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

This study is currently recruiting participants. (see Contacts and Locations)
Verified August 2014 by National Institutes of Health Clinical Center (CC)
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Institute of Nursing Research (NINR) )
ClinicalTrials.gov Identifier:
NCT02007213
First received: December 5, 2013
Last updated: August 29, 2014
Last verified: August 2014
  Purpose

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


Condition
Neuromuscular Disease

Study Type: Observational
Study Design: Time Perspective: Prospective
Official Title: Development of a Proxy Motor Outcome Measure in Young Children With Neuromuscular Disease

Resource links provided by NLM:


Further study details as provided by National Institutes of Health Clinical Center (CC):

Primary 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. [ Time Frame: 2-4 hours ] [ Designated as safety issue: No ]

Estimated Enrollment: 165
Study Start Date: November 2013
Estimated Study Completion Date: September 2016
Estimated Primary Completion Date: September 2016 (Final data collection date for primary outcome measure)
Detailed 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

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   Yes
Criteria
  • 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: 1) clinical history with features before the age of five, 2) physical examination, and 3) confirmation by muscle biopsy or DNA testing. Medical records documenting the muscle biopsy 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

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02007213

Contacts
Contact: Irene C Arveson (301) 451-4881 irene.arveson@nih.gov
Contact: Katherine G Meilleur, C.R.N.P. (301) 435-1503 meilleurk@mail.nih.gov

Locations
United States, Maryland
National Institutes of Health Clinical Center, 9000 Rockville Pike Recruiting
Bethesda, Maryland, United States, 20892
Contact: For more information at the NIH Clinical Center contact Patient Recruitment and Public Liaison Office (PRPL)    800-411-1222 ext TTY8664111010    prpl@mail.cc.nih.gov   
United States, Pennsylvania
University of Pittsburgh Cancer Center Recruiting
Pittsburgh, Pennsylvania, United States
Sponsors and Collaborators
Investigators
Principal Investigator: Katherine G Meilleur, C.R.N.P. National Institute of Nursing Research (NINR)
  More Information

Additional Information:
Publications:
Responsible Party: National Institutes of Health Clinical Center (CC) ( National Institute of Nursing Research (NINR) )
ClinicalTrials.gov Identifier: NCT02007213     History of Changes
Other Study ID Numbers: 140023, 14-NR-0023
Study First Received: December 5, 2013
Last Updated: August 29, 2014
Health Authority: United States: Federal Government

Keywords provided by National Institutes of Health Clinical Center (CC):
Neuromuscular Disease
Motor Function
Proxy Reported Outcome
Young Children

Additional relevant MeSH terms:
Neuromuscular Diseases
Nervous System Diseases

ClinicalTrials.gov processed this record on October 21, 2014