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

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational



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

Brief 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. 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.