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Table 2 Overall completion rates for clinical outcome assessments

From: Enabling endpoint development for interventional clinical trials in individuals with Angelman syndrome: a prospective, longitudinal, observational clinical study (FREESIAS)

 

AS (N = 55)

TDC (N = 20)

Clinic Visit 1, n (%)

Clinic Visit 2,* n (%)

Clinic Visit 1, n (%)

Clinic Visit 2,* n (%)

Bayley-III

55 (100.0)

34 (61.9)

N/A

N/A

Vineland-3

55 (100.0)

50 (90.9)

20 (100.0)

20 (100.0)

Communication Matrix

52 (94.5)

50 (90.9)

19 (95.0)

20 (100.0)

SNAKE

49 (89.1)

43 (78.2)

N/A

N/A

CSDI

49 (89.1)

42 (76.4)

N/A

N/A

ABC-2-C

50 (90.9)

44 (80.0)

N/A

N/A

CGI-S

55 (100.0)

49 (89.1)

N/A

N/A

FMS

55 (100.0)

48 (87.3)

20 (100.0)

20 (100.0)

PedsQLâ„¢ 4.0 Core

53 (96.4)

44 (80.0)

N/A

N/A

EQ-5D-Y

49 (89.1)

43 (78.2)

N/A

N/A

HOST

50 (90.9)

43 (78.2)

N/A

N/A

PedsQLâ„¢-FIM

53 (96.4)

44 (80.0)

N/A

N/A

ESS

50 (90.9)

42 (76.4)

N/A

N/A

PSQI

50 (90.9)

42 (76.4)

N/A

N/A

EQ-5D-5L

49 (89.1)

42 (76.4)

N/A

N/A

  1. Percentages are derived based on the number of participants who completed assessments at that visit
  2. ABC-2-C Aberrant Behavior Checklist Second Edition – Community Version, AS Angelman syndrome, Bayley-III Bayley Scales of Infant and Toddler Development® – Third Edition, CGI-S Clinical Global Impression – Severity, COVID-19 coronavirus disease 2019, CSDI Composite Sleep Disturbance Index, EQ-5D-5L European Quality of Life 5-Dimensions Questionnaire-Five Levels, EQ-5D-Y European Quality of Life 5-Dimensions Questionnaire-Youth, ESS Epworth Sleepiness Scale, FMS Functional Mobility Scale, HOST holistic assessment of sleep and daily troubles in parents of children with severe psychomotor impairment, N/A not assessed, PedsQL™ 4.0 Core Pediatric Quality of Life Inventory™ Generic Core Scales, Version 4.0, PedsQL™-FIM Pediatric Quality of Life Inventory™ Family Impact Module, PSQI Pittsburgh Sleep Quality Index, SNAKE Schlaffragebogen für Kinder mit Neurologischen und Anderen Komplexen Erkrankungen (Sleep Questionnaire for Children with Severe Psychomotor Impairment), TDC typically developing children, Vineland-3 Vineland Adaptive Behavior Scales® – Third Edition
  3. *Clinic Visit 2 includes data captured during Clinic Visit 2, Early Withdrawal, Remote Visit, and out-of-time window (due to COVID-19)