Recruiting

Observational Study

Sponsor:

Target ALS Foundation, Inc.

Code:

NCT05137665

Conditions

Amyotrophic Lateral Sclerosis

Movement Disorders

Degenerative Disorder

Motor Neuron Disease

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Accepted

Study Details

Brief summary:

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.

Conditions

Amyotrophic Lateral Sclerosis

Movement Disorders

Degenerative Disorder

Motor Neuron Disease

Study ID

NCT05137665

Start date

Jun 1, 2021

Status verified date

Nov, 2025

Completion date

Dec 31, 2031

Anticipated

Primary completion date

Dec 31, 2031

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Accepted

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 Design

Enrollment

1000 participants

Anticipated

Interventions and Outcome Measures

Arms

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.

Primary outcome measure

  • Biofluid Biorepository [ Time Frame: + 3.5 Years ]

Central Contacts and Locations

Central contacts

Laura Dugom, MPH

919-440-2073laura.dugom@targetals.org

Robert Bowser, PhD

602-406-8989robert.bowser@dignityhealth.org

Locations

Barrow Neurological Institute

Recruiting

Phoenix, Arizona, United States, 85013

Contacts

Whitney Dailey, MS

602-406-7804whitney.dailey@dignityhealth.org

Principal Investigator:

Shafeeq Ladha, MD

University of California San Diego

Recruiting

San Diego, California, United States, 92121

Contacts

Gil Gutierrez

grg005@health.ucsd.edu

Principal Investigator:

John Ravits, MD

Georgetown University

Recruiting

Georgetown, District of Columbia, United States, 20007

Contacts

Cassie Holmes, NP

cjh278@georgetown.edu

Principal Investigator:

Brent Harris, MD

Mayo Clinic

Recruiting

Jacksonville, Florida, United States, 32224

Contacts

Huy Tran

tran.huy1@mayo.edu

Principal Investigator:

Bjorn Oskarsson, MD

Northwestern University

Recruiting

Chicago, Illinois, United States, 60611

Contacts

Arleen Matos

(312) 503-0748arleen.matos@northwestern.edu

Emma Schmidt

emma.schmidt@northwestern.edu

Principal Investigator:

Senda Ajroud- Driss, MD

Massachusetts General Hospital

Recruiting

Boston, Massachusetts, United States, 02114

Contacts

Lada Filippov

617-724-7048lfilippov@mgh.harvard.edu

Carolyn Dwyer

617-724-7928targetals@mgb.org

Principal Investigator:

James Berry, MD MPH

Washington University

Recruiting

St Louis, Missouri, United States, 63110

Contacts

Nathan Budden

budden@wustl.edu

Principal Investigator:

Tim Miller, MD

Columbia University

Recruiting

New York, New York, United States, 10032

Contacts

Ben Hoover

bnh2119@cumc.columbia.edu

Sarah Griffen

sjg2220@cumc.columbia.edu

Principal Investigator:

Neil Shneider, MD

Baylor College of Medicine

Recruiting

Houston, Texas, United States, 77030

Contacts

Hala Khan, MPH

Hala.Khan@bcm.edu

Jorge Zapiain

Jorge.ZaragozaZapiain@bcm.edu

Principal Investigator:

Lydia Jane Sharp, MD

University of Washington

Recruiting

Seattle, Washington, United States, 98195

Contacts

Kaycie Opiyo

tkaycie@uw.edu

Principal Investigator:

Michael Weiss, MD

More Information

Sponsor

Target ALS Foundation, Inc.

Last update posted

Nov 21, 2025

Last verified

Nov, 2025

Keywords

  • Amyotrophic Lateral Sclerosis
  • ALS Amyotrophic Lateral Sclerosis
  • Target ALS
  • Longitudinal Biofluids
  • Barrow Neurological Institute
  • New York Genome Center
  • Biofluids Biorepository
  • Genomic-wide association studies
  • observational study
  • Biofluid Samples

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-04. This information was provided to ClinicalTrials.gov by Target ALS Foundation, Inc. on 2025-11-21.