ALS longitudinal studies with frequent data collection at home: study design and baseline data

Area of Science:

• Neurology
• Clinical Research Methodology
• Digital Health

Background:

• Amyotrophic Lateral Sclerosis (ALS) presents challenges for traditional in-person clinical studies.
• Remote monitoring offers a potential solution to improve patient participation and data collection frequency.

Purpose of the Study:

• To design and evaluate a novel clinical study for ALS patients.
• To implement remote recruitment, screening, enrollment, and at-home data collection.

Main Methods:

• An observational, natural-history study utilizing online recruitment (websites, social media) and remote enrollment.
• Participants used internet-connected devices (dynamometer, spirometer, myography, activity tracker) for daily/weekly self-measurement.
• ALS Functional Rating Scale-Revised (ALSFRS-R) data were also collected.

Main Results:

• 111 ALS patients and 30 healthy individuals enrolled from 41 states.
• High adherence rates observed: 72% of ALS patients and 88% of healthy subjects completed initial measurements.
• Baseline differences between ALS patients and healthy controls were identified across all measures.

Conclusions:

• It is feasible to conduct ALS clinical studies remotely.
• State-of-the-art approaches like web-based consenting, training, and connected devices enable at-home data collection.
• This methodology can facilitate broader participation and more frequent monitoring in ALS research.