A Study to Investigate the Efficacy and Safety of Balovaptan (RO5285119) in Participants With Autism Spectrum Disorder (ASD)

Stopped early · Phase 2 · Has a placebo group

Conditions studied: Autism Spectrum Disorder

In brief

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.

Key facts

Study ID

NCT02901431

Run by

Hoffmann-La Roche

People needed

339

Starts

2016-11-21

Expected to finish

2020-06-30

Last updated by the study team

2021-02-08

Who can join

Age: 5 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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)

Where it is running

• Harmonex Neuroscience Research — Dothan, Alabama, United States
• Southwest Autism Research & Resource Center — Phoenix, Arizona, United States
• NRC Research Institute — Orange, California, United States
• PCSD Feighner Research — San Diego, California, United States
• University of California at San Francisco — San Francisco, California, United States
• Children's Hospital of Colorado — Aurora, Colorado, United States
• DBA IMMUNOe Int'l Res Center — Centennial, Colorado, United States
• Yale University / Yale-New Haven Hospital — New Haven, Connecticut, United States
• Sarkis Clinical Trials — Gainesville, Florida, United States
• Segal trials — North Miami, Florida, United States
• Medical Research Group of Central Florida — Orange City, Florida, United States
• APG- Advanced Psychiatric Group — Orlando, Florida, United States
• USF Rothman Center — St. Petersburg, Florida, United States
• Rush University Medical Center — Chicago, Illinois, United States
• Capstone Clinical Research — Libertyville, Illinois, United States
• Kennedy Krieger Institute — Baltimore, Maryland, United States
• Massachusetts General Hospital — Boston, Massachusetts, United States
• Boston Childrens Hospital — Boston, Massachusetts, United States
• Massachusetts General Hospital; Lurie Center for Autism — Lexington, Massachusetts, United States
• UMASS Medical School — Worcester, Massachusetts, United States
• University of Minnesota; Clin. Neuro Research Unit — Minneapolis, Minnesota, United States
• St. Charles Psychiatric Associates — Saint Charles, Missouri, United States
• Midwest Childrens Health Research Institute — Lincoln, Nebraska, United States
• Center for Autism and the Developing Brain — New York, New York, United States
• Nathan S. Kline Institute for Psychiatric Research — Orangeburg, New York, United States

Full record on ClinicalTrials.gov

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