Cerebral PalsyNCT02153970
Calibration and Validation of the PROMIS and Neuro-QOL Questionnaires in Cerebral Palsy and Congenital Muscular Dystrophy
An observational study of Cerebral Palsy and Neuromuscular Disease, run by National Institute of Nursing Research (NINR) at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 8 years and older
- Conditions the study is for
- Cerebral Palsy, Neuromuscular Disease
- How many people
- 137 participants
- Where
- 1 siteUnited States
- Study dates
- 31 May 2014to 12 Jun 2019
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- -Caregivers may not be eligible for this research study if they become distressed when talking about their child s illness.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, 20892
About this study
Official title: 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.
Read more
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.
Read the 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.
<TAB>
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.
<TAB>
The outcome measures are the responses to the questionnaires.
<TAB>
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.
- Conditions
- Cerebral Palsy, Neuromuscular Disease
- Keywords
- Clinical Outcomes Measures, Proxy Reported Outcome, Pediatric, Neuromuscular Disease, Cerebral Palsy
- Medical subject headings
- Cerebral Palsy, Neuromuscular Diseases
What is being measured
The primary outcome is the main question the study is designed to answer.
Responses to NeuroQol and PROMIS questionnaires
Measured: ongoing
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Jun 2014 (estimated)
- Study start31 May 2014
- Primary completion4 Apr 2018
- Study completion12 Jun 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Cerebral PalsyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 137 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Institute of Nursing Research (NINR)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Katherine G Meilleur, C.R.N.P., Principal investigator, National Institute of Nursing Research (NINR)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02153970
- Sponsor's protocol ID
- 140118
- Other IDs
- 14-NR-0118
- Status last verified
- 12 Jun 2019
- First submitted
- 31 May 2014
- Last updated
- 14 Jun 2019
- Results posted
- No
Publications
Gershon RC, Lai JS, Bode R, Choi S, Moy C, Bleck T, Miller D, Peterman A, Cella D. Neuro-QOL: quality of life item banks for adults with neurological disorders: item development and calibrations based upon clinical and general population testing. Qual Life Res. 2012 Apr;21(3):475-86. doi: 10.1007/s11136-011-9958-8. Epub 2011 Aug 27.
PubMed 21874314Cella D, Riley W, Stone A, Rothrock N, Reeve B, Yount S, Amtmann D, Bode R, Buysse D, Choi S, Cook K, Devellis R, DeWalt D, Fries JF, Gershon R, Hahn EA, Lai JS, Pilkonis P, Revicki D, Rose M, Weinfurt K, Hays R; PROMIS Cooperative Group. The Patient-Reported Outcomes Measurement Information System (PROMIS) developed and tested its first wave of adult self-reported health outcome item banks: 2005-2008. J Clin Epidemiol. 2010 Nov;63(11):1179-94. doi: 10.1016/j.jclinepi.2010.04.011. Epub 2010 Aug 4.
PubMed 20685078Cella D, Nowinski C, Peterman A, Victorson D, Miller D, Lai JS, Moy C. The neurology quality-of-life measurement initiative. Arch Phys Med Rehabil. 2011 Oct;92(10 Suppl):S28-36. doi: 10.1016/j.apmr.2011.01.025.
PubMed 21958920
Study at a glance
From the registry record, last updated by the sponsor on 14 Jun 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Calibration and Validation of the PROMIS and Neuro-QOL Questionnaires in Cerebral Palsy and Congenital Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT02153970
- Acronym / sponsor ID
- 140118
- Conditions
- Cerebral Palsy, Neuromuscular Disease
- Keywords
- Clinical Outcomes Measures, Proxy Reported Outcome, Pediatric, Neuromuscular Disease, Cerebral Palsy
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 137 participants (actual)
- Lead sponsor
- National Institute of Nursing Research (NINR)US National Institutes of Health
- Study start
- 31 May 2014
- Primary completion
- 4 Apr 2018 (actual)
- Study completion
- 12 Jun 2019 (actual)
- First posted
- 3 Jun 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
<TAB>
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.
<TAB>
The outcome measures are the responses to the questionnaires.
<TAB>
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 8 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (1 listed)
- -Caregivers may not be eligible for this research study if they become distressed when talking about their child s illness.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Responses to NeuroQol and PROMIS questionnaires
Time frame: ongoing
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jun 2019.
- Background: PubMed 21874314Gershon RC, Lai JS, Bode R, Choi S, Moy C, Bleck T, Miller D, Peterman A, Cella D. Neuro-QOL: quality of life item banks for adults with neurological disorders: item development and calibrations based upon clinical and general population testing. Qual Life Res. 2012 Apr;21(3):475-86. doi: 10.1007/s11136-011-9958-8. Epub 2011 Aug 27.
- Background: PubMed 20685078Cella D, Riley W, Stone A, Rothrock N, Reeve B, Yount S, Amtmann D, Bode R, Buysse D, Choi S, Cook K, Devellis R, DeWalt D, Fries JF, Gershon R, Hahn EA, Lai JS, Pilkonis P, Revicki D, Rose M, Weinfurt K, Hays R; PROMIS Cooperative Group. The Patient-Reported Outcomes Measurement Information System (PROMIS) developed and tested its first wave of adult self-reported health outcome item banks: 2005-2008. J Clin Epidemiol. 2010 Nov;63(11):1179-94. doi: 10.1016/j.jclinepi.2010.04.011. Epub 2010 Aug 4.
- Background: PubMed 21958920Cella D, Nowinski C, Peterman A, Victorson D, Miller D, Lai JS, Moy C. The neurology quality-of-life measurement initiative. Arch Phys Med Rehabil. 2011 Oct;92(10 Suppl):S28-36. doi: 10.1016/j.apmr.2011.01.025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Katherine G Meilleur, C.R.N.P. principal investigator
National Institute of Nursing Research (NINR)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cerebral Palsy and Neuromuscular Disease, run by National Institute of Nursing Research (NINR) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Jun 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Responses to NeuroQol and PROMIS questionnaires (time frame: ongoing). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02153970, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Jun 2019.