD, HSP, PLS, PMA and MSP (sporadic or familial).
b, Family member of an enrolled affected individual.
-Able and willing to comply with relevant procedures.
-Affected with end or late stage disease.
-A condition or situation which, in the PI's opinion, could confound the study finding or may interfere significantly with the individual's participation and compliance with the study protocol. This includes (but is not limited to) neurological, psychological and/or medical conditions.
Site Contact Information
Kansas University Medical Center (KUMC)
Kansas City, Kansas 66160
University of Miami
Miami, Florida 33139
Mayo Clinic, Jacksonville
Jacksonville, Florida 32224
University of California San Diego (UCSD)
San Diego, California 92093
University of Virginia (UVA)
Charlottesville, Virginia 22908
University of Texas Health Science Center San Antonio (UTHSCSA)
San Antonio, Texas 78229
University of Texas Southwestern (UTSW)
Dallas, Texas 75390
Cleveland, Ohio 44195
Wake Forest University
Winston-Salem, North Carolina 27157
University of Iowa
Iowa City, Iowa 52242
California Pacific Medical Center (CPMC)
San Francisco, California 94115
Eberhard Karls University of Tubingen