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    Summary
    EudraCT Number:2021-003851-41
    Sponsor's Protocol Code Number:Bumetanide/Autism
    National Competent Authority:Sweden - MPA
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-09-09
    Trial 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 ConcernedSweden - MPA
    A.2EudraCT number2021-003851-41
    A.3Full title of the trial
    A Randomized Waitlist-Control Trial with Bumetanide in Children with Autism
    En Väntelist-kontrollerad Behandlingsstudie med Bumetanide till Barn med Autism
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Treatment Study with Bumetanide (a diuretic) aimed for Children with Autism
    En Behandlingsstudie med Bumetanide (ett diuretikum) riktad till Barn med Autism
    A.4.1Sponsor's protocol code numberBumetanide/Autism
    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 SponsorGillberg Neuropsychiatry Centre
    B.1.3.4CountrySweden
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportGillberg Neuropsychiatry Centre
    B.4.2CountrySweden
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGillberg Neuropsychiatry Centre
    B.5.2Functional name of contact pointGNC
    B.5.3 Address:
    B.5.3.1Street AddressKungsgatan 12
    B.5.3.2Town/ cityGothenburg
    B.5.3.3Post code411 19
    B.5.3.4CountrySweden
    B.5.4Telephone number00460313435970
    B.5.6E-mailchristopher.gillberg@gnc.gu.se
    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 Yes
    D.2.1.1.1Trade name Burinex
    D.2.1.1.2Name of the Marketing Authorisation holderKaro Pharma
    D.2.1.2Country which granted the Marketing AuthorisationSweden
    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 nameBurinex
    D.3.2Product code 00289/0322
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Autism spectrum disorder, a neurodevelopmental disorder with onset in childhood.
    The disorder implies deficits in social and communicative abilities and restricted interests and behaviors. Many children with autism also have other coexisting disorders, such as intellectual disability.
    Autism, en utvecklingsneurologisk funktionsnedsättning, som innebär nedsatt social och kommunikativ förmåga samt begränsade beteenden och intressen. Många barn med autism har samtidigt andra funktionsnedsättningar som intellektuell funktionsnedsättning.
    E.1.1.1Medical condition in easily understood language
    Autism is a neurodevelopmental disorder that affects about one percent of children.
    Symptoms may be apparent during the first years of life, but sometimes symptoms are not evident until school age.
    Autism är en utvecklingsneurologisk funktionsnedsättning som innefattar brister i social och kommunikativ förmåga samt begränsade beteende och intressen. Autism förekommer hos drygt 1% av barn.
    E.1.1.2Therapeutic area Psychiatry and Psychology [F] - Behaviours [F01]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To analyse the effect of Bumetanide on social and communicative abilities in children with autism spectrum disorder.
    Att studera effekten av Bumetanide på social och kommunikativ förmåga hos barn med autism.
    E.2.2Secondary objectives of the trial
    To test the safety and tolerability of the drug in children with autism spectrum disorder.
    Att testa säkerhet och tolerans för läkemedlet hos barn med autism.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male and female patients 4-12 years of age with autism of at least moderate level, diagnosed according to DSM-IV-TR or DSM-5. The children may have coexisting moderate or severe intellectual disability and/or coexisting epilepsy. Any identified etiology of autism is allowed.
    2. Age 4-12 years
    3. Ability to comply with the treatment, blood sampling and scheduled follow-up visits and examinations and normal pre-treatment blood tests and ECG.
    4. Written informed consent from parents (and assent for children, those with an age above 6 years)
    1. Pojkar och flickor i ålder 4-12 år med autism av en åtminstone måttlig grad, diagnostiserad i enlighet med DSM-IV-TR eller DSM-5. Barnen kan ha samexisterande måttlig och svår intellektuell funktionsnedsättning och/eller epilepsi. Olika etiologier till autism kan ingå.
    2. Ålder 4-12 år
    3. Möjlighet att klara av behandlingen, blodprovstagningar och schemalagda uppföljningsbesök i och undersökningar i studien och normala blodprover och EKG innan behandlingsstart.
