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

• ClinicalTrials.gov Identifier: NCT03016897
• Recruitment Status: Completed
• First Posted: January 11, 2017
• Last Update Posted: February 8, 2023
• Sponsor: Jeremy Shefner
• Collaborator: Beth Israel Deaconess Medical Center
• Information provided by (Responsible Party): Jeremy Shefner, Barrow Neurological Institute

Tracking Information

• First Submitted Date: December 22, 2016
• First Posted Date: January 11, 2017
• Last Update Posted Date: February 8, 2023
• Actual Study Start Date: February 22, 2017
• Actual Primary Completion Date: June 28, 2019 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: January 9, 2017)

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

• Original Primary Outcome Measures: Same as current
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: ALS Testing Through Home-based Outcome Measures
• Official Title: ALS Testing Through Home-based Outcome Measures (ALS AT HOME)
• 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

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
• Study Design: Observational Model: Case-Control; Time Perspective: Prospective
• Target Follow-Up Duration: Not Provided
• Biospecimen: Not Provided
• 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.
• Condition: Amyotrophic Lateral Sclerosis

Intervention

Other: Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter

Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter

Study Groups/Cohorts

• Group 1
  Participants enrolled solely in ALS AT HOME Outcome measurement devices will be provided. Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter
  Intervention: Other: 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
  Intervention: Other: 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
  Intervention: Other: Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: September 21, 2018): 144
• Original Estimated Enrollment (submitted: January 9, 2017): 250
• Actual Study Completion Date: September 30, 2019
• Actual Primary Completion Date: June 28, 2019 (Final data collection date for primary outcome measure)

Eligibility Criteria

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years to 85 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: Yes
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT03016897
• Other Study ID Numbers: BNI_ALS_001
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided

IPD Sharing Statement

• Plan to Share IPD: Yes
• Plan Description: IPD may be shared with researchers conducting the Answer ALS study. It will be available through the GUID.

• Current Responsible Party: Jeremy Shefner, Barrow Neurological Institute
• Original Responsible Party: Same as current
• Current Study Sponsor: Jeremy Shefner
• Original Study Sponsor: Same as current
• Collaborators: Beth Israel Deaconess Medical Center

Investigators

• Principal Investigator: Jeremy Shefner, MD; Barrow Neurological Institute

• PRS Account: Barrow Neurological Institute
• Verification Date: February 2023