Fig 1.
Natural course of CLN2 disease.
CLN2: Neuronal ceroid lipofuscinosis type 2. Sources: Steinfeld R, et al. 2002 [6] and Nickel M, et al. 2018 [7] image.
Table 1.
Comparison between the Hamburg Scale and Clinical Rating Scale.
Fig 2.
Study 190-201/202 summary diagram.
a10 patients had a DE period ranging from 5–23 weeks. b14 patients enrolled directly to the 300 mg SD for 48 weeks of treatment/assessment. At the parents’ request, one patient withdrew after the receipt of one dose of the study drug owing to an unwillingness to continue with study visits and procedures; the other 23 patients completed the dosing and follow-up for Study 190–201 [9]. c23 patients continued onto Study 190–202 receiving their first dose immediately after the Week 49 assessment in Study 190–201; dData are available for all patients up to Week 49 of Study 190–202; ePatients in Study 109–202 continue treatment for up to 239 weeks. DE: dose escalation; SD: stable dose.
Fig 3.
Scatter plots of domain scores of the Clinical Rating Scale from Study 201/202.
Table 2.
Spearman correlation coefficients of domain scores of the Clinical Rating Scale from Study 201/202.
Table 3.
Variance inflation factors for the domains of the Clinical Rating Scale—Study 201/202.
Table 4.
Variance inflation factors for all pairwise domain comparisons of the Clinical Rating Scale—Study 201/202.
Table 5.
Condition indices for the domains of the Clinical Rating Scale—Study 201/202.
Table 6.
Results of the linear mixed effects models comparing the PedsQL to the total and ML scores of the Clinical Rating Scale.
Table 7.
Results of the linear mixed effects models comparing the PedsQL to the domains of the Clinical Rating Scale.
Table 8.
Results of the multivariate linear mixed effects model comparing the Motor and Language domains of the Clinical Rating Scale to the PedsQL Total score—Study 201/202.