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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

1000 participants

Study Classification

OBSERVATIONAL

Study Start Date

2021-06-01

Study Completion Date

2031-12-31

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Amyotrophic Lateral Sclerosis Movement Disorders Degenerative Disorder Motor Neuron Disease

Keywords

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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

Study Design

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Observational Model Type

OTHER

Study Time Perspective

OTHER

Study Groups

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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.

No interventions assigned to this group

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.

No interventions assigned to this group

Eligibility Criteria

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Inclusion 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.

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Laura Dugom, MPH

Role: STUDY_DIRECTOR

Target ALS Foundation, Inc.

Amy Easton, PhD

Role: STUDY_DIRECTOR

Target ALS Foundation, Inc.

Reach out to these primary contacts for questions about participation or study logistics.

Laura Dugom, MPH

Role: CONTACT

Phone: 919-440-2073

Role: primary

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21-500-101-70-09

Identifier Type: -

Identifier Source: org_study_id

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

Study Results

Basic Information

Brief Summary

Detailed Description

Conditions

Keywords

Study Design

Study Groups

Amyotrophic Lateral Sclerosis ALS

Healthy

Eligibility Criteria

Inclusion Criteria

Sponsors

Target ALS Foundation, Inc.

Responsible Party

Principal Investigators

Laura Dugom, MPH

Amy Easton, PhD

Locations

Barrow Neurological Institute

University of California San Diego

Georgetown University

Mayo Clinic

Northwestern University

Massachusetts General Hospital

Washington University

Columbia University

Baylor College of Medicine

University of Washington

Instituto Roosevelt

CHALS-CCT UPR MScience

Countries

Central Contacts

Laura Dugom, MPH

Robert Bowser, PhD

Facility Contacts

Whitney Dailey, MS

Gil Gutierrez

Cassie Holmes, NP

Huy Tran

Arleen Matos

Emma Schmidt

Jingqi Zhu

Aisling Finnegan

Nathan Budden

Ben Hoover

Sarah Griffen

Hala Khan, MPH

Jorge Zapiain

Kaycie Opiyo

Manuela Quiroga Carrillo

Luisa Fernanda Acevedo Moreno

Frances Aponte

References

Chipika RH, Finegan E, Li Hi Shing S, Hardiman O, Bede P. Tracking a Fast-Moving Disease: Longitudinal Markers, Monitoring, and Clinical Trial Endpoints in ALS. Front Neurol. 2019 Mar 19;10:229. doi: 10.3389/fneur.2019.00229. eCollection 2019.

Benatar M, Wuu J, Lombardi V, Jeromin A, Bowser R, Andersen PM, Malaspina A. Neurofilaments in pre-symptomatic ALS and the impact of genotype. Amyotroph Lateral Scler Frontotemporal Degener. 2019 Nov;20(7-8):538-548. doi: 10.1080/21678421.2019.1646769. Epub 2019 Aug 21.

Vieira H, Costa N, Sousa T, Reis S, Coelho L. Voice-Based Classification of Amyotrophic Lateral Sclerosis: Where Are We and Where Are We Going? A Systematic Review. Neurodegener Dis. 2019;19(5-6):163-170. doi: 10.1159/000506259. Epub 2020 Mar 3.

Rutkove SB, Narayanaswami P, Berisha V, Liss J, Hahn S, Shelton K, Qi K, Pandeya S, Shefner JM. Improved ALS clinical trials through frequent at-home self-assessment: a proof of concept study. Ann Clin Transl Neurol. 2020 Jul;7(7):1148-1157. doi: 10.1002/acn3.51096. Epub 2020 Jun 9.

Schievink WI. Spontaneous spinal cerebrospinal fluid leaks and intracranial hypotension. JAMA. 2006 May 17;295(19):2286-96. doi: 10.1001/jama.295.19.2286.

Other Identifiers

21-500-101-70-09