A Patient Centric Motor Neuron Disease Activities of Daily Living Scale

Amyotrophic Lateral Sclerosis Clinical Trial

Official title:

A Patient Centric Motor Neuron Disease Activities of Daily Living Scale

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02852278
• Other study ID #: CReATe 8003
• Secondary ID: U01TR001263
• Status: Completed
• Start date: December 2016
• Est. completion date: September 13, 2019

Study information

• Verified date: October 2019
• Source: University of South Florida
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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.

Description:

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 410
• Est. completion date: September 13, 2019
• Est. primary completion date: September 13, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Self-reported diagnosis of motor neuron disease (amyotrophic lateral sclerosis, amyotrophic lateral sclerosis - frontotemporal dementia, primary lateral sclerosis, progressive muscular atrophy, or hereditary spastic paraplegia)

• Enrolled in CReATe Connect, an RDCRN Contact Registry

o Individuals of any age, race, ethnicity, and from any location will be able to participate

• Participants will need to be able to fill out the survey in English

Exclusion Criteria:

• Inability to provide informed consent and complete survey

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Atrophy
• Hereditary Spastic Paraplegia
• Motor Neuron Disease
• Muscle Spasticity
• Muscular Atrophy
• Muscular Atrophy, Spinal
• Paraplegia
• Primary Lateral Sclerosis
• Progressive Muscular Atrophy
• Sclerosis
• Spastic Paraplegia, Hereditary

Intervention

Other:
• Web-based Survey
This is a prospective 12-month study of patients with motor neuron disease enrolled in CReATe Connect, an RDCRN Contact Registry.

Locations

Country	Name	City	State
n/a

Sponsors (4)

Lead Sponsor	Collaborator
University of South Florida	National Institutes of Health (NIH), University of Kansas, University of Miami

References & Publications (24)

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* Note: There are 24 references in all — Click here to view all references

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	PADL-ALS Survey	The PADL-ALS survey is a revision of the ALSFR-R, which consists 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 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 for easier understanding. The PADL-ALS also includes questions about pain, emotional lability and a general non-denominational question about faith. The survey is composed of an initial survey and monthly follow up surveys. The initial survey will include additional questions about symptom onset, date of diagnosis, initial region involved, patient impression of diagnosis, general demographic questions, and medications related to their diagnosis of motor neuron disease.	12 months