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A Study of Balovaptan in Adults With Autism Spectrum Disorder With a 2-Year Open-Label Extension

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ClinicalTrials.gov Identifier: NCT03504917
Recruitment Status : Recruiting
First Posted : April 20, 2018
Last Update Posted : November 28, 2018
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche

Brief Summary:
This study will evaluate the efficacy, safety, and pharmacokinetics of 10 mg of oral administration balovaptan once a day (QD) compared with matching placebo in adults (18 years and older) with autism spectrum disorder (ASD).

Condition or disease Intervention/treatment Phase
Autism Spectrum Disorder Drug: Balovaptan Drug: Placebo Phase 3

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 350 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Phase III, Randomized, Double-Blind, Placebo-Controlled, Efficacy, and Safety Study of Balovaptan in Adults With Autism Spectrum Disorder With a 2-Year Open-Label Extension
Actual Study Start Date : August 8, 2018
Estimated Primary Completion Date : December 31, 2020
Estimated Study Completion Date : March 31, 2023

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Balovaptan Drug: Balovaptan
Participants will receive 10 mg of oral administration balovaptan once a day (QD).

Placebo Comparator: Placebo Drug: Placebo
Participants will receive matching placebo.




Primary Outcome Measures :
  1. Change from baseline at Week 24 on the Vineland Adaptive Behavior Scales (Vineland-II) two-domain composite (2DC) score [ Time Frame: Baseline, Week 24 ]

Secondary Outcome Measures :
  1. Change from baseline at Week 12 on the Vineland-II 2DC score [ Time Frame: Baseline, Weeks 12 ]
  2. Change from baseline at Weeks 12 and 24 in the Pediatric Quality of Life (PedsQL) Inventory Generic Core Scales, Version 4.0, on summary and total scores [ Time Frame: Baseline, Weeks 12 and 24 ]
  3. Change from baseline at Weeks 12 and 24 in the Vineland-II A composite standard score [ Time Frame: Baseline, Weeks 12 and 24 ]
  4. Change from baseline at Week 12 and 24 on the Vineland-II Socialization domain standard score [ Time Frame: Baseline, Weeks 12 and 24 ]
  5. Change from baseline at Weeks 12 and 24 on the Vineland-II Communication domain standard score [ Time Frame: Baseline, Weeks 12 and 24 ]
  6. Change from baseline at Weeks 12 and 24 on the Vineland-II Daily Living Skills domain standard score [ Time Frame: Baseline, Weeks 12 and 24 ]
  7. Change from baseline in severity of clinical impressions as measured by Clinical Global Impression-Severity (CGI-S) [ Time Frame: Baseline, After 12 weeks and 24 weeks ]
  8. Improvements in clinical impressions, as measured by Clinical Global Impression-Improvement (CGI-I) [ Time Frame: After 12 weeks and 24 weeks of treatment ]
  9. Change from baseline at Weeks 12 and 24 in the Hamilton Anxiety Rating Scale (HAM-A) total and domain scores [ Time Frame: Baseline, Weeks 12 and 24 ]
  10. Proportion of subjects with a >=6-point improvement in Vineland-II 2DC score [ Time Frame: Weeks 12 and 24 ]
  11. Area Under the Concentration-time Curve of RO5285119 in Plasma at Steady State Over 24 Hours (AUC0-24,ss) [ Time Frame: Weeks 2, 8, 12, 20, 24 or early termination ]
  12. Maximum plasma concentration [ Time Frame: Weeks 2, 8, 12, 20, 24 or early termination ]
  13. Apparent Clearance (CL) of RO5285119 [ Time Frame: Weeks 2, 8, 12, 20, 24 or early termination ]
  14. Volume of Distribution (VD) of RO5285119 [ Time Frame: Weeks 2, 8, 12, 20, 24 or early termination ]
  15. Plasma concentration of M2 metabolites (as applicable) [ Time Frame: Weeks 2, 8, 12, 20, 24 or early termination ]
  16. Plasma concentrations of M3 metabolites [ Time Frame: Weeks 2, 8, 12, 20, 24 or early termination ]
  17. Ratios of metabolite to parent drug concentration at trough [ Time Frame: Weeks 2, 8, 12, 20, 24 or early termination ]
  18. Percentage of participants with adverse events [ Time Frame: Week 24 ]


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Subject meets the DSM-5 criteria for ASD for an autism diagnosis and is confirmed using ADOS-2 criteria
  • SRS-2, proxy version, total t-score >=66 at screening
  • A full scale IQ score >=70 on the WASI®-II
  • Subject has an appropriate study partner, in the opinion of the investigator
  • For women of childbearing potential: agreement to remain abstinent or use a contraceptive method with a failure rate of <1% per year during the treatment period and for at least 30 days after the last dose of study drug
  • Treatment with permitted medications (at a stable dose for 12 weeks before screening) and behavioral therapy regimens (regimens stable for 6 weeks before screening), with the intent that such treatments remain stable throughout the study and with no expected changes before the Week 24 visit

Exclusion Criteria:

  • Pregnancy or breastfeeding, or intention to become pregnant during the study
  • Previous initiation of new or major change in psychosocial intervention within 6 weeks prior to screening
  • Unstable or uncontrolled clinically significant affective or psychotic disorders and/or neurologic disorder that may interfere with the assessment of safety or efficacy endpoints
  • Alcohol or substance abuse or dependence disorder during the last 12 months
  • Significant risk for suicidal behavior, in the opinion of the investigator
  • Epilepsy or seizure disorder considered not well controlled within the past 6 months or changes in anticonvulsive therapy within the last 6 months
  • Clinical diagnosis of peripheral neuropathy
  • Within the last 2 years, unstable or clinically significant cardiovascular disease
  • Uncontrolled hypertension
  • Positive serology results for hepatitis B surface antigen (HBsAg), hepatitis C virus (HCV) antibody, or human immunodeficiency virus (HIV) 1 or 2
  • History of coagulopathies, bleeding disorders, blood dyscrasias, hematological malignancies, myelosuppression (including iatrogenic), or current major bleeding event
  • Concomitant disease or condition that could interfere with, or treatment of which might interfere with, the conduct of the study, or what would, in the opinion of the investigator, pose an unacceptable risk to the subject in this study
  • Confirmed clinically significant abnormality in parameters of hematology
  • Confirmed clinically significant abnormality in parameters of clinical chemistry, coagulation, or urinalysis
  • Medical history of malignancy, if not considered cured

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03504917


Contacts
Contact: Reference Study ID Number: WN39434 www.roche.com/about_roche/roche_worldwide.htm 888-662-6728 (U.S. Only) global-roche-genentech-trials@gene.com

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Sponsors and Collaborators
Hoffmann-La Roche
Investigators
Study Director: Clinical Trials Hoffmann-La Roche

Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT03504917     History of Changes
Other Study ID Numbers: WN39434
First Posted: April 20, 2018    Key Record Dates
Last Update Posted: November 28, 2018
Last Verified: November 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Disease
Autistic Disorder
Autism Spectrum Disorder
Child Development Disorders, Pervasive
Pathologic Processes
Neurodevelopmental Disorders
Mental Disorders