A Longitudinal Analysis of Biomarkers in Patients With ALS

Last updated: September 21, 2023
Sponsor: Holy Cross Hospital, Florida
Overall Status: Active - Recruiting

Phase

N/A

Condition

Myasthenia Gravis (Chronic Weakness)

Amyotrophic Lateral Sclerosis (Als)

Treatment

Venipuncture

Lumbar Puncture

Clinical Study ID

NCT05309408
HNR21-001
  • Ages > 18
  • All Genders
  • Accepts Healthy Volunteers

Study Summary

Longitudinally collect biomarkers from patients with amyotrophic lateral sclerosis (ALS) and control participants in order to further elucidate both potential causes and treatments for ALS. Samples and clinical information will be collected from patients with ALS and controls.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. 18 years of age or older at time of consent.
  2. Capable of providing informed consent.
  3. Capable of complying with study procedures.
  4. ALS Subjects only: ALS subjects diagnosed with familial or sporadic ALS as accordingto the El Escorial Criteria (suspected, possible, probable - lab supported, probable,or definite) or individuals with known gene mutations associated with ALS (regardlessof clinical phenotype)
  5. Control Subjects only: Absence of autoimmune myopathy, neuropathy, ALS mimic disorder,or any other neurodegenerative disease or known diagnosis of ALS, or known ALScausative gene
  6. Lumbar Puncture volunteers only: Clinically appropriate to have a lumbar puncture

Exclusion

Exclusion Criteria:

  1. Presence of a neurodegenerative disease other than ALS
  2. Clinically significant history of unstable medical illness
  3. Inability to comply with study procedures, in the view of the investigator
  4. Lumbar Puncture volunteers only: Presence of bleeding disorder, problems withcerebrospinal fluid pressure, allergy to local anesthetics, a topical or other skininfection at the lumbar puncture site
  5. Lumbar Puncture volunteers only: Taking an anti-platelet or anticoagulant drug, suchas Plavix, Brilinta, Ticlid, Warfarin/Coumadin, Lovenox, Elaquis, Pradaxa, Xarelto,etc, and unable to safely hold it for this study. (Aspirin is allowed)
  6. Taking any immunomodulatory or immunosuppressive medication, as determined by theprincipal investigator.

Study Design

Total Participants: 90
Treatment Group(s): 2
Primary Treatment: Venipuncture
Phase:
Study Start date:
June 11, 2021
Estimated Completion Date:
September 30, 2024

Study Description

The objective of the proposed study is to longitudinally collect biomarkers from patients with amyotrophic lateral sclerosis (ALS) and control participants in order to further elucidate both potential causes and treatments for ALS. The investigators will collect blood or any of the following biofluids: blood, urine, and cerebrospinal fluid (CSF), and process, analyze and store plasma, serum, whole blood, cerebrospinal fluid (CSF), urine, peripheral blood mononuclear cells (PBMC), deoxyribonucleic acid (DNA), and/or ribonucleic acid (RNA). Blood collected in this study may also potentially be used to create induced pluripotent stem cells (iPSCs). Excess samples will be stored in a repository at Massachusetts General Hospital ALS Sample Repository for future research on ALS and related motor neuron diseases. Planned analyses may include but will not be limited to: exploring immune cell populations, gene expression profiling, and analysis or metabolites, proteins, RNA, DNA obtained from blood and/or exploring associated clinical parameters. Analyses will explore the differences of these factors between ALS and control subjects.

Samples and clinical information will be collected from patients with ALS and controls. Samples will be collected from ALS subjects a maximum of 7 times over a period of up to 30 months, with approximately 3 months (and no less than 2 months) between visits. Controls will consist of participants who do not have chronic neurodegenerative diseases or diseases that mimic ALS. Up to 60 ALS subjects and 30 control subjects will be enrolled in the study.

Connect with a study center

  • Holy Cross Hospital

    Fort Lauderdale, Florida 33308
    United States

    Active - Recruiting

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