Amyotrophic Lateral Sclerosis Clinical Trial
Official title:
A Patient Centric Motor Neuron Disease Activities of Daily Living Scale
The purpose of this study is to learn about rates of patient-reported disease progression in patients with motor neuron diseases (amyotrophic lateral sclerosis, progressive muscular atrophy, primary lateral sclerosis, hereditary spastic paraplegia) outside the clinical setting, and the patient-reported clinical characteristics that influence this rate of progression. All patients enrolled in CReATe Connect, a Rare Diseases Clinical Research Network (RDCRN) Contact Registry, will be invited via email to participate in this study.
The patient activities of daily living for amyotrophic lateral sclerosis survey (PADL-ALS,
Appendix B) is a patient-centric revision of the standard revised ALS functional rating scale
used in clinical trials, the ALSFRS-R. The PADL-ALS was developed based on patient
interviews, and patient focus groups. The ALSFRS-R is made up of 12 categories detailing
various activities of daily living and includes six bulbar-respiratory functions, three upper
extremity functions (writing, cutting food, and dressing), and three gross motor functions
(walking, climbing, and turning in bed). Each activity is recorded to the closest
approximation from a list of five choices, scored 0-4, with the total score ranging from 48
(normal function) to 0 (no function). The PADL-ALS includes questions from the ALSFRS-R, with
revisions to make the questions easier to understand. In addition the PADL-ALS contains a
question about pain; a question about emotional lability; and a general non-denominational
question about faith. The survey will be composed of two parts, the initial survey, and then
monthly follow up surveys. The initial survey will include the PADL-ALS with additional
questions about symptom onset, date of diagnosis, initial region involved, patient impression
of diagnosis, general demographic questions (age, gender, race, ethnicity, education), and
medications related to their diagnosis of motor neuron disease.
The survey data will be stored by the Rare Diseases Clinical Research Network's Data
Management and Coordinating Center (DMCC) at the University of South Florida. Upon conclusion
of the study period, the data will be sent to Jeffrey Statland, MD, University of Kansas
Medical Center (Study Chair) and Michael Benatar, MD, PhD, University of Miami (CReATe
Consortium PI). All data collected will be sent to the database of Genotypes and Phenotypes
(dbGaP) to be stored indefinitely.
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