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- W4240612949 abstract "The North Star Ambulatory Assessment (NSAA) is a validated measure of physical function designed for use in Duchenne muscular dystrophy (DMD). While reproducibility of assessor grading has been demonstrated, test-retest variability has not been available in a clinical trial setting for boys as young as 4. Edasalonexent (CAT-1004) inhibits NF-kB, a key link between loss of dystrophin and skeletal and cardiac muscle disease progression. The global Phase 3 PolarisDMD trial utilizes NSAA as the primary endpoint and enrolled 131 boys from 37 sites. To assess variability of repeat NSAA assessments prior to treatment, and any meaningful learning effect or age dependence. PolarisDMD enrolled boys with DMD 4 to 7 years of age (before their 8th birthday) with any mutation who could stand from supine in ≤10 sec. Key exclusion criteria included glucocorticoid use within the last 6 months and recent investigational therapies. Clinical evaluators received standardized protocol-specific training, and reliability testing with ongoing quality control of assessments by video review and support. After evaluator training, boys were screened, including completing the NSAA. Baseline NSAA was repeated 1 to 4 weeks later. The primary endpoint is change in NSAA after 12 months. Here we compare pre-treatment assessments to determine intra-patient variability. For the 124 boys with valid screening and baseline assessments, change in NSAA was +0.3 points (SD 2.6) from a mean screening score of 20.7 (SD 4.7). The intraclass correlation coefficient was 0.84. Change in NSAA between screening and baseline was not correlated with age, nor with baseline function as measured by the average of screening and baseline. Similar NSAAs between two pretreatment measurements at screening and baseline demonstrated a Phase 3 population with reliable and consistent NSAA scores and implied no significant learning effect over this age range even at the youngest ages." @default.
- W4240612949 created "2022-05-12" @default.
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- W4240612949 date "2020-10-01" @default.
- W4240612949 modified "2023-09-30" @default.
- W4240612949 title "DMD – THERAPY" @default.
- W4240612949 doi "https://doi.org/10.1016/j.nmd.2020.08.283" @default.
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