PHARM - V1aduct Study - A PHASE III, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, EFFICACY, AND SAFETY STUDY OF BALOVAPTAN IN ADULTS WITH ASD
Project Description:
This study will evaluate the efficacy, safety, and pharmacokinetics of 10 mg of oral administration balovaptan once a day (QD) compared with matching placebo in adults (18 years and older) with autism spectrum disorder (ASD). Study WN39434 is a Phase III, randomized, double-blind, placebo-controlled, parallel-group, multicenter, 24-week, efficacy, and safety study of 10 mg of oral balovaptan QD in adults (18 years old or older) with ASD, followed by a 2-year open-label extension period for subjects completing the double-blind treatment period. Subjects providing informed consent will undergo screening within 4 weeks prior to the first double-blind study drug (balovaptan or placebo) administration. Eligible subjects will be
randomized in a 1:1 ratio in a blinded fashion to either 10 mg of balovaptan or matching oral placebo QD for 24 weeks. The primary efficacy endpoint is the change from baseline at Week 24 on the Vineland Adaptive Behavior ScalesVineland-II two-domain composite
(2DC) score.Thus, for subjects remaining in the full study, the approximate length of the study will be 144 weeks or 34 months (screening [approximately 4 weeks], double-blind treatment [24 weeks], and an open-label extension period [104 weeks] with a follow-up period of 12 weeks
after completion of the treatment phase). Subjects will be recruited from international sites, including sites in North America and limited rest of the world (ROW) countries. Subjects who prematurely discontinue from study treatment or from the study will not be replaced. However, all subjects should be followed and their data collected up to and including the Week 24 visit, regardless of their adherence to treatment. Randomization will be stratified by baseline Vineland-II two domain composite (2DC) (the average of Communication and Socialization domains) (60 vs. ≥60), sex (male vs. female), geographical region (North America vs. ROW), and age (25 years vs. 25 years). The main analysis of the primary and secondary efficacy endpoints will occur once the final data from all visits, up to and including Week 24, have been collected and cleaned and the database has
been locked. An interim futility analysis may be performed by an independent Data Monitoring Committee (iDMC) when approximately 50% of subjects complete the Week 24 visit.The iDMC will meet regularly to oversee safety throughout the trial and its activities will be described in a separate iDMC charter. Secondary and exploratory endpoints will examine core autism symptoms (social interaction, social communication, and functional deficits), associated symptoms, impact on health-related quality of life, and broader impacts on study partners and family. Safety will be examined via adverse events, clinical laboratory values, ECGs, physical and neurologic examinations, and safety outcome assessments such as suicidality.
Unserved or Under-served Populations:
Racial or Ethnic Minorities, Disadvantaged Circumstances, Geographic Areas
