ALS Testing Through Home-based Outcome Measures

ALS AT HOME is a single-center study of up to 150 participants being done to determine the
extent to which frequent sampling can improve the qualities of outcome measures collected at
home by study participants.

The objectives of this study are to

1. assess the extent to which frequent sampling can reduce variability for the following
outcome measures in ALS patients: ALSFRS-R, quantitative hand grip, pulmonary function,
EIM in 4 extremities, and actigraphy and voice/speech tracking;

2. assess the compliance of ALS patients in obtained outcome measures at home over the
course of 9 months;

3. directly compare outcome measures collected by patients with measurements obtained at
study sites at less frequent intervals by co-enrolling 50 patients participating in the
Answer ALS study; and

4. provide information to Answer ALS to more fully characterize patients in that study.

The study proposed here will assess the extent to which reducing variability of measurement
through frequent sampling can reduce both sample size and duration of clinical trials,
provide a first-of-its-kind detailed assessment of disease progression, and evaluate an
entirely new paradigm for patient participation in such trials without the need to be
geographically yoked to a clinical study center. An important component of this proposal is
to jointly recruit 50 patients who also participate in the Answer ALS study that intends to
generate 1000 IP cell lines from patients who receive ongoing assessment at 5 clinical sites.
The 100 participants that will be co-enrolled in Answer ALS and ALS AT HOME will perform
outcome measures at home in addition to having outcome measures performed at clinic sites
through Answer ALS participation. Therefore, the investigators will be able to directly
assess how the measures obtained by patients at home relate to those obtained by a trained
evaluator at a study center using standard metrics. The investigators will also provide all
data generated at home to the Answer ALS investigators, further increasing the phenotypic
data available for that project.

Inclusion Criteria:

- male or female, age 18 to 85 years old,

- ownership of a Smart Device (phone, tablet, etc) with Bluetooth capabilities,

- continuous internet access at home,

- willing and able to provide informed consent in compliance with the regulatory
requirements through a web based interface,

- definite, Probable, or Possible ALS by modified El Escorial criteria, as documented by
medical records, with duration from diagnosis 60 months or less, and

- for the 50 participants in Group 2, participation in the Answer ALS study.

Exclusion Criteria:

- diagnosed and actively undergoing treatment for cancer, heart failure, end stage renal
disease, or another significant medical condition deemed by the PI to likely affect
the participant's ability to comply with the protocol,

- unwilling or unable to comply with the requirements of this protocol, including the
presence of any condition (physical, mental, or social) that is likely to affect the
participant's ability to comply with the protocol,

- any other reasons that, in the opinion of the PI, the candidate is determined to be
unsuitable for entry into the study.

- Healthy volunteers who have family members with ALS