Creating a biorepository of biofluids and clinical data for ALS research

Target ALS Biomarker Study; Longitudinal Biofluids, Clinical Measures, and At - Home Measures

This study is collecting blood, spinal fluid, and urine samples from people with ALS and healthy individuals to help find new ways to understand and treat the disease.

Quick facts

• Study type: Observational
• Enrollment: 1000 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Target ALS Foundation, Inc. (Academic / other)
• Locations: 12 sites (Phoenix, Arizona and 11 other locations)
• Trial ID: NCT05137665 on ClinicalTrials.gov

What this trial studies

This study aims to establish a biorepository of longitudinal biofluids, including blood, cerebrospinal fluid, and urine, linked to genetic and clinical information from patients with Amyotrophic Lateral Sclerosis (ALS) and healthy controls. Over a five-year period, 800 ALS patients and 200 healthy participants will be enrolled from various global sites. Participants will undergo regular clinical assessments and provide biofluid samples, which will be stored in a de-identified manner for future research. The goal is to facilitate the identification and validation of ALS biomarkers to enhance drug development.

Who should consider this trial

Good fit: Ideal candidates include adults aged 18 or older with a confirmed diagnosis of ALS and the ability to comply with study procedures.

Not a fit: Patients with neurological diseases other than ALS or those with known ALS-associated genetic mutations may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could significantly advance the understanding and treatment of ALS by providing critical biomarker data for future research.

How similar studies have performed: Previous studies have attempted similar approaches but have been limited in sample size, making this biorepository a potentially novel and valuable resource.

Eligibility criteria

ALS Participants:

1. Age 18 or older.
2. A diagnosis of ALS in accordance with Gold Coast criteria.
3. Full Vital Capacity (FVC) of ≥30% or at the discretion of the Principal Investigator for the participant's predicted value for gender, height, and age at the time of screening.
4. Ability to provide informed consent and understand the purpose and risks of the study.
5. Ability to comply with study procedures and assessments, in the opinion of the Principal Investigator.

Healthy Control Participants:

1. Age 18 or older.
2. No history of neurological disease, in the opinion of the Principal Investigator.
3. No known ALS- associated genetic mutations at the time of consent.
4. Ability to provide informed consent and understand the purpose and risks of the study.
5. Ability to comply with study procedures and assessments, in the opinion of the Principal Investigator.

Where this trial is running

Phoenix, Arizona and 11 other locations

• Barrow Neurological Institute — Phoenix, Arizona, United States (Recruiting)
• University of California San Diego — San Diego, California, United States (Recruiting)
• Georgetown University — Georgetown, District of Columbia, United States (Recruiting)
• Mayo Clinic — Jacksonville, Florida, United States (Recruiting)
• Northwestern University — Chicago, Illinois, United States (Recruiting)
• Massachusetts General Hospital — Boston, Massachusetts, United States (Recruiting)
• Washington University — St Louis, Missouri, United States (Recruiting)
• Columbia University — New York, New York, United States (Recruiting)
• Baylor College of Medicine — Houston, Texas, United States (Recruiting)
• University of Washington — Seattle, Washington, United States (Recruiting)
• Instituto Roosevelt — Bogotá, Colombia (Recruiting)
• CHALS-CCT UPR MScience — San Juan, Puerto Rico (Recruiting)

Study contacts

• Study coordinator: Laura Dugom, MPH

Conditions Amyotrophic Lateral Sclerosis; Movement Disorders; Degenerative Disorder; Motor Neuron Disease; ALS Amyotrophic Lateral Sclerosis; Target ALS; Longitudinal Biofluids; Barrow Neurological Institute