A Phase III, Randomized, Double-Blind, Placebo-Controlled, Efficacy, and Safety Study of Balovaptan in Adults With Autism Spectrum Disorder With a 2-Year Open-Label Extension
Overview
- Phase
- Phase 3
- Intervention
- Balovaptan
- Conditions
- Autism Spectrum Disorder
- Sponsor
- Hoffmann-La Roche
- Enrollment
- 322
- Locations
- 51
- Primary Endpoint
- Change From Baseline at Week 24 on the Vineland Adaptive Behavior Scales (Vineland-II) Two-domain Composite (2DC) Score.
- Status
- Terminated
- Last Updated
- 4 years ago
Overview
Brief Summary
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).
Investigators
Eligibility Criteria
Inclusion Criteria
- •Subject meets the DSM-5 criteria for ASD for an autism diagnosis and is confirmed using ADOS-2 criteria
- •SRS-2, proxy version, total t-score \>=66 at screening
- •A full scale IQ score \>=70 on the WASI®-II
- •Subject has an appropriate study partner, in the opinion of the investigator
- •For women of childbearing potential: agreement to remain abstinent or use a contraceptive method with a failure rate of \<1% per year during the treatment period and for at least 28 days after the last dose of study drug
- •Treatment with permitted medications (at a stable dose for 12 weeks before screening) and behavioral therapy regimens (regimens stable for 6 weeks before screening), with the intent that such treatments remain stable throughout the study and with no expected changes before the Week 24 visit
Exclusion Criteria
- •Pregnancy or breastfeeding, or intention to become pregnant during the study
- •Previous initiation of new or major change in psychosocial intervention within 6 weeks prior to screening
- •Unstable or uncontrolled clinically significant affective or psychotic disorders and/or neurologic disorder that may interfere with the assessment of safety or efficacy endpoints
- •Substance use disorders during the last 12 months
- •Significant risk for suicidal behavior, in the opinion of the investigator
- •Epilepsy or seizure disorder considered not well controlled within the past 6 months or changes in anticonvulsive therapy within the last 6 months
- •Clinical diagnosis of peripheral neuropathy
- •Within the last 2 years, unstable or clinically significant cardiovascular disease
- •Uncontrolled hypertension
- •Unexplained syncopal episode within the last 12 months
Arms & Interventions
Balovaptan
Intervention: Balovaptan
Placebo
Intervention: Placebo
Outcomes
Primary Outcomes
Change From Baseline at Week 24 on the Vineland Adaptive Behavior Scales (Vineland-II) Two-domain Composite (2DC) Score.
Time Frame: Week 24
Vineland™-II Adaptive Behavior Scales 2-Domain Composite (2DC) Score is defined as mean of the Communication domain standard score \& Socialization domain standard score. If any of the 2 individual domain standard scores is missing 2DC score is not computed. Vineland™-II is an instrument that measures communication, daily living skills, socialization, motor skills and maladaptive behavior of individuals with developmental disabilities. Survey Interview Form will be administered to a subject's reliable study partner in this study, during which the rater or clinician will ask to the study partner open ended questions relating to the subject's activities and behavior. Standardized scores on the Adaptive behavior composite range from 20-160 with higher scores indicating better functioning.
Secondary Outcomes
- Change From Baseline in Severity of Clinical Impressions as Measured by Clinical Global Impression-Severity (CGI-S)(Weeks 12 and 24)
- Improvements in Clinical Impressions, as Measured by Clinical Global Impression-Improvement (CGI-I)(Weeks 12 and 24)
- Change From Baseline at Weeks 12 and 24 in the Hamilton Anxiety Rating Scale (HAM-A) Total and Domain Scores(Weeks 12 and 24)
- Change From Baseline at Week 12 on the Vineland-II 2DC Score(Week 12)
- Change From Baseline at Weeks 12 and 24 in the Pediatric Quality of Life (PedsQL) Inventory Generic Core Scales, Version 4.0, on Summary and Total Scores(Weeks 12 and 24)
- Change From Baseline at Weeks 12 and 24 in the Vineland-II Adaptive Behavior Composite Standard Score(Weeks 12 and 24)
- Change From Baseline at Week 12 and 24 on the Vineland-II Socialization Domain Standard Score(Baseline, Weeks 12 and 24)
- Change From Baseline at Weeks 12 and 24 on the Vineland-II Daily Living Skills Domain Standard Score(Weeks 12 and 24)
- Change From Baseline at Weeks 12 and 24 on the Vineland-II Communication Domain Standard Score(Weeks 12 and 24)
- Proportion of Subjects With a >=6-point Improvement in Vineland-II 2DC Score(Weeks 12 and 24)
- Percentage of Participants With Adverse Events(Week 24 and Up to Approximately 2 Years)