Purpose

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.

Condition

Eligibility

Eligible Ages
Between 5 Years and 17 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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)

Study Design

Phase
Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Double (Participant, Investigator)

Arm Groups

ArmDescriptionAssigned Intervention
Placebo Comparator
Placebo
Participants will receive a matching placebo orally. Approximate treatment duration will be up to 24 weeks.
  • Drug: Placebo
    Participants will receive a matching placebo orally. Approximate treatment duration will be up to 24 weeks.
Experimental
Balovaptan (RO5285119) 10 mg/d equivalent
Participants will receive age-adjusted total daily oral dose approximately equivalent to the adult dose of 10 milligrams per day (mg/d) of balovaptan (RO5285119). Approximate treatment duration will be up to 24 weeks (up to 52 additional weeks for those enrolled in the OLE).
  • Drug: RO5285119
    Participants will receive age-adjusted total daily oral dose approximately equivalent to the adult doses of either 4 mg/d or 10 mg/d of balovaptan (RO5285119). Approximate treatment duration will be up to 24 weeks (up to 52 additional weeks for those enrolled in the OLE).
Experimental
Balovaptan (RO5285119) 4 mg/d equivalent
Participants will receive age-adjusted total daily oral dose approximately equivalent to the adult dose of 4 mg/d of balovaptan (RO5285119). Approximate treatment duration will be up to 24 weeks. This arm is open only to those participants enrolled prior to Version 6 of the study protocol.
  • Drug: RO5285119
    Participants will receive age-adjusted total daily oral dose approximately equivalent to the adult doses of either 4 mg/d or 10 mg/d of balovaptan (RO5285119). Approximate treatment duration will be up to 24 weeks (up to 52 additional weeks for those enrolled in the OLE).

Recruiting Locations

Massachusetts General Hospital
Boston, Massachusetts 02114

Massachusetts General Hospital; Lurie Center for Autism
Lexington, Massachusetts 02421

More Details

NCT ID
NCT02901431
Status
Recruiting
Sponsor
Hoffmann-La Roche

Study Contact

Reference Study ID Number: BP30153 www.roche.com/about_roche/roche_worldwide.htm
888-662-6728 (U.S. and Canada)
global-roche-genentech-trials@gene.com

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.