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‘ALS AT HOME’ Study Allows People to Participate Remotely

A new ALS study at Barrow Neurological Institute that allows people to participate remotely could change the way clinical trials for this disease are performed.

ALS Testing Through Home-Based Outcome Measures, or ALS AT HOME, aims to improve how amyotrophic lateral sclerosis is measured and provide evidence that people can participate in clinical trials without ever having to visit a study center.

“This is the first time we’ve done a study where we can send equipment to patients, train them to take their own measurements, and collect data without ever actually seeing them,” said Dr. Jeremy Shefner, chair of neurology at Barrow and the principal investigator for the study.

Dr. Shefner and co-principal investigator Dr. Seward Rutkove of Beth Israel Deaconess Medical Center in Boston created the study to address two main problems. The first is a statistical one.

“When we do ALS studies, we have people come into the study center and we measure whatever we happen to be measuring them on,” Dr. Shefner said. “They go home, they come back a month or two or three later, and we get another point. When you do that, you get a line that reflects the progression of the disease, but there’s a fair amount of scatter points from time to time. So, you don’t really know whether the variability that you see is due to the fact that the measurement is imperfect or if this is a true reflection of disease progression. You can’t really tease these things apart if you have interval measurements like that.”

ALS AT HOME requires participants to measure their breathing function, grip strength, muscle quality, speech, and activity level every day for three months and then twice a week for six months. This will help investigators better understand how often this data needs to be recorded to reduce variability and the extent to which reducing variability through frequent sampling can reduce both the sample size and duration of clinical trials.

photo of phoenix neurologist jeremy shefner

Jeremy Shefner, MD, PhD
Kemper and Ethel Marley Professor and Chair of Neurology
Senior Vice President, Barrow Neurological Institute

The second reason for the study is to address the problem of ALS patients having increasing difficulty visiting a study center to be part of a treatment trial.

“In our center, we have people come from Northern Arizona, Mexico, New Mexico, and sometimes Texas,” Dr. Shefner said. “They can come here once for a second opinion, but they’re really unlikely to come here on a regular basis for a trial. If people can measure themselves reliably and we can get accurate data, then we could potentially enroll people into clinical trials who are very far away from any study center and get these data remotely.”

The study team hopes to recruit 250 people from across the country: 220 with ALS and 30 without. One hundred of the participants with ALS will be co-enrolled into the Answer ALS study, which was created to build the largest and most comprehensive foundation of ALS data ever amassed in hopes of uncovering ALS causes, subtypes, and drug targets. Participants enrolled in both studies will take measurements at home in addition to having them taken at clinic sites in less frequent intervals so that investigators can assess how measures obtained by the patients remotely relate to those obtained by trained evaluators at a study center. This will also provide more data for the Answer ALS study.

Participants must be between the ages of 18 and 85. Currently, people with ALS must have been diagnosed within the past three years to participate, but people who have been diagnosed within the past five years are encouraged to check back as they may be eligible under an amendment to the requirements.

People who are eligible to participate in the study will receive their equipment in the mail, along with instruction manuals and access to training videos. Participants must pass a post-training test to show that they understand how to use the equipment.

Participants will manually enter some of the data onto the study website, but other data will automatically upload through their smartphones. Participants must have a smartphone with Bluetooth capabilities and continuous Internet access. In addition to taking measurements, they will also complete intermittent surveys about their experience in the study and their ability to perform certain daily tasks.

Dr. Shefner is particularly excited about the speech tracking technology. Participants will record their voices on their smartphones as they read certain sentences and repeat sounds and words. A group called Oral Analytics will then analyze various aspects of the participants’ speech.

Dr. Shefner and Study Manager Kerisa Shelton, who has a PhD in neuroscience and behavior, are optimistic about obtaining reliable data from study participants. They have already enrolled about 30 people from several different states across the country.

“They haven’t all gotten to the stage yet where they are actually taking measures, but we have over a dozen who are taking measurements right now and they seem to be doing a pretty good job,” Dr. Shelton said.

For more information about the study or to become a participant, visit the ALS AT HOME website.

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About Barrow Neurological Institute

Since our doors opened as a regional specialty center in 1962, we have grown into one of the premier destinations in the world for neurology and neurosurgery. Our experienced, highly skilled, and comprehensive team of neurological specialists can provide you with a complete spectrum of care–from diagnosis through outpatient neurorehabilitation–under one roof. Barrow Neurological Institute: Discover. Educate. Heal.