Phenotype, Genotype & Biomarkers in ALS and Related Disorders

Study Purpose:

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

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Michael Benatar, University of Miami

Clinicaltrials.gov ID (11 digit #):

NCT02327845

Neals Affiliated?

No

Coordinating Center Contact Information


Michael Benatar, MBChB, MS, DPhil; Stacy S. Merritt, MA, CCRP / .(JavaScript must be enabled to view this email address) / 305-243-4015; 305-243-6554
.(JavaScript must be enabled to view this email address) United States

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

Participant Duration:

Estimated Enrollment:

700

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
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    N/A

    Maximum Age:

    N/A

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?


    Study Population
    Patients with ALS or a related neurodegenerative disorder, including FTD, HSP, PLS, PMA and MSP. Select family members of affected participants.
    Criteria

    Inclusion Criteria:
    -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.

    Exclusion Criteria:
    -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

    California Pacific Medical Center (CPMC)
    San Francisco, California 94115
    United States

    University of California San Diego (UCSD)
    San Diego, California 92093
    United States

    University of Miami
    Miami, Florida 33139
    United States

    Mayo Clinic, Jacksonville
    Jacksonville, Florida 32224
    United States

    University of Iowa
    Iowa City, Iowa 52242
    United States

    Kansas University Medical Center (KUMC)
    Kansas City, Kansas 66160
    United States

    Wake Forest University
    Winston-Salem, North Carolina 27157
    United States

    Cleveland Clinic
    Cleveland, Ohio 44195
    United States

    University of Texas Health Science Center San Antonio (UTHSCSA)
    San Antonio, Texas 78229
    United States

    University of Texas Southwestern (UTSW)
    Dallas, Texas 75390
    United States

    University of Virginia (UVA)
    Charlottesville, Virginia 22908
    United States

    Eberhard Karls University of Tubingen
    Tubingen
    Germany