Table 9.

Summary table of anchor-based NPCCSS results for the CGI-I

Visit	Collapsed / Uncollapsed	Anchor Category	N	Mean Change (SD)	95% CI	Effect Size	Median Change
Visit 1 to 6	Collapsed	No Change	18	0.83 (2.176)	(-0.249, 1.915)	0.38	0.00
		Worsening	13	2.69 (3.225)	(0.744, 4.641)	0.83	2.00

Abbreviations: CGI-I = Clinician Global Impression of Improvement; CI = confidence interval; NPCCSS = Niemann-Pick type C Clinical Severity Scale; SD = standard deviation

Visit 1 = Screening; Visit 3 = 3 months; Visit 4 = 6 months; Visit 5 = 9 months; Visit 6 = 12 months

All patients in the study (age range 2–19 years) were assessed using the CGI-I