Development of a Proxy Motor Outcome Measure in Young Children With Neuromuscular Disease
| Status: | Completed |
|---|---|
| Conditions: | Neurology |
| Therapuetic Areas: | Neurology |
| Healthy: | No |
| Age Range: | 18 - Any |
| Updated: | 4/6/2019 |
| Start Date: | December 5, 2013 |
| End Date: | March 31, 2018 |
Patient reported outcomes (PROs) instruments are often used to measure meaningful treatment
benefit or risk in clinical trials. PROs allow patients voices to be heard in ways that
assist healthcare clinicians to address treatment effects and individual patient preferences.
Unfortunately, infants and young children, especially those with a debilitating disease, such
as neuromuscular disorders (NMD), may be less able to provide clear and concise information
about treatment effects. In this case, we often defer to parents and guardians (to be
referred to collectively as parents throughout protocol) to provide their perception of their
child s overall health and wellbeing. Including parents in the assessment process recognizes
the unique knowledge parents have of their child s development, reinforces their central role
in implementing interventions, and aids in their ability to make better-informed healthcare
decisions [1]. Yet, most parent-reports for young children are confined to overall quality of
life (QoL). While QoL is an important area of assessment, its meaning varies among different
ages and populations, and its results are often not precise enough to reveal small
differences within samples [2]. A more specific area of concern in children with NMD is
early-onset muscle weakness leading to difficulties in motor function.
The current lack of patient-centered, sensitive measures (based on motor function and item
difficulty hierarchy) that are suited for repeated assessments in infants and young children
with NMD represents a major obstacle to the rapid translation of promising therapeutic
interventions from preclinical models to clinical research studies. Multiple clinical outcome
measures used at a single time-point for capturing a child s functional status are
burdensome, difficult to interpret and do not provide us with comprehensive, meaningful
information to detect changes following an intervention [3]. Psychometric measures that can
be completed by parents make it possible to collect a considerable amount of data over many
time-points rather than being limited to a single clinical observation. Moreover, a
parent-observational measure that focuses on their child s functional performance in their
real-life will maximize the ecological validity of measures of motor development used for
clinical trials.
Objective: To develop a parent reported observational measure of motor development in infants
and young children, which will serve as a complimentary tool to clinical observation by
reporting motor function as observed in the home setting and which will be used in clinical
trials.
Study population: Parents of children aged 0-5 with neuromuscular disease and neuromuscular
experts in pediatrics.
Design: Qualitative (parent interviews, focus groups) and quantitative study (analysis of
newly developed questionnaire)
Outcome Measures: parental responses to phone interviews, neuromuscular expert responses to
focus groups, parental responses to cognitive interviews, validity and reliability of newly
developed questionnaire
benefit or risk in clinical trials. PROs allow patients voices to be heard in ways that
assist healthcare clinicians to address treatment effects and individual patient preferences.
Unfortunately, infants and young children, especially those with a debilitating disease, such
as neuromuscular disorders (NMD), may be less able to provide clear and concise information
about treatment effects. In this case, we often defer to parents and guardians (to be
referred to collectively as parents throughout protocol) to provide their perception of their
child s overall health and wellbeing. Including parents in the assessment process recognizes
the unique knowledge parents have of their child s development, reinforces their central role
in implementing interventions, and aids in their ability to make better-informed healthcare
decisions [1]. Yet, most parent-reports for young children are confined to overall quality of
life (QoL). While QoL is an important area of assessment, its meaning varies among different
ages and populations, and its results are often not precise enough to reveal small
differences within samples [2]. A more specific area of concern in children with NMD is
early-onset muscle weakness leading to difficulties in motor function.
The current lack of patient-centered, sensitive measures (based on motor function and item
difficulty hierarchy) that are suited for repeated assessments in infants and young children
with NMD represents a major obstacle to the rapid translation of promising therapeutic
interventions from preclinical models to clinical research studies. Multiple clinical outcome
measures used at a single time-point for capturing a child s functional status are
burdensome, difficult to interpret and do not provide us with comprehensive, meaningful
information to detect changes following an intervention [3]. Psychometric measures that can
be completed by parents make it possible to collect a considerable amount of data over many
time-points rather than being limited to a single clinical observation. Moreover, a
parent-observational measure that focuses on their child s functional performance in their
real-life will maximize the ecological validity of measures of motor development used for
clinical trials.
Objective: To develop a parent reported observational measure of motor development in infants
and young children, which will serve as a complimentary tool to clinical observation by
reporting motor function as observed in the home setting and which will be used in clinical
trials.
Study population: Parents of children aged 0-5 with neuromuscular disease and neuromuscular
experts in pediatrics.
Design: Qualitative (parent interviews, focus groups) and quantitative study (analysis of
newly developed questionnaire)
Outcome Measures: parental responses to phone interviews, neuromuscular expert responses to
focus groups, parental responses to cognitive interviews, validity and reliability of newly
developed questionnaire
Patient reported outcomes (PROs) instruments are often used to measure meaningful treatment
benefit or risk in clinical trials. PROs allow patients voices to be heard in ways that
assist healthcare clinicians to address treatment effects and individual patient preferences.
