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

NCT03504917 | Terminated Phase 3 Results DMC FDA Drug

• 1 other identifier: WN39434
• Study Type: interventional
• Target: 322
• Locations: 6 countries
• Sites: 51

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).

Conditions

Autism Spectrum Disorder

Outcome Measures

Primary Outcomes (1)

• Change From Baseline at Week 24 on the Vineland Adaptive Behavior Scales (Vineland-II) Two-domain Composite (2DC) Score.

  Vineland™-II Adaptive Behavior Scales 2-Domain Composite (2DC) Score is defined as mean of the Communication domain standard score \& Socialization domain standard score. If any of the 2 individual domain standard scores is missing 2DC score is not computed. Vineland™-II is an instrument that measures communication, daily living skills, socialization, motor skills and maladaptive behavior of individuals with developmental disabilities. Survey Interview Form will be administered to a subject's reliable study partner in this study, during which the rater or clinician will ask to the study partner open ended questions relating to the subject's activities and behavior. Standardized scores on the Adaptive behavior composite range from 20-160 with higher scores indicating better functioning.

  Week 24

Secondary Outcomes (11)

• Change From Baseline at Week 12 on the Vineland-II 2DC Score

  Week 12

• 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

  Weeks 12 and 24

• Change From Baseline at Weeks 12 and 24 in the Vineland-II Adaptive Behavior Composite Standard Score

  Weeks 12 and 24

• Change From Baseline at Week 12 and 24 on the Vineland-II Socialization Domain Standard Score

  Baseline, Weeks 12 and 24

• Change From Baseline at Weeks 12 and 24 on the Vineland-II Communication Domain Standard Score

  Weeks 12 and 24

Study Arms (2)

Balovaptan

EXPERIMENTAL

Drug: Balovaptan

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

Balovaptan DRUG

Participants will receive 10 mg of oral administration balovaptan once a day (QD).

Balovaptan

Placebo DRUG

Participants will receive matching placebo.

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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 28 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

You may not qualify if:

• 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
• Substance use disorders 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
• Unexplained syncopal episode within the last 12 months
• Confirmed elevation above upper limit of normal of CK-MB, high sensitivity cardiac troponin T, cardiac troponin I, and/or N-terminal pro B-type natriuretic peptide
• 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

Sponsors & Collaborators

• Hoffmann-La Roche: lead

Study Sites (51)

Harmonex Neuroscience Research

Dothan, Alabama, 36303, United States

Location

Southwest Autism Research & Resource Center

Phoenix, Arizona, 85006, United States

Location

Woodland Research Northwest, LLC

Rogers, Arkansas, 72758, United States

Location

University of California , Los Angeles (UCLA); Child, Adolescent Psychiatry

Los Angeles, California, 90095, United States

Location

PCSD Feighner Research

San Diego, California, 92108, United States

Location

University of California at San Francisco

San Francisco, California, 94115, United States

Location

MCB Clinical Research Centers

Colorado Springs, Colorado, 80910, United States

Location

Yale University / Yale-New Haven Hospital

New Haven, Connecticut, 06519-1124, United States

Location

Sarkis Clinical Trials

Gainesville, Florida, 32607, United States

Location

APG- Advanced Psychiatric Group

Orlando, Florida, 32803, United States

Location

IMIC Inc.

Palmetto Bay, Florida, 33157, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

Uni of Chicago; Centre For Advanced Medicine

Chicago, Illinois, 60637, United States

Location

Lake Charles Clinical Trials, LLC

Lake Charles, Louisiana, 70601, United States

Location

The Johns Hopkins Hospital

Baltimore, Maryland, 21287, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

University of Minnesota Medical Center-Fairview

Minneapolis, Minnesota, 55414, United States

Location

Millennium Psychiatric Associates, LLC

St Louis, Missouri, 63132, United States

Location

Hapworth Research Inc.

New York, New York, 10019, United States

Location

Center for Autism and the Developing Brain

New York, New York, 10032, United States

Location

Nathan S. Kline Institute for Psychiatric Research

Orangeburg, New York, 10962, United States

Location

Richmond Behavioral Associates

Staten Island, New York, 10312, United States

Location

University Hospitals

Cleveland, Ohio, 44106, United States

Location

Ohio State University

Columbus, Ohio, 43210, United States

Location

Cutting Edge Research Group

Oklahoma City, Oklahoma, 73116, United States

Location

UPMC Western Psychiatric Institute and Clinic

Pittsburgh, Pennsylvania, 15203, United States

Location

Vanderbilt University Medical Center; Department of Psychiatry

Nashville, Tennessee, 37212, United States

Location

BioBehavioral Research of Austin, PC

Austin, Texas, 78759, United States

Location

Red Oak Psychiatry Associates, PA

Houston, Texas, 77090, United States

Location

Aspen Clinical Research

Orem, Utah, 84058, United States

Location

Northwest Clinical Research Center

Bellevue, Washington, 98007, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

