Clinical trial NCT05067179: Analysis of Human ALS Tissues and Registry of ALS Patients
Official title: Analysis of Human ALS Tissues and Registry of ALS Patients
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Amyotrophic Lateral Sclerosis
Keywords: Amyotrophic Lateral Sclerosis (ALS)
Lou Gehrig's Disease
Neurodegenerative Diseases
Neuromuscular Diseases
Nervous System Diseases
Lead sponsor: University of Illinois at Chicago
Sponsor class: OTHER
Collaborators: name: Mitsubishi Tanabe Pharma America Inc.
First posted: 2021-10-05
Enrollment: 40
Sex: ALL
Minimum age (years): 18
Maximum age (years): 90
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients aged \> 18 years with a diagnosis of ALS being seen at the Neuromuscular Center Clinics, University of Illinois at Chicago

Brief summary: Amyotrophic Lateral Sclerosis (ALS), often referred to as Lou Gehrig's Disease, is a progressive, terminal condition of muscle weakness that is associated with degeneration of neurons in the spinal cord and brain. This devastating disorder afflicts people in the prime of their lives. At the present time, there are no cures for this disorder, and current treatments are marginal at best. Despite years of intensive research, a fundamental understanding of this disease is still lacking. There is a need to identify both reliable markers of disease progression and effective treatments. The goal of this research is to bring a greater understanding of ALS patients closer to the research studies that can lead to new hypotheses and approaches.

Detailed description: Patients will be approached during in-person or phone/online appointments and asked their consent to enter information and results from their physical examinations, electrodiagnostic testing, and other testing done as a standard of care and into a database to monitor disease progression and use them for research purposes. Also, the patients will be asked for consent to undergo a MRI scan.

In a second informed consent form, patients will be enrolled to perform a rapid postmortem autopsy and have the tissue banked for research purposes. It is important to keep the consents separate because some patients may not consent to this postmortem study, but would consent to the premortem studies.

The information gained from the premortem studies will be used during the postmortem examination to select tissue for further processing. Tissue from different regions, including brain, spinal cord, nerve and muscle will be processed in parallel for cellular and molecular analyses that include histology, immunostaining, in situ hybridization, protein, RNA, and small molecule analyses.

Eligibility criteria: Inclusion Criteria:

* Patients over the age of 18
* Established diagnosis of ALS
* Able and willing to give written informed consent and must authorize release and use of protected health information

Exclusion Criteria:

* Patients below the age of 18
* No diagnosis of ALS

Primary outcomes:
measure: Imaging biomarkers
timeFrame: Within 6 months of participant enrollment
measure: Molecular biomarkers
timeFrame: Within 12 months of participant's passing
Secondary outcomes:
Other outcomes:
Locations:
city: Chicago
country: United States
facility: University of Illinois at Chicago
state: Illinois
Study officials:
affiliation: University of Illinois at Chicago
name: Jeffrey Loeb, MD
role: PRINCIPAL_INVESTIGATOR
References: