A Patient Centric Motor Neuron Disease Activities of Daily Living Scale

NCT02852278 | Completed

• 2 other identifiers: CReATe 8003U01TR001263
• Study Type: observational
• Target: 410
• Locations: 0 countries
• Sites: N/A

Brief 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.

Conditions

Amyotrophic Lateral Sclerosis; Progressive Muscular Atrophy; Primary Lateral Sclerosis; Hereditary Spastic Paraplegia

Keywords

Amyotrophic Lateral Sclerosis; ALS; motor neuron diseases; Progressive Muscular Atrophy; Primary Lateral Sclerosis; Hereditary Spastic Paraplegia

Outcome Measures

Primary Outcomes (1)

• 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

Interventions

Web-based Survey OTHER

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

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with motor neuron disease enrolled in CReATe Connect, an RDCRN Contact Registry

You may qualify if:

• 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

You may not qualify if:

• Inability to provide informed consent and complete survey

Sponsors & Collaborators

• University of South Florida: lead
• National Institutes of Health (NIH): collaborator
• University of Kansas: collaborator
• University of Miami: collaborator

Related Publications (24)

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MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis; Muscular Atrophy, Spinal; Motor Neuron Disease; Spastic Paraplegia, Hereditary

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases; Hereditary Sensory and Motor Neuropathy; Nervous System Malformations; Heredodegenerative Disorders, Nervous System; Polyneuropathies; Peripheral Nervous System Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Study Officials

• Jeffrey Statland, MD

  University of Kansas Medical Center

  PRINCIPAL INVESTIGATOR

• Michael Benatar, MD, PhD

  University of Miami

  PRINCIPAL INVESTIGATOR

• Jeffery Krischer, PhD

  University of South Florida, Data Management Coordinating Center

  PRINCIPAL INVESTIGATOR

• Callyn Kirk, MSPH

  University of South Florida

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 28, 2016
• First Posted: August 2, 2016
• Study Start: December 1, 2016
• Primary Completion: September 13, 2019
• Study Completion: September 13, 2019
• Last Updated: October 16, 2019

Record last verified: 2019-10

Data Sharing

• IPD Sharing: Will not share