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Calibration and Validation of the PROMIS and Neuro-QOL Questionnaires in Cerebral Palsy and Congenital Muscular Dystrophy

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ClinicalTrials.gov Identifier: NCT02153970
Recruitment Status : Completed
First Posted : June 3, 2014
Last Update Posted : June 14, 2019
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Institute of Nursing Research (NINR) )

Brief Summary:

Background:

- Neuromuscular diseases (NMDs) do not have cures. But future treatments will try to improve the health-related quality of life (HRQoL) in people with NMD. Computer questionnaires can help test HRQoL in people with NMD. They could help clinicians and researchers know how people with NMD and their caregivers are doing. They could also help show if treatments are making a difference.

Researchers want to make sure two of these questionnaires PROMIS (Patient Reported Outcomes Measurement Information System) and Neuro-QOL (Quality of Life in Neurological Disorders) work the same way every time. They also want to make sure the questionnaires test the same things every time.

Objective:

- To make sure the PROMIS and Neuro-QOL questionnaires are valid.

Eligibility:

- Children age 8 17 who have NMD. Also, caregivers of children age 5 17 who have NMD.

Design:

  • Participants will complete the PROMIS and Neuro-QOL questionnaires on a computer. The caregiver and child versions are not the same.
  • Participants will complete the questionnaires at the beginning of the study. It will take about 15 20 minutes. They will complete the questionnaires again after 2 4 weeks. They may receive phone or email reminders.
  • Participants will complete the questionnaires at the NIH outpatient clinic and/or on their own device. At NIH, they will use a computer or tablet.

Condition or disease
Cerebral Palsy Neuromuscular Disease

Detailed Description:

Health-related quality of life (HRQoL) is defined as an individual s perception of his or her well-being and satisfaction with their current circumstances. As is oftentimes the case, in childhood-onset neuromuscular disease (NMD) and cerebral palsy (CP), no definitive cures currently exist. As a result, the primary goal of treatment for these patient populations is to improve quality of life or, more specifically, HRQoL. The medical community s interest in patient-based outcomes such as HRQoL has increased in the last two decades as it allows a multidimensional consideration of a patient s health. This focus has been the impetus for the development of computerized adaptive testing (CAT) instruments such as Quality of Life in Neurological Disorders (Neuro-QOL) and Patient Reported Outcomes Measurement Information System (PROMIS) to provide a means of assessing quality of life in patient populations with chronic diseases, such as neuromuscular disease and cerebral palsy.

The objective of this study is thus to validate the PROMIS and Neuro-QOL scales pediatric domains of self-assessment for individuals with CP and NMD and the PROMIS proxy domains for proxy-assessment of the same populations. The questionnaires will be administered to a combined total of 650 individuals from two sites in order to also calibrate the items and convert the questionnaires into CAT format.

The study population is patients with CP or NMD, ages 8-17 years, caregivers of patients with CP or NMD, whose children are ages 5-17 years. Caregivers are defined in this study as parents or legal guardians of children.

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The design is a prospective study in which patients and caregivers will complete online versions of the PROMIS and Neuro-QOL questionnaires at one of two different sites.

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The outcome measures are the responses to the questionnaires.

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The validation of self-report and proxy-report questionnaires for use in the pediatric population with childhood-onset disease, specifically CP and NMD, will serve as an adjunct to the clinical exam and provide a way for clinicians and researchers to track the effectiveness of regular care and surgical interventions in these populations. The ultimate goal is to produce reliable, validated assessments of HRQoL in these populations via the CAT testing format with the potential to use these tools to inform both clinical decision-making and further research efforts for patients.


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Study Type : Observational
Actual Enrollment : 137 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: The Calibration and Validation of the PROMIS and Neuro-QOL Questionnaires in Cerebral Palsy and Congenital Muscular Dystrophy
Study Start Date : May 31, 2014
Actual Primary Completion Date : April 4, 2018
Actual Study Completion Date : June 12, 2019





Primary Outcome Measures :
  1. Responses to NeuroQol and PROMIS questionnaires [ Time Frame: ongoing ]


Information from the National Library of Medicine

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Ages Eligible for Study:   8 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria
  • INCLUSION CRITERIA:
  • Children 8-17 years old who have been diagnosed with a neuromuscular disorder.
  • Caregivers of children 5-17 years old diagnosed with a neuromuscular disorder.
  • Caregivers have primary responsibility for the care of the child.
  • All participants must be able to read English fluently.
  • Caregivers are able to give consent for themselves and/or their child.

EXCLUSION CRITERIA:

-Caregivers may not be eligible for this research study if they become distressed when talking about their child s illness.


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): NCT02153970


Locations
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United States, Maryland
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States, 20892
Sponsors and Collaborators
National Institute of Nursing Research (NINR)
Investigators
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Principal Investigator: Katherine G Meilleur, C.R.N.P. National Institute of Nursing Research (NINR)

Publications:
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Responsible Party: National Institute of Nursing Research (NINR)
ClinicalTrials.gov Identifier: NCT02153970     History of Changes
Other Study ID Numbers: 140118
14-NR-0118
First Posted: June 3, 2014    Key Record Dates
Last Update Posted: June 14, 2019
Last Verified: June 12, 2019
Keywords provided by National Institutes of Health Clinical Center (CC) ( National Institute of Nursing Research (NINR) ):
Clinical Outcomes Measures
Proxy Reported Outcome
Pediatric
Neuromuscular Disease
Cerebral Palsy
Additional relevant MeSH terms:
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Muscular Dystrophies
Paralysis
Cerebral Palsy
Neuromuscular Diseases
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Brain Damage, Chronic
Brain Diseases
Central Nervous System Diseases
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Genetic Diseases, Inborn