TRIAL READY (Clinical Trial Readiness)

NCT03912987 | Active Not Recruiting

• 2 other identifiers: 20180908U01NS107027
• Study Type: observational
• Target: 610
• Locations: 1 country
• Sites: 3

Brief 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.

Conditions

Amyotrophic Lateral Sclerosis; Frontotemporal Dementia; ALS-Frontotemporal Dementia; Primary Lateral Sclerosis; Progressive Muscular Atrophy

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• Prognostic utility of leading biological-fluid biomarker candidates.

  12 months

Study Arms (2)

Affected

Affected with ALS or a related disorder, including ALS-FTD, FTD, PLS, and PMA.

Healthy Controls

Those never diagnosed with and not at particular risk for developing ALS or a related disorder.

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

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.

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• University of Miami: lead
• National Institutes of Health (NIH): collaborator
• National Institute of Neurological Disorders and Stroke (NINDS): collaborator

Study Sites (3)

University of Miami

Miami, Florida, 33136, United States

Location

University of Kansas

Kansas City, Kansas, 66160, United States

Location

University of Pennsylvania

Philadelphia, Pennsylvania, 19107, United States

Location

Biospecimen

Retention: SAMPLES WITH DNA

DNA, serum, plasma, buffy coat, urine and CSF

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis; Frontotemporal Dementia; Motor Neuron Disease; Muscular Atrophy, Spinal

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases; Frontotemporal Lobar Degeneration; Dementia; Brain Diseases; Neurocognitive Disorders; Mental Disorders

Study Officials

• Michael Benatar

  University of Miami

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Chief, Neuromuscular Division

Study Record Dates

• First Submitted: February 27, 2019
• First Posted: April 12, 2019
• Study Start: January 22, 2019
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): June 1, 2026
• Last Updated: October 20, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)