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A Patient Centric Motor Neuron Disease Activities of Daily Living Scale

This study is enrolling participants by invitation only.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02852278
First Posted: August 2, 2016
Last Update Posted: March 3, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborators:
National Institutes of Health (NIH)
University of Kansas
University of Miami
Information provided by (Responsible Party):
University of South Florida
  Purpose
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.

Condition Intervention
Amyotrophic Lateral Sclerosis Progressive Muscular Atrophy Primary Lateral Sclerosis Hereditary Spastic Paraplegia Other: Web-based Survey

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Patient Centric Motor Neuron Disease Activities of Daily Living Scale

Resource links provided by NLM:


Further study details as provided by University of South Florida:

Primary Outcome Measures:
  • PADL-ALS Survey [ Time Frame: 12 months ]
    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.


Estimated Enrollment: 356
Actual Study Start Date: December 2016
Estimated Study Completion Date: December 2017
Estimated Primary Completion Date: December 2017 (Final data collection date for primary outcome measure)
Intervention Details:
    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.
Detailed 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.

  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with motor neuron disease enrolled in CReATe Connect, an RDCRN Contact Registry
Criteria

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

  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02852278


Sponsors and Collaborators
University of South Florida
National Institutes of Health (NIH)
University of Kansas
University of Miami
Investigators
Study Chair: Jeffrey Statland, MD University of Kansas Medical Center
Study Chair: Michael Benatar, MD, PhD University of Miami
Study Chair: Jeffery Krischer, PhD University of South Florida, Health Informatics Institute
Principal Investigator: Callyn Kirk, MSPH University of South Florida, Health Informatics Institute
  More Information

Publications:

Responsible Party: University of South Florida
ClinicalTrials.gov Identifier: NCT02852278     History of Changes
Other Study ID Numbers: CReATe 8003
U01TR001263 ( U.S. NIH Grant/Contract )
First Submitted: July 28, 2016
First Posted: August 2, 2016
Last Update Posted: March 3, 2017
Last Verified: March 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by University of South Florida:
Amyotrophic Lateral Sclerosis
ALS
motor neuron diseases
Progressive Muscular Atrophy
Primary Lateral Sclerosis
Hereditary Spastic Paraplegia

Additional relevant MeSH terms:
Sclerosis
Atrophy
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Muscular Atrophy
Muscle Spasticity
Paraplegia
Spastic Paraplegia, Hereditary
Muscular Atrophy, Spinal
Pathologic Processes
Pathological Conditions, Anatomical
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Signs and Symptoms
Muscular Diseases
Musculoskeletal Diseases
Muscle Hypertonia
Paralysis
Hereditary Sensory and Motor Neuropathy
Nervous System Malformations
Heredodegenerative Disorders, Nervous System
Polyneuropathies