Summary
For participants enrolled prior to Version 6 of the protocol: This was a Phase II multi-center, randomized, double-blind, 24-week, 3-arm, parallel group, placebo-controlled study to investigate the efficacy, safety, and pharmacokinetics of balovaptan in children and adolescents aged 5-17 years with ASD who are high functioning (intelligence quotient [IQ] greater than or equal to [>=] 70). For participants enrolled according to Version 6 of the protocol: This was a Phase II multi-center, randomized, double-blind, 24-week, parallel group, placebo-controlled, 2-arm study with participants assigned either to a 10 milligram (mg) or equivalent dose of balovaptan, or placebo. All other study parameters remained as stated above. There are three parts to this study: PK Part (Study part 1) included up to 8 weeks of treatment, Main Treatment Part (Study part 2) included 24 week of treatment, and the Open Label Extension Part (Study part 3) included Week 24 to Week 76 of treatment. All participants that completed the 24-week treatment period were eligible to participate in an optional 52-week open-label extension (OLE) during which they received balovaptan treatment.
Conditions
Autism Spectrum Disorder
Recruitment Status
Terminated
Eligibility Criteria
Inclusion Criteria:
Fluent in English
Diagnostic and Statistical Manual of Mental Disorders, 5th Edition (DSM-5) criteria for ASD or International Statistical Classification of Diseases and Related Health Problems, 10th revision (ICD10) criteria for Autism diagnosis confirmed by Autism Diagnostic Observational Schedule (ADOS-2) criteria
Social Responsiveness Scale, second edition (SRS-2) (T-score) >= 66
Clinical Global Impressions of Severity (CGI-S) >= 4 (moderately ill) at screening
IQ >= 70 as assessed by Wechsler Abbreviated Scale of Intelligence Scale: Second Edition (WASI-II) or Wechsler Preschool and Primary Scale of Intelligence: Fourth Edition (WPPSI-IV) intelligence test
Language, hearing, and vision compatible with the study measurements as judged by the investigator
Inclusion Criteria for the OLE:
Have completed the blinded treatment phase of the study OR were required to stop dosing at or before Week 8
Have no adverse events that would prohibit starting the OLE
Exclusion Criteria:
Initiation of a major change in psychosocial intervention (including investigational) within 4 weeks prior to screening
Unstable or uncontrolled clinically significant psychiatric and/or neurological disorder that may interfere with the safety or efficacy endpoints
Known personal or family history of cerebral aneurysm
Risk of suicidal behavior
Seizure within the past 6 months
Medical history of alcohol or substance abuse/dependence
Concurrent cardio-vascular disease not considered well controlled by the Investigator
Clinically significant abnormality on electrocardiogram at screening
Concomitant disease or condition (pulmonary, gastro-intestinal, hepatic, renal, metabolic, immunological system, or obesity that could interfere with the conduct of the study
Evidence for current gastro-intestinal bleeding, e.g., active stomach ulcer disease
History of coagulopathies, bleeding disorders, or blood dyscrasias
Positive serology for hepatitis B (HBV), hepatitis C (HCV), human immunodeficiency virus (HIV) 1, or HIV 2
Confirmed clinically significant abnormality in parameters of hematology, clinical chemistry, coagulation, or urinalysis
Medical history of malignancy if not considered cured
Participation in an investigational drug study within 90 days or 5 times the half-life of the investigational molecule (whichever is longer) prior to randomization
Loss of blood over 250 milliliters within three months prior to screening
Allowed medications have not been stable since 4 weeks before screening, and allowed medications for treatment of epilepsy have not been stable since 3 months before screening
Use of prohibited medications within 2 weeks prior to screening visit or 5 times the half-life prior to randomization (whichever is longer)
Intervention
Intervention Type
Intervention Name
Drug
Placebo
Drug
RO5285119
Phase
Phase 2
Gender
All
Min Age
5 Years
Max Age
17 Years
Download Date
February 8, 2021
Principal Investigator
N/A
Primary Contact Information
For more information on this trial, visit clinicaltrials.gov.
Contact
For more information and to contact the study team: