Target ALS Biomarker Study Open to Patients and Healthy Family, Friends
The Barrow Neurological Institute in Phoenix, Arizona, is calling on people with amyotrophic lateral sclerosis (ALS) and their family members and friends to participate in a biomarker study that aims to create a bank of biological samples and clinical data collected over time.
Data will be made available to the research community to further the understanding of how ALS progresses and how to best treat it.
Researchers expect to collect samples from at least 200 ALS patients and 80 healthy individuals over a period of five years. The samples include blood, urine, and cerebrospinal fluid (the fluid that flows in and around the brain and the spinal cord), plus speech records and lung health measures.
The observational Target ALS Biomarker, or TALSLB, study (NCT05137665) is open to adults, 18 or older, with a suspected, possible, probable, or definite ALS diagnosis. Patients will complete a total of five clinic visits at four-month intervals across 16 months, according to a press release.
Healthy adults, family or friends of ALS patients who are willing to complete a total of two clinic visits across one year, are also welcome. To be eligible, they must have no history of neurological disease but can carry a genetic mutation known to cause ALS, provided they have no disease symptoms.
All enrolled must have internet access in the home and access to either a smartphone or tablet.
Clinics involved with this study, in addition to the Barrow institute, are those also taking part in the Target ALS Postmortem Core and include Columbia University in New York, Georgetown University in Washington D.C., and University of California at San Diego. Contact and further site information is available here.
At visits, the researchers will perform clinical exams and collect the biological samples, which will stripped of identifying information and stored for use in future research. Blood samples also will be used to obtain DNA for genetic studies.
The first symptoms of ALS can occur at any age and vary in severity, and they usually include muscle weakness and wasting. Such symptoms can affect how patients speak or breathe. As the disease progresses, patients may experience slurred speech and difficulty in projecting the voice.
To gather data about these problems and how they may correlate with disease biomarkers, the researchers will collect voice samples, which will be recorded at home on a weekly basis as the patients speak to an application installed on a smartphone or tablet.
Every two weeks, about half of the patients and 30 healthy adults also will take at-home measurements of their vital capacity using spirometry, a test that measures how much air can be breathed out in one forced breath.
“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,” the researchers wrote.
“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,” they added.
The trial is sponsored by Target ALS, a research foundation that supports the search for better ALS treatments.
“There is a long history of benefit for biorepositories with linked clinical data to be instrumental in research progress,” the investigators wrote. “Most studies that identify biomarkers or validate biomarkers for human diseases typically require banked samples that are linked to clinical information to determine sensitivity/specificity of the biomarker for that disease or to demonstrate change over course of disease.”
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