Physician Information

Study rationale

Although medications that treat some of the secondary conditions associated with autism spectrum disorder (ASD) are available, none have been approved to address ASD’s core symptoms. RO5285119 (Balovaptan) is a potent and highly selective human vasopressin 1a (V1a) receptor antagonist (shown to modulate key social behaviors). It has been shown to normalize the social behavior and cognitive impairments induced in an animal model of ASD.

Primary objectives

We’ll aim to evaluate the safety, tolerability and efficacy (measured by the VinelandTM-II Adaptive Behavior Scales, second edition composite standard score) of 24-week treatment with balovaptan compared to placebo.

Study design


(3-8 weeks)


(24 weeks)
Balovaptan (n=200) or a Placebo (n=100)


(up to 7 weeks)

Health monitoring

Most participants will have approximately 7 clinic visits and 4 home visits (where possible) in total. We’ll monitor their general health and ASD symptoms using assessments such as blood tests, physical examinations and questionnaires. Please note: the schedule for the first 24 participants and those "on hold" will be slightly different.

Key eligibility criteria

  • Males or females aged 5-17 years at randomization
  • DSM-5 criteria for ASD or ICD10 criteria for Autism diagnosis confirmed by ADOS-2 criteria
  • SRS-2 (T-score) ≥ 66
  • CGI-S ≥ 4 (moderately ill) at screening
  • IQ ≥ 70 as assessed by WASI-II or WPPSI-IV intelligence test
  • A parent or guardian who will accompany the participant to all clinic visits and provide information

For more information or to refer a patient

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