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

NCT ID: NCT03504917

Last Updated: 2021-10-27

Study Results

Results available

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE3

Total Enrollment

322 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-08-08

Study Completion Date

2020-07-01

Brief Summary

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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).

Detailed Description

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Conditions

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Autism Spectrum Disorder

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Balovaptan

Group Type EXPERIMENTAL

Balovaptan

Intervention Type DRUG

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

Placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Participants will receive matching placebo.

Interventions

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Balovaptan

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

Intervention Type DRUG

Placebo

Participants will receive matching placebo.

Intervention Type DRUG

Eligibility Criteria

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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 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

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
* 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
* Confirmed clinically significant abnormality in parameters of clinical chemistry, coagulation, or urinalysis
* Medical history of malignancy, if not considered cured
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Hoffmann-La Roche

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Clinical Trials

Role: STUDY_DIRECTOR

Hoffmann-La Roche

Locations

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Harmonex Neuroscience Research

Dothan, Alabama, United States

Site Status

Southwest Autism Research & Resource Center

Phoenix, Arizona, United States

Site Status

Woodland Research Northwest, LLC

Rogers, Arkansas, United States

Site Status

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

Los Angeles, California, United States

Site Status

PCSD Feighner Research

San Diego, California, United States

Site Status

University of California at San Francisco

San Francisco, California, United States

Site Status

MCB Clinical Research Centers

Colorado Springs, Colorado, United States

Site Status

Yale University / Yale-New Haven Hospital

New Haven, Connecticut, United States

Site Status

Sarkis Clinical Trials

Gainesville, Florida, United States

Site Status

APG- Advanced Psychiatric Group

Orlando, Florida, United States

Site Status

IMIC Inc.

Palmetto Bay, Florida, United States

Site Status

Rush University Medical Center

Chicago, Illinois, United States

Site Status

Uni of Chicago; Centre For Advanced Medicine

Chicago, Illinois, United States

Site Status

Lake Charles Clinical Trials, LLC

Lake Charles, Louisiana, United States

Site Status

The Johns Hopkins Hospital

Baltimore, Maryland, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

University of Minnesota Medical Center-Fairview

Minneapolis, Minnesota, United States

Site Status

Millennium Psychiatric Associates, LLC

St Louis, Missouri, United States

Site Status

Hapworth Research Inc.

New York, New York, United States

Site Status

Center for Autism and the Developing Brain

New York, New York, United States

Site Status

Nathan S. Kline Institute for Psychiatric Research

Orangeburg, New York, United States

Site Status

Richmond Behavioral Associates

Staten Island, New York, United States

Site Status

University Hospitals

Cleveland, Ohio, United States

Site Status

Ohio State University

Columbus, Ohio, United States

Site Status

Cutting Edge Research Group

Oklahoma City, Oklahoma, United States

Site Status

UPMC Western Psychiatric Institute and Clinic

Pittsburgh, Pennsylvania, United States

Site Status

Vanderbilt University Medical Center; Department of Psychiatry

Nashville, Tennessee, United States

Site Status

BioBehavioral Research of Austin, PC

Austin, Texas, United States

Site Status

Red Oak Psychiatry Associates, PA

Houston, Texas, United States

Site Status

Aspen Clinical Research

Orem, Utah, United States

Site Status

Northwest Clinical Research Center

Bellevue, Washington, United States

Site Status

Seattle Children's Hospital

Seattle, Washington, United States

Site Status

Okanagan Clinical Trials

Kelowna, British Columbia, Canada

Site Status

Holland Bloorview Kids Rehabilitation Hospital; Autism Research Centre

East York, Ontario, Canada

Site Status

University of Western Ontario

London, Ontario, Canada

Site Status

McGill University Health Centre - Glen Site

Montreal, Quebec, Canada

Site Status

Hopital Charles Perrens; Centre de Ressources Autisme Aquitaine

Bordeaux, , France

Site Status

Hospices Civils de Lyon; Centre d'Investigation Clinique Pédiatrique

Lyon, , France

Site Status

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

Sotteville-lès-Rouen, , France

Site Status

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

Pavia, Lombardy, Italy

Site Status

AUSL di Piacenza; Psichiatria di Collegamento

Piacenza, Lombardy, Italy

Site Status

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

Turin, Piedmont, Italy

Site Status

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

Catania, Sicily, Italy

Site Status

Hospital Mutua de Terrassa; Departamento de Psiquiatria

Terrassa, Barcelona, Spain

Site Status

Hospital Universitari Vall d'Hebron; Sevicio de Psiquiatría

Barcelona, , Spain

Site Status

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

Madrid, , Spain

Site Status

Hospital Universitario Rio Hortega; Departamento de Psiquiatria

Valladolid, , Spain

Site Status

Western General Hospital; Wellcome Trust CRF

Edinburgh, , United Kingdom

Site Status

Queen Elizabeth University Hospital; Clinical Research Facility

Glasgow, , United Kingdom

Site Status

Kings College Hospital; Kings Clinical Research Facility

London, , United Kingdom

Site Status

RE:Cognition Health; RE:Cognition Health

London, , United Kingdom

Site Status

Countries

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United States Canada France Italy Spain United Kingdom

References

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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.

Reference Type DERIVED
PMID: 35151410 (View on PubMed)

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

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WN39434

Identifier Type: -

Identifier Source: org_study_id