Target ALS Biomarker Study; Longitudinal Biofluids, Clinical Measures, and At Home Measures (TALSLB)

November 20, 2025 updated by: Target ALS Foundation, Inc.

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

The goal of the study is to generate a biorepository of longitudinal biofluids-blood (plasma and serum), cerebral spinal fluid (CSF) and urine linked to genetics and longitudinal clinical information that are made available to the research community. To accomplish these goals, we will enroll 800 Amyotrophic Lateral Sclerosis (ALS) patients and 200 healthy controls from sites globally, over a 5 year time frame. Additionally, speech and motor function and spirometry measures will be collected bi-weekly in a subset of participants. ALS participants will be asked to come to the clinic for 5 study visits approximately every 4 months. Healthy participants will be coming for 2 study visits with a 12-month interval between visits. These samples and clinical information will be stored in a de-identified manner and made available for investigators to use in future research studies.

Study Overview

Status

Recruiting

Conditions

Detailed Description

An industry wide survey performed by Dr. Lyle Ostrow at Johns Hopkins University indicated that longitudinal biofluids linked to detailed clinical information are critical to continued drug development for amyotrophic lateral sclerosis (ALS), with CSF being the top biofluid often lacking in longitudinal sample biorepositories. There have been prior efforts for longitudinal collection of biofluids matched to clinical information, but those sample sets are limited in size and quickly utilized by the research community. Based upon input from industry leaders, we propose the creation of a Target ALS longitudinal biofluids biorepository linked to patient genetic and clinical information.

Given the heterogeneous clinical and biologic nature of ALS, a repository of longitudinal samples linked to clinical and genetic information is essential to help identify and verify ALS biomarkers (1,2). Recent studies to identify ALS biomarkers have used longitudinal samples from either the sporadic patient population or from those that harbor genetic mutations known to cause ALS but are not yet symptomatic (3,4). Beyond exploring the relationship between known causative genes and candidate biomarkers, the Target ALS Postmortem Core has collected postmortem ALS tissue samples that have been used to generate large transcriptomic databases that are linked to whole genome sequencing information. These data have been valuable at finding new subtypes of ALS linked to transcriptomic profiles from the tissue samples (5). The current study utilizes some of the medical centers participating in the Target ALS Postmortem Core to create a longitudinal biofluids repository form living ALS patients and health controls. A select number of participating sites will have a portion of ALS participants (approx. 150) have the option to take part in at home speech measures. Additionally, a select number of participating sites will also take part in at- home spirometry measures for approximately 100 ALS participants and approximately 50 healthy case control participants, for a total of 150 participants. The added feature of these at- home measures are to further evaluate the potential for at home measures in future clinical trials and ability to obtain enriched speech and vital capacity measures to correlate to downstream biomarker studies using biofluid or genetic data. There is a growing interest in the use of at home speech analytics to classify and monitor ALS patients, with recent studies indicating the value for these at home measures in both clinical research and clinical trial settings (6-8). Our study will not only expand upon these early findings but also include at home spirometry measures of vital capacity to evaluate the ability to obtain reliable vital capacity measures at home.

Our proposed study will provide valuable longitudinal biofluids linked to clinical information, genetic data, at-home speech measures, and vital capacity measures for use in future research studies. Target ALS has planned to generate proteomic, lipidomic and metabolomic datasets using the longitudinal biofluids collected in this study. Inclusion of samples with racial and ethnic diversity will further strengthen study outcomes to be applied to ALS communities more broadly. These de-identified samples and clinical information will be available to investigators throughout the world to enhance ALS research and ultimately improved treatments for ALS. There is a long history of benefit for biorepositories with linked clinical data to be instrumental in research progress. Most studies that identify biomarkers or validate biomarkers for human diseases typically require banked samples that are linked to clinical information to determine sensitivity and specificity of the biomarker for that disease or to demonstrate change over the course of the disease.

