Summary

for people ages 5-17 (full criteria)
at San Francisco, California and other locations
study started
estimated completion

Description

Summary

For participants enrolled prior to Version 6 of the protocol: This is 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 is 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 remain as stated above. All participants that complete the 24-week treatment period will be eligible to participate in an optional 52-week open-label extension (OLE) during which they will receive balovaptan treatment.

Official Title

A Phase II Multi-Center, Randomized, Double-Blind, 24-Week, Parallel Group, Placebo-Controlled Study to Investigate the Efficacy and Safety of Balovaptan (RO5285119) in Children and Adolescents Age 5-17 With Autism Spectrum Disorder (ASD)

Keywords

Autism Spectrum Disorder Disease Autistic Disorder Child Development Disorders, Pervasive Balovaptan (RO5285119) 10 mg/d equivalent Balovaptan (RO5285119) 4 mg/d equivalent

Eligibility

You can join if…

Open to people ages 5-17

  • 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 CAN'T join 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)

Locations

  • University of California at San Francisco accepting new patients
    San Francisco California 94115 United States
  • NRC Research Institute accepting new patients
    Orange California 92868 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Hoffmann-La Roche
ID
NCT02901431
Phase
Phase 2
Study Type
Interventional
Last Updated