A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Efficacy and Safety Study of Pitolisant Followed by an Open-Label Extension in Patients with Prader-Willi Syndrome

Prader-Willi syndrome

Efficacy: Change in severity of EDS as measured by PROMIS SRI T-score from Baseline to the end of the Double Blind Treatment Period (Day 77)

Efficacy: Change in severity of irritable and disruptive behaviors as measured by ABC-C Irritability Domain score from Baseline to the end of the Double-Blind Treatment Period (Day 77), Efficacy: Change in overall severity of EDS as measured by the CaGI-S for EDS score from Baseline to the end of the Double-Blind Treatment Period (Day 77), Efficacy: Change in overall severity of EDS as measured by the CGI-S for EDS score from Baseline to the end of the Double-Blind Treatment Period (Day 77), Efficacy: Change in overall severity of irritable and disruptive behaviors as measured by the CaGI-S for Irritable and/or Disruptive Behaviors score from Baseline to the end of the Double-Blind Treatment Period (Day 77), Efficacy: Change in severity of hyperphagia as measured by HQ CT score in conjunction with the FSZQ score, from Baseline to the end of the Double-Blind Treatment Period (Day 77), Efficacy: Change in severity of EDS as measured by ESS CHAD (parent/caregiver version) total score from Baseline to the end of the Double-Blind Treatment Period (Day 77), Efficacy: Change in severity of other behavioral problems as measured by the ABC-C Hyperactivity/Noncompliance, Inappropriate Speech, Social Withdrawal, and Stereotypic Behavior Domain scores from Baseline to the end of the Double-Blind Treatment Period (Day 77), Safety and Pharmacokinetic: Percentage of patients reporting TEAEs during each study period and during the entire study, Safety and Pharmacokinetic: Measured concentration of pitolisant

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