ALS Testing Through Home-based Outcome Measures
Condition: Amyotrophic Lateral Sclerosis
MeSH terms: Motor Neuron Disease; Amyotrophic Lateral Sclerosis
Study type: Observational

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.
    

Intervention name(s): Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter
Intervention type(s): Other
Intervention description(s): Respirometer, Handgrip Meter, Skulpt Chisel, ActigraphyMeter
Observational model: Case-Control
Time perspective: Prospective
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.
      
Keywords: ALS; neurodegenerative