A Study of Balovaptan in Adults With Autism Spectrum Disorder With a 2-Year Open-Label Extension
Condition: Autism Spectrum Disorder
MeSH terms: Autistic Disorder; Autism Spectrum Disorder; Child Development Disorders, Pervasive
Intervention: Balovaptan; Placebo
Intervention description: Participants will receive 10 mg of oral administration balovaptan once a day (QD).; Participants will receive matching placebo.
Intervention MeSH terms: Balovaptan
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Enrollment: 322.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No

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).
    

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

          -  Confirmed elevation above upper limit of normal of CK-MB, high sensitivity cardiac
             troponin T, cardiac troponin I, and/or N-terminal pro B-type natriuretic peptide

          -  Positive serology results for hepatitis B surface antigen (HBsAg), hepatitis C virus
             (HCV) antibody, or human immunodeficiency virus (HIV) 1 or 2

          -  History of coagulopathies, bleeding disorders, blood dyscrasias, hematological
             malignancies, myelosuppression (including iatrogenic), or current major bleeding event

          -  Concomitant disease or condition that could interfere with, or treatment of which
             might interfere with, the conduct of the study, or what would, in the opinion of the
             investigator, pose an unacceptable risk to the subject in this study

          -  Confirmed clinically significant abnormality in parameters of hematology

          -  Confirmed clinically significant abnormality in parameters of clinical chemistry,
             coagulation, or urinalysis

          -  Medical history of malignancy, if not considered cured