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Autism Spectrum DisorderPhase 3NCT03504917

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

A Phase 3 study testing Balovaptan in people with Autism Spectrum Disorder, run by Hoffmann-La Roche at 51 sites in 6 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial 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). Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Autism Spectrum Disorder
What is being tested?
Balovaptan
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
322 participants
Where
51 sitesin 6 countries
Study dates
8 Aug 2018to 1 Jul 2020
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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
Show all 17Show fewer
  • 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

Trial locations and contacts

51 sites in 6 countries.

51 sites in 6 countries

United States32 sites

  • BioBehavioral Research of Austin, PC

    Austin, Texas, 78759

  • The Johns Hopkins Hospital

    Baltimore, Maryland, 21287

  • Northwest Clinical Research Center

    Bellevue, Washington, 98007

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114

  • Rush University Medical Center

    Chicago, Illinois, 60612

  • Uni of Chicago; Centre For Advanced Medicine

    Chicago, Illinois, 60637

  • University Hospitals

    Cleveland, Ohio, 44106

  • MCB Clinical Research Centers

    Colorado Springs, Colorado, 80910

  • Ohio State University

    Columbus, Ohio, 43210

  • Harmonex Neuroscience Research

    Dothan, Alabama, 36303

  • Sarkis Clinical Trials

    Gainesville, Florida, 32607

  • Red Oak Psychiatry Associates, PA

    Houston, Texas, 77090

  • Lake Charles Clinical Trials, LLC

    Lake Charles, Louisiana, 70601

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

    Los Angeles, California, 90095

  • University of Minnesota Medical Center-Fairview

    Minneapolis, Minnesota, 55414

  • Vanderbilt University Medical Center; Department of Psychiatry

    Nashville, Tennessee, 37212

  • Yale University / Yale-New Haven Hospital

    New Haven, Connecticut, 06519-1124

  • Hapworth Research Inc.

    New York, New York, 10019

  • Center for Autism and the Developing Brain

    New York, New York, 10032

  • Cutting Edge Research Group

    Oklahoma City, Oklahoma, 73116

  • Nathan S. Kline Institute for Psychiatric Research

    Orangeburg, New York, 10962

  • Aspen Clinical Research

    Orem, Utah, 84058

  • APG- Advanced Psychiatric Group

    Orlando, Florida, 32803

  • IMIC Inc.

