Psychometric performance of the Primary Mitochondrial Myopathy Symptom Assessment (PMMSA) in a randomized, double-blind, placebo-controlled crossover study in subjects with mitochondrial disease.
Chad GwaltneyJonathan StokesAnthony AiudiIyar MazarSarah OllisEmily LoveAmel KaraaCarrie R HoutsR J WirthAlan L ShieldsPublished in: Journal of patient-reported outcomes (2022)
Upon evaluation of its psychometric properties, the PMMSA, specifically the 4FS domain, demonstrated strong reliability and construct-related validity. The PMMSA can be used to evaluate treatment benefit in clinical trials with individuals with PMM. Trial registration ClinicalTrials.gov identifier, NCT02805790; registered June 20, 2016; https://clinicaltrials.gov/ct2/show/NCT02805790 .
Keyphrases
- clinical trial
- placebo controlled
- double blind
- phase iii
- phase ii
- psychometric properties
- study protocol
- oxidative stress
- open label
- computed tomography
- late onset
- magnetic resonance imaging
- contrast enhanced
- image quality
- phase ii study
- radiation therapy
- positron emission tomography
- replacement therapy
- drug induced
- patient reported
- early onset
- muscular dystrophy
- squamous cell carcinoma