TRIAL READY (Clinical Trial Readiness)
Last updated on July 2021Recruitment
- Recruitment Status
- Recruiting
- Estimated Enrollment
- Same as current
Summary
- Conditions
- ALS-Frontotemporal Dementia
- Amyotrophic Lateral Sclerosis
- Frontotemporal Dementia
- Primary Lateral Sclerosis
- Progressive Muscular Atrophy
- Type
- Observational
- Design
- Observational Model: CohortTime Perspective: Prospective
Participation Requirements
- Age
- Between 18 years and 125 years
- Gender
- Both males and females
Description
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 cons...
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.
Tracking Information
- NCT #
- NCT03912987
- Collaborators
- National Institutes of Health (NIH)
- National Institute of Neurological Disorders and Stroke (NINDS)
- Investigators
- Principal Investigator: Michael Benatar University of Miami