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

June 13, 2019 updated by: National Institute of Nursing Research (NINR)

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

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.

Study Overview

Status

Completed

Conditions

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.

Study Type

Observational

Enrollment (Actual)

137

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Maryland
      • Bethesda, Maryland, United States, 20892
        • National Institutes of Health Clinical Center, 9000 Rockville Pike

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

8 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

  • 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Responses to NeuroQol and PROMIS questionnaires
Time Frame: ongoing
ongoing

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

National Institute of Nursing Research (NINR)

Investigators

  • Principal Investigator: Katherine G Meilleur, C.R.N.P., National Institute of Nursing Research (NINR)

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

May 31, 2014

Primary Completion (Actual)

April 4, 2018

Study Completion (Actual)

June 12, 2019

Study Registration Dates

First Submitted

May 31, 2014

First Submitted That Met QC Criteria

May 31, 2014

First Posted (Estimate)

June 3, 2014

Study Record Updates

Last Update Posted (Actual)

June 14, 2019

Last Update Submitted That Met QC Criteria

June 13, 2019

Last Verified

June 12, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 140118
  • 14-NR-0118

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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