TRIAL READY (Clinical Trial Readiness)

Study Purpose:

This study, being conducted under the auspice of the CReATe Consortium, will enroll patients with ALS and related disorders as well as healthy controls, with the goal of facilitating clinical validation of leading biological-fluid based biomarker candidates that may aid therapy development for patients with ALS and related disorders.
 

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS, Primary Lateral Sclerosis (PLS), Healthy Volunteer

Study Type:

Observational Study

Study Category:

Study Status:

Enrolling

Phase:

Not Applicable

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

Michael Benatar, MBChB, MS, DPhil University of Miami

Clinicaltrials.gov ID (11 digit #):

NCT03912987

Neals Affiliated?

No

Coordinating Center Contact Information

University of Miami
Anne Cooley / .(JavaScript must be enabled to view this email address) / 844-837-1031
.(JavaScript must be enabled to view this email address) Miami, Florida 33136 United States

Full Study Summary:

This multi-center study aims to clinically validate leading biological-fluid-based biomarker candidates as potential prognostic and pharmacodynamic biomarkers that have the potential to facilitate therapy development for patients with ALS and related disorders. Biomarker candidates that will be considered include: urinary p75 neurotrophin receptor extracellular domain (p75ECD), blood and cerebrospinal fluid (CSF) phosphorylated neurofilament heavy (pNfH), blood and CSF neurofilament light (NfL) and, in the population with a C9orf72 hexanucleotide repeat expansion, peripheral blood mononuclear cell (PBMC) and CSF levels of the dipeptide repeat protein poly(GP). In pursuit of these goals, the CReATe Consortium is already collecting longitudinal biological samples from patients with ALS and related disorders through the ongoing Phenotype-Genotype-Biomarker (PGB) study. TRIAL READY aims to identify additional patients with the C9orf72 hexanucleotide repeat expansion mutation (HREM), the most common genetic cause of ALS, who may be further followed through the PGB study. This study will also enroll and longitudinally evaluate a cohort of age- and gender-match healthy controls.

Study Sponsor:

University of Miami

Participant Duration:

Estimated Enrollment:

610

Estimated Study Start Date:

01/22/2019

Estimated Study Completion Date:

01/31/2022

Posting Last Modified Date:

05/21/2019

Date Study Added to alsconsortium.org:

05/21/2019
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Study Population
    Patients with ALS or a related neurodegenerative disorder, including FTD, ALS-FTD, PLS and PMA. Individuals never diagnosed with and not at particular risk of developing ALS or a related disorder.
    Criteria

    Inclusion Criteria:

    Member of at least one of the following categories:

    • Individuals with a clinical diagnosis of ALS or a related disorder, including FTD, ALS-FTD, PLS, and PMA.
    • Individuals never diagnosed with and not at particular risk of developing ALS or a related disorder.

    Able and willing to comply with relevant procedures.

    Exclusion Criteria:

    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

    University of Miami
    Kristina Reyes / .(JavaScript must be enabled to view this email address) / 305-243-4997
    Miami, Florida 33136
    United States