Supplementary Materials

This PDF file includes:

  • Materials and Methods
  • Fig. S1. Distribution and q-q plots of baseline scores on the SRS-2 and Vineland-II composite and domain scores.
  • Fig. S2. Percentage of participants exceeding the MCID for the Vineland-II composite score at week 12.
  • Fig. S3. Individual drug exposure versus change from baseline at week 12 on Vineland-II composite and domain scores.
  • Fig. S4. Frequency of responders on the CGI-I scale at week 12.
  • Table S1. Correlations at baseline between SRS-2 total proxy T score and Vineland-II Adaptive Behavior Scales composite and domain scores.
  • Table S2. Serious adverse events and withdrawals due to adverse events.
  • Table S3. Change from baseline and ES at week 12 on outcome assessments in the placebo group.
  • Table S4. Sensitivity analyses of the Vineland-II Adaptive Behavior composite standard score.
  • Table S5. Change from baseline at week 12 in the PedsQL Generic Core Scales, Cognitive Functioning Scale, and Family Impact Module in the ITT population.
  • Table S6. Analyses of the changes from baseline at week 12 in the PedsQL Generic Core Scales, Cognitive Functioning Scale, and Family Impact Module in the ITT population.
  • Table S7. Adverse events reported in >5% of participants in any treatment group by Medical Dictionary for Regulatory Activities Preferred Term.
  • Table S8. Treatment-emergent orthostatic hypotension.
  • Table S9. Balovaptan steady-state pharmacokinetic parameters.
  • Table S10. Full eligibility criteria.
  • Table S11. Efficacy assessment schedule.

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