Unfortunately, infants and young children, especially those with a debilitating disease, such
as neuromuscular disorders (NMD), may be less able to provide clear and concise information
about treatment effects. In this case, we often defer to parents and guardians (to be
referred to collectively as parents throughout protocol) to provide their perception of their
child s overall health and wellbeing. Including parents in the assessment process recognizes
the unique knowledge parents have of their child s development, reinforces their central role
in implementing interventions, and aids in their ability to make better-informed healthcare
decisions [1]. Yet, most parent-reports for young children are confined to overall quality of
life (QoL). While QoL is an important area of assessment, its meaning varies among different
ages and populations, and its results are often not precise enough to reveal small
differences within samples [2]. A more specific area of concern in children with NMD is
early-onset muscle weakness leading to difficulties in motor function.
The current lack of patient-centered, sensitive measures (based on motor function and item
difficulty hierarchy) that are suited for repeated assessments in infants and young children
with NMD represents a major obstacle to the rapid translation of promising therapeutic
interventions from preclinical models to clinical research studies. Multiple clinical outcome
measures used at a single time-point for capturing a child s functional status are
burdensome, difficult to interpret and do not provide us with comprehensive, meaningful
information to detect changes following an intervention [3]. Psychometric measures that can
be completed by parents make it possible to collect a considerable amount of data over many
time-points rather than being limited to a single clinical observation. Moreover, a
parent-observational measure that focuses on their child s functional performance in their
real-life will maximize the ecological validity of measures of motor development used for
clinical trials.
Objective: To develop a parent reported observational measure of motor development in infants
and young children, which will serve as a complimentary tool to clinical observation by
reporting motor function as observed in the home setting and which will be used in clinical
trials.
Study population: Parents of children aged 0-5 with neuromuscular disease and neuromuscular
experts in pediatrics.
Design: Qualitative (parent interviews, focus groups) and quantitative study (analysis of
newly developed questionnaire)
Outcome Measures: parental responses to phone interviews, neuromuscular expert responses to
focus groups, parental responses to cognitive interviews, validity and reliability of newly
developed questionnaire
benefit or risk in clinical trials. PROs allow patients voices to be heard in ways that
assist healthcare clinicians to address treatment effects and individual patient preferences.
Unfortunately, infants and young children, especially those with a debilitating disease, such
as neuromuscular disorders (NMD), may be less able to provide clear and concise information
about treatment effects. In this case, we often defer to parents and guardians (to be
referred to collectively as parents throughout protocol) to provide their perception of their
child s overall health and wellbeing. Including parents in the assessment process recognizes
the unique knowledge parents have of their child s development, reinforces their central role
in implementing interventions, and aids in their ability to make better-informed healthcare
decisions [1]. Yet, most parent-reports for young children are confined to overall quality of
life (QoL). While QoL is an important area of assessment, its meaning varies among different
ages and populations, and its results are often not precise enough to reveal small
differences within samples [2]. A more specific area of concern in children with NMD is
early-onset muscle weakness leading to difficulties in motor function.
The current lack of patient-centered, sensitive measures (based on motor function and item
difficulty hierarchy) that are suited for repeated assessments in infants and young children
with NMD represents a major obstacle to the rapid translation of promising therapeutic
interventions from preclinical models to clinical research studies. Multiple clinical outcome
measures used at a single time-point for capturing a child s functional status are
burdensome, difficult to interpret and do not provide us with comprehensive, meaningful
information to detect changes following an intervention [3]. Psychometric measures that can
be completed by parents make it possible to collect a considerable amount of data over many
time-points rather than being limited to a single clinical observation. Moreover, a
parent-observational measure that focuses on their child s functional performance in their
real-life will maximize the ecological validity of measures of motor development used for
clinical trials.
Objective: To develop a parent reported observational measure of motor development in infants
and young children, which will serve as a complimentary tool to clinical observation by
reporting motor function as observed in the home setting and which will be used in clinical
trials.
Study population: Parents of children aged 0-5 with neuromuscular disease and neuromuscular
experts in pediatrics.
Design: Qualitative (parent interviews, focus groups) and quantitative study (analysis of
newly developed questionnaire)
Outcome Measures: parental responses to phone interviews, neuromuscular expert responses to
focus groups, parental responses to cognitive interviews, validity and reliability of newly
developed questionnaire
- INCLUSION CRITERIA
- Parent or guardian who has primary responsibility for care (hereafter referred to as
parent ) of an infant or child(ren) under the age of 6 years who has been diagnosed
with an early onset neuromuscular disorder confirmed by muscle biopsy and/or DNA
testing. Medical records documenting the muscle biopsy and/or genetic testing will be
submitted to the investigators.
- Ability to provide consent
- Age 18 years and above
- Sufficient English language skills to read and participate in discussions about the
study questionnaire
- For the focus group of experts:
Expertise (at least 5 years experience in pediatric neuromuscular disease or questionnaire
development). Experts will representatives from the following categories:
- pediatric therapists (physical, occupational, speech, respiratory therapists)
- pediatric medical doctors
- nurses and nurse practitioners
- social scientists with expertise in questionnaire development
- patient advocacy group representatives
EXCLUSION CRITERIA
-Participants with a reported history of emotional distress when discussing their child s
illness
We found this trial at
1
site
9000 Rockville Pike
Bethesda, Maryland 20892
Bethesda, Maryland 20892
Phone: 800-411-1222
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