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.
Study Status:
Not recruiting
Disease:
Amyotrophic Lateral Sclerosis (ALS) , Familial ALS , Sporadic ALS , Primary Lateral Sclerosis (PLS) , Healthy Volunteer
Study Type:
Observational
Type of Intervention:
N/A
Intervention Name:
N/A
Placebo:
N/A
Phase:
N/A
Study Chair(s)/Principal Investigator(s):
Michael Benatar, MBChB, MS, DPhil University of Miami
Clinicaltrials.gov ID:
Neals Affiliated?
No
Coordinating Center Contact Information
University of Miami
Anne Cooley / email hidden; JavaScript is required / 844-837-1031
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
Estimated Enrollment:
610
Estimated Study Start Date:
01 / 22 / 2019
Estimated Study Completion Date:
01 / 31 / 2022
Posting Last Modified Date:
11 / 01 / 2021
Date Study Added to neals.org:
05 / 21 / 2019
Minimum Age:
18
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.
University of Miami
Jessica Hernandez / 305-243-2345 / email hidden; JavaScript is required
Miami, Florida
33136
United States
University of Kansas
Collin Gerringer / 913-574-0008 / email hidden; JavaScript is required
Kansas City, Kansas
66160
United States
University of Pennsylvania
Luis Rosario / 215-898-3081 / email hidden; JavaScript is required
Philadelphia, Pennsylvania
19107
United States