Okanagan Clinical Trials

Kelowna, British Columbia, V1Y 1Z9, Canada

Location

Holland Bloorview Kids Rehabilitation Hospital; Autism Research Centre

East York, Ontario, M4G 1R8, Canada

Location

University of Western Ontario

London, Ontario, N6A 4G5, Canada

Location

McGill University Health Centre - Glen Site

Montreal, Quebec, H4A 3J1, Canada

Location

Hopital Charles Perrens; Centre de Ressources Autisme Aquitaine

Bordeaux, 33076, France

Location

Hospices Civils de Lyon; Centre d'Investigation Clinique Pédiatrique

Lyon, 69003, France

Location

Centre hospitalier du Rouvray; CRAHN Centre de Ressources Autisme Haute-Normandie

Sotteville-lès-Rouen, 76300, France

Location

ASST di Pavia; Dip. di Scienze del Sistema Nervoso e del Comportamento

Pavia, Lombardy, 27100, Italy

Location

AUSL di Piacenza; Psichiatria di Collegamento

Piacenza, Lombardy, 29121, Italy

Location

ASL TO2; Centro Pilota Regione Piemonte - Dip. Salute Mentale

Turin, Piedmont, 10138, Italy

Location

A.O.U. Policlinico - V. Emanuele - P.O. Gaspare Rodolico; Dip. Terapia integrata disturbi resistenti

Catania, Sicily, 95123, Italy

Location

Hospital Mutua de Terrassa; Departamento de Psiquiatria

Terrassa, Barcelona, 08221, Spain

Location

Hospital Universitari Vall d'Hebron; Sevicio de Psiquiatría

Barcelona, 08035, Spain

Location

Hospital General Universitario Gregorio Marañon; Servicio de Psiquiatria del niño y del adolescente

Madrid, 28009, Spain

Location

Hospital Universitario Rio Hortega; Departamento de Psiquiatria

Valladolid, 47012, Spain

Location

Western General Hospital; Wellcome Trust CRF

Edinburgh, EH4 2XU, United Kingdom

Location

Queen Elizabeth University Hospital; Clinical Research Facility

Glasgow, G51 4TF, United Kingdom

Location

Kings College Hospital; Kings Clinical Research Facility

London, SE5 9RS, United Kingdom

Location

RE:Cognition Health; RE:Cognition Health

London, W1G 9JF, United Kingdom

Location

Related Publications (2)

• Aponte EA, Tillmann J, Gleissl T, Del Valle Rubido M, Murtagh L, Sanders K, Chatham CH, Wiese T, Suter EE. Anxiety, repetitive and restricted behaviors and interests, and social communication in autistic adults: an exploratory analysis of a phase 3, randomized clinical trial. Sci Rep. 2025 Nov 6;15(1):38912. doi: 10.1038/s41598-025-22659-y.

  PMID: 41198718 DERIVED

• Jacob S, Veenstra-VanderWeele J, Murphy D, McCracken J, Smith J, Sanders K, Meyenberg C, Wiese T, Deol-Bhullar G, Wandel C, Ashford E, Anagnostou E. Efficacy and safety of balovaptan for socialisation and communication difficulties in autistic adults in North America and Europe: a phase 3, randomised, placebo-controlled trial. Lancet Psychiatry. 2022 Mar;9(3):199-210. doi: 10.1016/S2215-0366(21)00429-6. Epub 2022 Feb 10.

  PMID: 35151410 DERIVED

MeSH Terms

Conditions

Autism Spectrum Disorder

Interventions

balovaptan

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Results Point of Contact

• Title: Reference Study ID Number: WN39434
• Organization: www.roche.com/about_roche/roche_worldwide.htm

global-roche-genentech-trials@gene.com

888-662-6728 (U.S. Only)

Study Officials

• Clinical Trials

  Hoffmann-La Roche

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 12, 2018
• First Posted: April 20, 2018
• Study Start: August 8, 2018
• Primary Completion: March 4, 2020
• Study Completion: July 1, 2020
• Last Updated: October 27, 2021
• Results First Posted: May 7, 2021

Record last verified: 2021-10

Locations

US (32); IT (4); ES (4); GB (4); FR (3); CA (4)