Trials / Active Not Recruiting

Active Not RecruitingNCT03912987

TRIAL READY (Clinical Trial Readiness)

Status: Active Not Recruiting
Phase: —
Study type: Observational
Enrollment: 610 (estimated)

Sponsor: University of Miami · Academic / Other
Sex: All
Age: 18 Years
Healthy volunteers: Accepted

Summary

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.

Detailed 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 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.

Conditions

Timeline

Start date: 2019-01-22
Primary completion: 2026-06-01
Completion: 2026-06-01
First posted: 2019-04-12
Last updated: 2025-10-20

Locations

3 sites across 1 country: United States

Source: ClinicalTrials.gov record NCT03912987. Inclusion in this directory is not an endorsement.