    Palmetto Bay, Florida, 33157

  • Southwest Autism Research & Resource Center

    Phoenix, Arizona, 85006

  • UPMC Western Psychiatric Institute and Clinic

    Pittsburgh, Pennsylvania, 15203

  • Woodland Research Northwest, LLC

    Rogers, Arkansas, 72758

  • PCSD Feighner Research

    San Diego, California, 92108

  • University of California at San Francisco

    San Francisco, California, 94115

  • Seattle Children's Hospital

    Seattle, Washington, 98105

  • Millennium Psychiatric Associates, LLC

    St Louis, Missouri, 63132

  • Richmond Behavioral Associates

    Staten Island, New York, 10312

Other trials in United States

Canada4 sites

  • Holland Bloorview Kids Rehabilitation Hospital; Autism Research Centre

    East York, Ontario, M4G 1R8

  • Okanagan Clinical Trials

    Kelowna, British Columbia, V1Y 1Z9

  • University of Western Ontario

    London, Ontario, N6A 4G5

  • McGill University Health Centre - Glen Site

    Montreal, Quebec, H4A 3J1

Other trials in Canada

Italy4 sites

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

    Catania, Sicily, 95123

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

    Pavia, Lombardy, 27100

  • AUSL di Piacenza; Psichiatria di Collegamento

    Piacenza, Lombardy, 29121

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

    Turin, Piedmont, 10138

Other trials in Italy

Spain4 sites

  • Hospital Universitari Vall d'Hebron; Sevicio de Psiquiatría

    Barcelona, 08035

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

    Madrid, 28009

  • Hospital Mutua de Terrassa; Departamento de Psiquiatria

    Terrassa, Barcelona, 08221

  • Hospital Universitario Rio Hortega; Departamento de Psiquiatria

    Valladolid, 47012

Other trials in Spain

United Kingdom4 sites

  • Western General Hospital; Wellcome Trust CRF

    Edinburgh, EH4 2XU

  • Queen Elizabeth University Hospital; Clinical Research Facility

    Glasgow, G51 4TF

  • Kings College Hospital; Kings Clinical Research Facility

    London, SE5 9RS

  • RE:Cognition Health; RE:Cognition Health

    London, W1G 9JF

Other trials in United Kingdom

France3 sites

  • Hopital Charles Perrens; Centre de Ressources Autisme Aquitaine

    Bordeaux, 33076

  • Hospices Civils de Lyon; Centre d'Investigation Clinique Pédiatrique

    Lyon, 69003

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

    Sotteville-lès-Rouen, 76300

Other trials in France

About this study

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

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).

Being tested
Balovaptan, Placebo
Medical subject headings
Autism Spectrum Disorder

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • BalovaptanDrug

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

  • PlaceboDrug

    Participants will receive matching placebo.

Study groups in NCT03504917
Study groupRoleWhat they receive
BalovaptanExperimentalNot described
Receives: Drug: Balovaptan
PlaceboPlacebo comparatorNot described
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

    Measured: Week 24

Secondary outcomes (11)

  1. Change From Baseline at Week 12 on the Vineland-II 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.

    Measured: Week 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

    The Pediatric Quality of Life Inventory PedsQL™4.0 Generic Core Scale assessment consists of a 23 item questionnaire encompassing 4 core scale domains: Physical Functioning (8 items); Emotional Functioning (5 items); Social Functioning (5 items); and School Functioning (5 items). Items are scored on a 5 point Likert-type response scale (0=never a problem; 1=almost never a problem; 2=sometimes a problem; 3=often a problem; and 4=almost always a problem). Once scored, items will be reverse scored and linearly transformed to a 0-100 scale (0=100, 1=75, 2=50, 3=25, 4=0), so that higher scores indicate better health-related quality of life.

    Measured: Weeks 12 and 24

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

    The Vineland-II is an instrument that measures communication, daily living skills, socialization, motor skills (only in children up to 6 years) and maladaptive (not assessed in this study) behavior of individuals with developmental disabilities. The Survey Interview Form (i.e., semi -structured interview) 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. Domain scores will be obtained for the individual domains of Socialization, Communication, Daily Living Skills, and motor skills (up to 6 years only) and used to calculate the Vineland-II Adaptive Behavior Composite score. Standardized scores on the Adaptive behavior composite range from 20-160 with higher scores indicating better functioning. Only descriptive statistics presented instead of the planned estimated due to the early discontinuation of the study due to futility.

    Measured: Weeks 12 and 24

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

    The Vineland-II is an instrument that measures communication, daily living skills, socialization, motor skills (only in children up to 6 years) and maladaptive (not assessed in this study) behavior of individuals with developmental disabilities. The Survey Interview Form (i.e., semi -structured interview) 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. Domain scores will be obtained for the individual domains of Socialization, Communication, Daily Living Skills, and motor skills (up to 6 years only) and used to calculate the Vineland-II Adaptive Behavior Composite score. Standardized scores on the Adaptive behavior composite range from 20-160 with higher scores indicating better functioning.

    Only descriptive statistics presented instead of the planned estimand due to the early discontinuation of the study due to futility.

    Measured: Baseline, Weeks 12 and 24

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

    The Vineland-II is an instrument that measures communication, daily living skills, socialization, motor skills (only in children up to 6 years) and maladaptive (not assessed in this study) behavior of individuals with developmental disabilities. The Survey Interview Form (i.e., semi -structured interview) 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. Domain scores will be obtained for the individual domains of Socialization, Communication, Daily Living Skills, and motor skills (up to 6 years only) and used to calculate the Vineland-II Adaptive Behavior Composite score. Standardized scores on the Adaptive behavior composite range from 20-160 with higher scores indicating better functioning.

