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

Abstract

Objective: To design an ALS clinical study in which patients are remotely recruited, screened, enrolled and then assessed via daily data collection at home by themselves or caregivers. Methods: This observational, natural-history study included two academic medical centers, one providing overall clinical management and the other overseeing computing and webservices design and management. Both healthy and ALS subjects were recruited on the Internet via advertisement on governmental and foundation websites as well as through Facebook and Google paid advertisements. Individuals underwent screening and enrollment remotely, including signing an electronic informed consent form. Participants were then provided self-measurement equipment and instructed on their use through a series of web-based videos. The equipment included a handgrip dynamometer, spirometer with smartphone connection, electrical impedance myography device, and an activity tracker. ALS Functional Rating Scale-Revised data were also collected. Subjects were asked to collect data daily for three months and twice-weekly for the subsequent six months. Results: One hundred and eleven ALS patients and 30 healthy individuals enrolled in the study from across 41 states (74 men, 62 women). Baseline median ALSFRS-R score was 33. Seventy two percent of the ALS patients sent equipment and 88% of the healthy subjects sent equipment were able to complete a first set of measurements. Expected baseline differences between the ALS patients and healthy participants were identified for all measures. Conclusions: It is possible to design and institute an at-home based study in ALS patients, using a number of state-of-the-art approaches, including web-based consenting and training and Internet-connected measurement devices.

Optimizing telemedicine to facilitate amyotrophic lateral sclerosis clinical trials

“…Patients who were interested in the study could go to the "ALS AT HOME" website to answer the prescreening questions and to provide contact information for the research team. 15 Managing a sprawling online presence for a given trial, and targeting a patient population with an uncommon disease can be a daunting task for a clinical research team. Additional tools exist to help researchers disseminate information about their projects and assist patients in locating and accessing available studies.…”

“…16 Finally, even in the face of powerful online recruitment tools, enrollment in trials across diseases is driven, to a large extent, by "physician factors," particularly the relationship between each patient and their treating physician and the physician's support for a study. 15,17 For this reason, a recruitment approach that combines patient-facing social media and Web-based materials with engaged physicians and site staff might be most effective.…”

“…18 Teleconsent offers tremendous potential, but the loss of personal connection can remain a challenge and should be factored in by researchers when designing the consent process. 15,17 Telescreening refers broadly to the process of assessing trial eligibility for a potential participant from afar. Such techniques can be used to reduce the in-person duration or complexity of a screening visit, or to replace an in-person screening visit altogether, although the latter might be complex in the context of interventional drug trials.…”

“…Remote review of medical records transmitted electronically to a study center can be used to confirm some aspects of patient eligibility and may be feasible as the sole means of screening for some observational studies. 15 The is widely used as the primary outcome measure in many ALS clinical trials and has been validated for both in-person and phone-based administration by a trained research coordinator. [21][22][23] In a study by rater and intra-rater reproducibility.…”

“…33 However, validation of these technologies and refinement of the study methods is still needed to reduce variability and enable wide adoption for clinical trial purposes. 15 Another concern and pending question is whether findings from observational studies of remote technologies might be directly appli- safety and ensure that all data that is required to support the drug development process is collected and documented.…”