    4. Skriftligt medgivande från föräldrar och medgivande från barn, i ålder över 6 år.
    E.4Principal exclusion criteria
    1. Clinical evidence of any significant acute or chronic disease that, in the opinion of the
    Investigator, may interfere with successful completion of the trial or place the subject at
    undue medical risk.
    2. Diabetes mellitus type 1 is an example of an exclusion criteria.
    3. The subject has Screening Visit values of aspartate aminotransferase (ASAT), alanine
    aminotransferase (ALAT)or creatinine not within the normal reference values for children.
    4. The subject has not a normal QT-interval measured on ECG.
    5. The subject/caregiver is unwilling to comply with any aspect of the protocol, including
    blood sampling.
    1. Exklusionskriterier är kronisk medicinsk sjukdom, som kan påverka behandlingen.
    2. Diabetes mellitus typ 1 är ett sådant exklusionskriterium.
    3. Avvikande blodvärden avseende lever och njurprover vid screeningbesöket.
    4. Avvikand fynd vid EKG, som lång QT-tid.
    5. Förälder/vårdnadshavare/patient har svårigheter att följa studieprotokollet.
    E.5 End points
    E.5.1Primary end point(s)
    Our primary outcome measure is the PASS scale (PArental Satisfaction Survey), filled in by parents after 3 months and after 6 months. Comparisons between treated and untreated patients.
    Vårt primära utfallsmått är PASS skalan (PArental Satisfaction Survey) ifylld av föräldrar efter 3 månader och efter 6 månader. Jämförelser mellan behandlade och icke behandlade barn.
    E.5.1.1Timepoint(s) of evaluation of this end point
    After 3 months and after 6 months
    Efter 3 månader och efter 6 månader
    E.5.2Secondary end point(s)
    Ratings by teachers according to the PASS scale (PArental Satisfaction Survey)
    Parental ratings according to PASS, other than primary evaluations
    The investigators assessment of CGI-S and CGI-I (Clinical Global Impression- Severity and Clinical Global Impression-Improvement)
    The ABAS-questionnaire (Adaptive Behavior Assessment Scale)
    Lärarskattning enligt PASS (PArental Satisfaction Survey)
    Föräldraskattning enligt PASS, andra än i de primära skattningarna
    Undersökarens skattningar enligt CGI-S and CGI-I (Clinical Global Impression- Severity and Clinical Global Impression-Improvement)
    Formuläret ABAS (Adaptive Behavior Assessment Scale)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Ratings by teachers according to the PASS scale after 3 and 6 months
    The investigators assessment of CGI-S and CGI-I (Clinical Global Impression- Severity and Clinical Global Impression- Improvement) at baseline (CGI-S) and CGI-S and CGI-I after 3, 6, and 9 months
    ABAS-questionnaire at baseline and at 9 months follow-up
    Parental ratings according to PASS at 6 and 9 months (within groups)
    Lärarskattningar (PASS) efter 3 månader och 6 månader
    Undersökarens skattningar CGI-S och CGI-I (Clinical Global Impression- Severity and Clinical Global Impression- Improvement) vid baslinjen (CGI-S) och vid 3, 6 och 9 månader
    ABAS vid baslinjen och vid 9 månadersuppföljningen
    Föräldraskattning enligt PASS efter 6 månader och 9 månader (inom gruppen)
    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 Yes
    E.6.4Safety Yes
    E.6.5Efficacy No
    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 Yes
    E.6.13.1Other scope of the trial description
    To study the effect of Bumetanide on social and communicative functioning in children with autism spectrum disorder
    Att studera effekten av Bumetanide på social och kommunikativ förmåga hos barn med autism
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    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 Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over Yes
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    väntelistkontrollerad studie
    waillist control
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee No
    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
    LVLS
    senaste besök för sista studiepatienten
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 30
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 25
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 5
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    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 No
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    If below the age of 6
    Children will intellectual disability
    Barn under 6 år kan ej själva ge medgivande till deltagande
    Barn med intellektuell funktionsnedsättning
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state30
    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)
    None
    Nej
    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 Decision2021-11-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-08-10
    P. End of Trial
    P.End of Trial StatusOngoing
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