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    Summary
    EudraCT Number:2017-004378-32
    Sponsor's Protocol Code Number:WN39434
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2018-08-14
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedFrance - ANSM
    A.2EudraCT number2017-004378-32
    A.3Full title of the trial
    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.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Evaluate Efficacy, and Safety of Balovaptan in Adults with Autism Spectrum Disorder with a 2 Year Open-Label Extension
    A.4.1Sponsor's protocol code numberWN39434
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorF. Hoffmann-La Roche Ltd
    B.1.3.4CountrySwitzerland
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportF. Hoffmann-La Roche Ltd
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationF. Hoffmann-La Roche Ltd
    B.5.2Functional name of contact pointTrial Information Support Line-TISL
    B.5.3 Address:
    B.5.3.1Street AddressGrenzacherstrasse 124
    B.5.3.2Town/ cityBasel
    B.5.3.3Post code4070
    B.5.3.4CountrySwitzerland
    B.5.6E-mailglobal.rochegenentechtrials@roche.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameBalovaptan
    D.3.2Product code RO5285119
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNBalovaptan
    D.3.9.2Current sponsor codeRO5285119
    D.3.9.3Other descriptive namev1a(2)
    D.3.9.4EV Substance CodeSUB32013
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboFilm-coated tablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Autism Spectrum Disorder
    E.1.1.1Medical condition in easily understood language
    ASD is a neurodevelopmental disorder that impairs the ability to communicate and interact
    E.1.1.2Therapeutic area Psychiatry and Psychology [F] - Mental Disorders [F03]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10063844
    E.1.2Term Autism spectrum disorder
    E.1.2System Organ Class 10037175 - Psychiatric disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10003805
    E.1.2Term Autism
    E.1.2System Organ Class 10037175 - Psychiatric disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10034739
    E.1.2Term Pervasive developmental disorder NOS
    E.1.2System Organ Class 10037175 - Psychiatric disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the efficacy of 10 mg of balovaptan compared with placebo based on the Vineland TM-II two-domain composite (2DC) score
    E.2.2Secondary objectives of the trial
    To evaluate the efficacy of 10 mg of balovaptan compared with placebo based on the Vineland-II 2DC score, Pediatric Quality of Life Inventory TM Generic Core Scales(PedsQLTM), Version 4.0, summary and total scores, Vineland-II composite standard score, Vineland-II Socialization, Communication, Daily Living Skills domain standard scores, Clinical Global Impressions(CGI) –Severity(S) and Improvement(I), Hamilton Anxiety Rating Scale (HAM-A) total and domain scores
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Males or females, age 18 years or older
    - Subject meets the Diagnostic and Statistical Manual of Mental Disorders, Version 5 (DSM-5) criteria for ASD for an autism diagnosis and is confirmed using Autism Diagnostic Observation Schedule (ADOS)-2 criteria
    - Social Responsiveness Scale (SRS)-2, proxy version, total t score >=66 at screening
    - A full scale IQ score >=70 on the Wechsler Abbreviated Scale of Intelligence®-II
    - Ability and willingness to fully comply with study visit schedule and regular assessments and fluency in the language of the site
    - Subject’s participation in the study or discontinuation of prohibited medication will not pose undue risks to the subject, in the investigator’s opinion
    - 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 and behavioral therapy regimens, with the intent that such treatments remain stable throughout the study and with no expected changes before the Week 24 visit
    E.4Principal exclusion criteria
    General Exclusion Criteria
    - Pregnancy or breastfeeding, or intention to become pregnant during the study
    Neurologic and Psychiatric Exclusion Criteria
    - 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, as defined using the DSM-5 criteria
    - 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
    Exclusions Related to Cardiovascular Disorders
    - Within the last 2 years, unstable or clinically significant cardiovascular disease
    - Uncontrolled hypertension
    Exclusions Related to Other Organ Systems
    - 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, 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, clinical chemistry, coagulation, or urinalysis
    - Medical history of malignancy, if not considered cured
    Additional Exclusion Criteria
    - Allowed medications have not been stable for 12 weeks prior to screening
    - Previous treatment with prohibited medications or herbal remedies within 2 weeks prior to randomization or 5 half-lives
    - Blood donation or loss of blood > 500 mL within 3 months prior to randomization
    - Previous participation in an investigational drug or device study within 60 days prior to randomization or previous enrollment in investigational trials of balovaptan
    E.5 End points
    E.5.1Primary end point(s)
    1. Change from baseline at Week 24 on the Vineland TM -II 2DC score
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. Baseline (Day 1) and Week 24
    E.5.2Secondary end point(s)
    1. Change from baseline at Week 12 on the Vineland-II 2DC score
    2. Change from baseline at Weeks 12 and 24 in the PedsQLTM Core module, Version 4.0, on summary and total scores
    3. Change from baseline at Weeks 12 and 24 in the Vineland-II a composite standard score
    4. Change from baseline at Weeks 12 and 24 in Vineland-II Socialization domain standard score
    5. Change from baseline at Weeks 12 and 24 in Vineland-II Communication domain standard score
    6. Change from baseline at Weeks 12 and 24 in Vineland-II Daily Living Skills domain standard score
    7. Change from baseline in severity of clinical impressions as measured by CGI-S after 12 weeks and 24 weeks of treatment
    8. Improvements in clinical impressions, as measured by CGI-I after 12 weeks and 24 weeks of treatment
    9. Change from baseline in the HAM-A total and domain scores at Weeks 12 and 24
    10. Proportion of subjects with a >=6-point improvement in Vineland-II 2DC score at Weeks 12 and 24
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Baseline and Week 12
    2-6. Baseline, Week 12 and Week 24
    7-8. Week 12 and Week 24
    9-10. Baseline, Week 12 and Week 24
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    2 year open label extension
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA20
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Canada
    France
    Germany
    Italy
    Spain
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    The date when the last subject, last visit occurs in the open-label extension period or the follow-up period.
    It is anticipated that it may take from 18 to 28 months to recruit all subjects for the study. Hence, the total length of the study is estimated to be approximately 4-5 years.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months1
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months7
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 349
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others Yes
    F.3.3.7.1Details of other specific vulnerable populations
    Patients aged 65 or above are not expected to be recruited but will not be excluded if eligible
    F.4 Planned number of subjects to be included
    F.4.1In the member state12
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 70
    F.4.2.2In the whole clinical trial 350
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    There are no plans to provide the Roche IMP (balovaptan) or any other study treatments or interventions to subjects who have completed the study.
    The Sponsor may evaluate whether to continue providing balovaptan in accordance with the Roche Global Policy on Continued Access to IMPs, available at: http://www.roche.com/policy_continued_access_to_investigational_medicines.pdf
    Subjects are eligible to receive balovaptan as part of the OLE, provided they have completed 24 weeks of treatment
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2018-10-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-04-09
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2020-07-01
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