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

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

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.

Study Overview

• Status: Completed
• Conditions: Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Other: Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter

Detailed Description

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.

• Study Type: Observational
• Enrollment (Actual): 144

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Phoenix, Arizona, United States, 85013
      • Barrow Neurological Institute

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 85 years (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: Yes
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

ALS patient volunteers will be invited to participate in this study as well as healthy subjects for the control group.

Description

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

Study Plan

How is the study designed?

Number of groups / cohorts

3

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Group 1; Participants enrolled solely in ALS AT HOME Outcome measurement devices will be provided. Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter	Other: Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter; Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter
Group 2; Current participants of the Answer ALS study Outcome measurement devices will be provided. Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter	Other: Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter; Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter
Group 3; Participants without neurological disease (controls) Outcome measurement devices will be provided. Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter	Other: Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter; Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in Pulmonary Function	Respirometer	Daily for the first 3 months; for the subsequent 6 months measures will be obtained twice weekly
Change in Quantitative Hand Grip	Digital Handgrip Meter	Daily for the first 3 months; for the subsequent 6 months measures will be obtained twice weekly
Change in Electrical Impedance Myography (EIM) Measurements	Skulpt Chisel	Daily for the first 3 months; for the subsequent 6 months measures will be obtained twice weekly
Change in Actigraphy	Activity Band	Daily for the first 3 months; for the subsequent 6 months measures will be obtained twice weekly
ALSFRS-R	Questionnaire	Weekly up to 9 months
Change in Patient-reported Experience measures (PREMs)	Questionnaire	At Week 1, and then at 3, 6, and 9 months
Adverse Events	Questionnaire	Monthly up to 9 months
Change in Voice/Speech Tracking	Smartphone application	Daily for the first 3 months; for the subsequent 6 months measures will be obtained twice weekly

Collaborators and Investigators

• Sponsor: Jeremy Shefner
• Collaborators: Beth Israel Deaconess Medical Center
• Investigators: Principal Investigator: Jeremy Shefner, MD, Barrow Neurological Institute

Study record dates

Study Major Dates

• Study Start (ACTUAL): February 22, 2017
• Primary Completion (ACTUAL): June 28, 2019
• Study Completion (ACTUAL): September 30, 2019

Study Registration Dates

• First Submitted: December 22, 2016
• First Submitted That Met QC Criteria: January 9, 2017
• First Posted (ESTIMATE): January 11, 2017

Study Record Updates

• Last Update Posted (ACTUAL): February 8, 2023
• Last Update Submitted That Met QC Criteria: February 6, 2023
• Last Verified: February 1, 2023

More Information

Terms related to this study

• Keywords: ALS; neurodegenerative
• Additional Relevant MeSH Terms: Metabolic Diseases; Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Motor Neuron Disease; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: BNI_ALS_001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: IPD may be shared with researchers conducting the Answer ALS study. It will be available through the GUID.