Summary

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

Description

Summary

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 (RO5285119) in children and adolescents aged 5-17 years with ASD who are high functioning (intelligence quotient [IQ] greater than or equal to [>=] 70). Enrollment will be staggered, starting first with adolescents (aged 13 to 17 years) and then with children (aged 5 to 12 years). Initially, a first cohort of approximately 24 adolescents will be enrolled together. Based on pharmacokinetic evaluation, safety, and tolerability in the first adolescent cohort and determined final doses, enrollment of adolescents will resume and the enrollment of a first cohort of approximately 24 children (aged 5 to 12 years) can commence. Based on acceptable safety and tolerability in the first children cohort and determined final doses, enrollment of children will resume.

Official Title

A Phase II Multi-center, Randomized, Double-blind, 24-Week, 3-Arm, 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 RO5285119 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

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