Study Type

Observational

Enrollment (Estimated)

1000

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Colombia
      • Bogotá, Colombia
  • Puerto Rico
      • San Juan, Puerto Rico, 00935
        • Recruiting
        • CHALS-CCT UPR MScience
        • Contact:
        • Principal Investigator:
          • Valerie Wojna, MD
        • Sub-Investigator:
          • Brenda Deliz, MD
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85013
        • Recruiting
        • Barrow Neurological Institute
        • Contact:
        • Principal Investigator:
          • Shafeeq Ladha, MD
    • California
      • San Diego, California, United States, 92121
        • Recruiting
        • University of California San Diego
        • Principal Investigator:
          • John Ravits, MD
        • Contact:
    • District of Columbia
      • Georgetown, District of Columbia, United States, 20007
        • Recruiting
        • Georgetown University
        • Principal Investigator:
          • Brent Harris, MD
        • Contact:
        • Principal Investigator:
          • Nicholas Streicher, MD
    • Florida
      • Jacksonville, Florida, United States, 32224
        • Recruiting
        • Mayo Clinic
        • Principal Investigator:
          • Bjorn Oskarsson, MD
        • Contact:
    • Illinois
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Recruiting
        • Massachusetts General Hospital
        • Principal Investigator:
          • James Berry, MD MPH
        • Contact:
        • Contact:
    • Missouri
      • St Louis, Missouri, United States, 63110
        • Recruiting
        • Washington University
        • Principal Investigator:
          • Tim Miller, MD
        • Principal Investigator:
          • Cindy Ly, MD
        • Contact:
    • New York
      • New York, New York, United States, 10032
    • Texas
      • Houston, Texas, United States, 77030
        • Recruiting
        • Baylor College of Medicine
        • Contact:
        • Contact:
        • Principal Investigator:
          • Lydia Jane Sharp, MD
        • Sub-Investigator:
          • James Orengo, MD PhD
    • Washington
      • Seattle, Washington, United States, 98195
        • Recruiting
        • University of Washington
        • Principal Investigator:
          • Michael Weiss, MD
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

Yes

Sampling Method

Non-Probability Sample

Study Population

A biorepository of longitudinal blood (plasma and serum), cerebral spinal fluid (CSF) and urine linked to genetics and longitudinal clinical information that are made available to the research community will be made from ALS and healthy control participants. This is a large multi-center study, including several international sites with the goal of developing a robust sample set from genetic and racially diverse populations. To enable well-powered biomarker research, the enrollment goal is at least 800 ALS patients and 200 healthy participants in five years.

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Amyotrophic Lateral Sclerosis ALS
This is a global, multi-center study of ALS participants and healthy controls that will have up to 20 sites globally. Target enrollment will be approximately 1000 participants with 800 ALS participants and 200 healthy control cases. Research participants with suspected, possible, probable, probable- laboratory supported, and definite Amyotrophic Lateral Sclerosis (ALS), according to the revised El Escorial Criteria (EEC) or with a diagnosis based on the Gold Coast Criteria will be seen at the Screening/Baseline Visit(s) (Visit 1) and follow-up will occur at approximate 4-month internals for up to 5 visits per participant.
Healthy
Healthy control participants will have a neurological exam to confirm non-neurologic disease status and participants will have one follow up visit approximately 12 months after their baseline visit. Upon consenting for participation, all study participants will undergo the activities and biofluid collections at each visit.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Biofluid Biorepository
Time Frame: + 3.5 Years
This project will create a biorepository of longitudinal biofluid samples, linked to clinical measures, and at home measures
+ 3.5 Years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Target ALS Foundation, Inc.

Investigators

  • Study Director: Amy Easton, PhD, Target ALS Foundation, Inc.
  • Study Director: Laura Dugom, MPH, Target ALS Foundation, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 1, 2021

Primary Completion (Estimated)

December 31, 2031

Study Completion (Estimated)

December 31, 2031

Study Registration Dates

First Submitted

November 16, 2021

First Submitted That Met QC Criteria

November 16, 2021

First Posted (Actual)

November 30, 2021

Study Record Updates

Last Update Posted (Actual)

November 21, 2025

Last Update Submitted That Met QC Criteria

November 20, 2025

Last Verified

November 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 21-500-101-70-09

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

These samples and clinical information will be stored in a de-identified manner and made available for investigators to use in future research studies.

It is expected to take approximately 5 years to complete all sample and data collection for the study. The first phase of the study will involve activating all sites participating in the study, and will take about 4 months to complete. The enrollment period is expected to be about 5 years. Active assessment for each subject will be for 16-18 months from the time of enrollment. After the end of active assessment period (5 years), the bio-samples and clinical information from the study will be made available for future research.

IPD Sharing Time Frame

Greater than 5+ Years

IPD Sharing Access Criteria

Access available using published manuscripts and stored biofluids using LIMS.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • ICF
  • CSR

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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