TRIAL READY (Clinical Trial Readiness)

Amyotrophic Lateral Sclerosis Clinical Trial

Official title:

TRIAL READY (Clinical Trial Readiness)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03912987
• Other study ID #: 20180908
• Secondary ID: U01NS107027
• Status: Active, not recruiting
• Start date: January 22, 2019
• Est. completion date: June 2025

Study information

• Verified date: May 2024
• Source: University of Miami
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 610
• Est. completion date: June 2025
• Est. primary completion date: June 2025
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Member of at least one of the following categories:

1. Individuals with a clinical diagnosis of ALS or a related disorder, including FTD, ALS-FTD, PLS, and PMA.

2. 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.

Related Conditions & MeSH terms

• ALS-Frontotemporal Dementia
• Amyotrophic Lateral Sclerosis
• Aphasia, Primary Progressive
• Dementia
• Frontotemporal Dementia
• Motor Neuron Disease
• Muscular Atrophy
• Muscular Atrophy, Spinal
• Pick Disease of the Brain
• Primary Lateral Sclerosis
• Progressive Muscular Atrophy
• Sclerosis

Locations

Country	Name	City	State
United States	University of Kansas	Kansas City	Kansas
United States	University of Miami	Miami	Florida
United States	University of Pennsylvania	Philadelphia	Pennsylvania

Sponsors (3)

Lead Sponsor	Collaborator
University of Miami	National Institute of Neurological Disorders and Stroke (NINDS), National Institutes of Health (NIH)

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Longitudinal trajectories (and variability) of leading biological-fluid biomarker candidates.	In a population of patients with ALS and related disorders who would meet typical clinical trial eligibility, this study aims to define the natural history (and variability) of urinary neurotrophin receptor extracellular domain (p75ECD); blood and cerebrospinal fluid (CSF) neurofilament light (NfL) and phosphorylated neurofilament heavy (pNfH); and among patients with the C9orf72 hexanucleotide repeat expansion mutation (HREM), CSF and peripheral blood mononuclear cell (PBMC) poly(GP).	12 months
Secondary	Prognostic utility of leading biological-fluid biomarker candidates.	In a population of patients with ALS and related disorders who would meet typical clinical trial eligibility, quantify the prognostic value that baseline biomarker levels add to readily available clinical factors that are known to predict prognosis.	12 months