    Measured: Weeks 12 and 24

  6. Change From Baseline at Weeks 12 and 24 on the Vineland-II Daily Living Skills Domain Standard Score

    The Vineland-II is an instrument that measures communication, daily living skills, socialization, motor skills (only in children up to 6 years) and maladaptive (not assessed in this study) behavior of individuals with developmental disabilities. The Survey Interview Form (i.e., semi -structured interview) 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. Domain scores will be obtained for the individual domains of Socialization, Communication, Daily Living Skills, and motor skills (up to 6 years only) and used to calculate the Vineland-II Adaptive Behavior Composite score. Standardized scores on the Adaptive behavior composite range from 20-160 with higher scores indicating better functioning.

    Only descriptive statistics presented instead of the planned estimated due to the early discontinuation of the study due to futility.

    Measured: Weeks 12 and 24

  7. Change From Baseline in Severity of Clinical Impressions as Measured by Clinical Global Impression-Severity (CGI-S)

    The CGI-S reflects the rater's impression of the subject's current autism severity on a 7-point scale ranging from no symptoms (1) to very severe symptoms (7). Changes in CGI-S score were calculated as increase or decrease in absolute CGI-S scores between Baseline and Weeks 12 and 24. Percentage of participants reported for each change in score from baseline.

    Measured: Weeks 12 and 24

  8. Improvements in Clinical Impressions, as Measured by Clinical Global Impression-Improvement (CGI-I)

    This is a 7-point Likert scale that assesses improvement of the patient's condition. Scores range from the worst score of 7 (Very much worse) to the best score of 1 (Very much improved). Lower scores are better on this scale, and indicate greater improvement. Percentage of participants reported for each score.

    Measured: Weeks 12 and 24

  9. Change From Baseline at Weeks 12 and 24 in the Hamilton Anxiety Rating Scale (HAM-A) Total and Domain Scores

    The HAM-A is a 14-item, rater administered interview, assessing the severity of anxiety symptoms during the past 7 days. Seven items assess psychic anxiety and seven assess somatic anxiety. Each item utilizes a 5-point symptom severity response scale, ranging from none (0) to very severe (4). A total score is calculated that ranges from 0 to 56; higher scores are indicative of more severe anxiety.

    Measured: Weeks 12 and 24

  10. Proportion of Subjects With a >=6-point Improvement in Vineland-II 2DC Score

    The Vineland-II is an instrument that measures communication, daily living skills, socialization, motor skills (only in children up to 6 years) and maladaptive (not assessed in this study) behavior of individuals with developmental disabilities. The Survey Interview Form (i.e., semi -structured interview) 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. Domain scores will be obtained for the individual domains of Socialization, Communication, Daily Living Skills, and motor skills (up to 6 years only) and used to calculate the Vineland-II Adaptive Behavior Composite score. Standardized scores on the Adaptive behavior composite range from 20-160 with higher scores indicating better functioning

    All participants who have an improvement of at least 6 points are included in the >=6 score threshold

    Measured: Weeks 12 and 24

  11. Percentage of Participants With Adverse Events

    According to the ICH guideline for Good Clinical Practice, an adverse event is any untoward medical occurrence in a clinical investigation subject administered a pharmaceutical product, regardless of causal attribution.

    The Blinded Treatment Period continued for 24 weeks, Open Label Extension (OLE) Treatment Period continued up to 2 years. The study was pre-maturely terminated, therefore did not reach the planned end date.

    Measured: Week 24 and Up to Approximately 2 Years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted20 Apr 2018
  2. Study start8 Aug 2018
  3. Primary completion4 Mar 2020
  4. Study completion1 Jul 2020

Why it stopped: A futility analysis assessed that the study is highly unlikely to meet the pre-defined primary objective of the study. No new safety concerns were identified.

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Double blind
Who is masked
Participant, Investigator
Enrolment
322 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03504917
Sponsor's protocol ID
WN39434
Status last verified
Oct 2021
First submitted
12 Apr 2018
Last updated
27 Oct 2021
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • 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.

    PubMed 41198718

  • 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.

    PubMed 35151410

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Balovaptan in people with Autism Spectrum Disorder, run by Hoffmann-La Roche at 51 sites in 6 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 27 Oct 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 51 sites in United States, Canada, Italy, Spain, United Kingdom and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 7 May 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change From Baseline at Week 24 on the Vineland Adaptive Behavior Scales (Vineland-II) Two-domain Composite (2DC) Score. (time frame: Week 24). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03504917, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Oct 2021.

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