Development of a Proxy Motor Outcome Measure in Young Children With Neuromuscular Disease

Neuromuscular Disease Clinical Trial

Official title: Development of a Proxy Motor Outcome Measure in Young Children With Neuromuscular Disease

Status Terminated

Clinical Trial Summary

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

Clinical Trial Description

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 ;

Related Conditions & MeSH terms

• Neuromuscular Disease
• Neuromuscular Diseases

• NCT number: NCT02007213
• Study type: Observational
• Source: National Institutes of Health Clinical Center (CC)
• Status: Terminated
• Start date: December 5, 2013
• Completion date: March 31, 2018