ALS Testing Through Home-based Outcome Measures (ALS AT HOME)

Jeremy Shefner1 site in 1 country144 target enrollmentStarted: February 22, 2017Last updated: February 8, 2023

Conditions

Trial Snapshot

Phase: Not Applicable
Status: Completed
Sponsor
Enrollment: 144
Locations: 1
Primary Endpoint: Adverse Events

Study Overview Study Design Eligibility Criteria Outcomes Investigators Sites Records & Identifiers Similar Trials

Study Overview

Brief Summary

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.

Detailed Description

The objectives of this study are to

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

Study Design

Study Type: Observational
Observational Model: Case Control
Time Perspective: Prospective

Eligibility Criteria

Ages: 18 Years to 85 Years (Adult, Older Adult)
Sex: All
Accepts Healthy Volunteers: Yes

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

Outcomes

Primary Outcomes

Adverse Events

Time Frame: Monthly up to 9 months

Questionnaire

Change in Pulmonary Function