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    Summary
    EudraCT Number:2017-002155-28
    Sponsor's Protocol Code Number:P160902J
    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:2018-06-07
    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 ConcernedFrance - ANSM
    A.2EudraCT number2017-002155-28
    A.3Full title of the trial
    Evaluation of allopurinol treatment for autistic disorders and epilepsy in adenylosuccinate lyase deficiency (ADSL)
    Evaluation d’un traitement à l’allopurinol sur les troubles autistiques et l’épilepsie dans le déficit en adénylosuccinate lyase (ADSL)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Evaluation of allopurinol treatment for autistic disorders and epilepsy in adenylosuccinate lyase deficiency (ADSL)
    Evaluation d'un traitement à l'allopurinol sur les troubles autistiques et l'épilepsie dans le déficit en adenylosuccinate lyase (ADSL)
    A.3.2Name or abbreviated title of the trial where available
    ADSL
    A.4.1Sponsor's protocol code numberP160902J
    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 SponsorASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)
    B.1.3.4CountryFrance
    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 supportDGOS
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)
    B.5.2Functional name of contact pointDRCI Hôpital St Louis
    B.5.3 Address:
    B.5.3.1Street Address 1 av. Claude Vellefaux
    B.5.3.2Town/ cityPARIS
    B.5.3.3Post code75010
    B.5.3.4CountryFrance
    B.5.6E-mailmandy.nizard@aphp.fr
    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 Zyloric 100 mg
    D.2.1.1.2Name of the Marketing Authorisation holderASPEN PHARMA TRADING LIMITED
    D.2.1.2Country which granted the Marketing AuthorisationFrance
    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 nameZyloric 100 mg
    D.3.4Pharmaceutical form 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 INNallopurinol
    D.3.9.3Other descriptive nameallopurinol
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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
    patient with adenulosuccinate lyase deficiency
    patient présentant un déficit en adénulosuccinate lyase
    E.1.1.1Medical condition in easily understood language
    patient with adenylosuccinate lyase deficiency
    patient présentant un déficit en adenylosuccinate lyase
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10062018
    E.1.2Term Inborn error of metabolism
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Evaluation of the 12-month efficacy of allopurinol treatment on adaptive and cognitive functioning in patients with adenylosuccinate lyase deficiency (ADSL)
    Evaluation de l'efficacité à 12 mois d'un traitement par l'allopurinol sur le fonctionnement adaptatif et cognitif des patients atteints du déficit en adénylosuccinate lyase (ADSL)
    E.2.2Secondary objectives of the trial
    -Evaluation of allopurinol efficacy on adaptive functioning and attention at 6 and 12 months, on cognitive functioning and autistic symptoms at 6 and 12 months.
    -Quantify the decrease in the concentration of SAICAR and S-Ado metabolites, markers of adenylosuccinate lyase (ADSL) deficiency.
    -Evaluation of the efficacy of allopurinol on seizures in epileptic patients and on EEG abnormalities.
    - Evaluation de l'efficacité de l'allopurinol sur le fonctionnement adaptatif et l'attention à 6 mois et 12 mois, sur le fonctionnement cognitif et les symptômes autistiques à 6 et 12 mois.
    - Quantifier la diminution de la concentration des métabolites SAICAR et S-Ado, marqueurs du déficit en adénylosuccinate lyase (ADSL).
    - Evaluation de l'efficacité de l'allopurinol sur les crises convulsives pour les patients épileptiques et sur les anomalies à l'EEG.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    -child (minimum age 18 months) or adult with adenylosuccinate lyase (ADSL) deficiency confirmed by quantification of SAICAr and urinary S-adenosine,
    -consent of the patient, parents or legal representative,
    -Beneficiary of social security coverage (affiliated or entitled).
    - enfant (âge minimum de 18 mois) ou adulte atteint de déficit en adenylosuccinate lyase (ADSL) confirmé par la quantification du SAICAr et S-adénosine urinaire,
    -consentement du patient, de ses parents ou de son représentant légal,
    -bénéficiaire d'une couverture sociale (affilié ou ayant droit).
    E.4Principal exclusion criteria
    -refusal by the child, his parents or his representative,
    -known allergy to allopurinol or to one of the constituents of the product (notably lactose),
    -Patients treated with Antipurines (azathioprine, mercaptopurine).
    -renal insufficiency characterized by creatine clearance < 80 mL/min/1.73m²,
    -breastfeeding,
    -pregnancy.
    - refus de l'enfant, de ses parents, ou de son représentant,
    - allergie connue à l'allopurinol ou à l'un des constituants du produit (au lactose notamment),
    - Patients traités par des Antipurines (azathioprine, mercaptopurine).
    -insuffisance rénale caractérisée par une clairance de la créatine < 80 mL/min/1,73m²,
    -allaitement,
    - grossesse.
    E.5 End points
    E.5.1Primary end point(s)
    Clinical Criterion:
    Adaptive functional improvement measured by the total composite score on the Vineland II scale. A semi-structured interview by the Vineland II scale at inclusion (M0) and M12.
    Critère clinique :
    Amélioration fonctionnelle adaptative mesurée par le score total composite de l'échelle de Vineland II. Un entretien semi-structuré par l'échelle de Vineland II à l'inclusion (M0) et M12.
    E.5.1.1Timepoint(s) of evaluation of this end point
    12 month
    12 mois
    E.5.2Secondary end point(s)
    Clinical criteria:
    Measurement of clinical progression using standardized tools for developmental and cognitive assessment, assessment of autistic symptoms, behavioural disorders and adaptive functioning at 0, 6 and 12 months according to tests:
    - Autism Diagnostic Interview (ADI-R), and Autism Diagnostic Observation Schedule (ADOS) at M0 and M12
    - Vineland II scale in different subdomains at M0, M6 and M12
    - Psycho-Educational Profile (PEP-3) or Wechsler tests at M0 and M12
    - Conners scales (parent and teacher version) and the Aberrant Behaviour Checklist (ABC) at M0, M6 and M12

    Biochemical criteria:
    Quantification of SAICAr and S-Ado urinary and plasma metabolites before (M0) and during treatment with M3, M6 and M12.

    Criteria for improving epilepsy:
    Evaluation of the number of seizures, anti-epileptic treatments and electroencephalogram (EEG) tracings for epileptic patients at M0, M6 and M12
    Critères cliniques:
    Mesure de l'évolution clinique par des outils standardisés permettant l'évaluation développementale et cognitive, l'évaluation des symptômes autistiques, des troubles du comportement et du fonctionnement adaptatif à 0, 6 mois et 12 mois selon les tests :
    - Autism Diagnostic Interview (ADI-R), et Autism Diagnostic Observation Schedule (ADOS) à M0 et M12
    - Echelle de Vineland II dans différents sous-domaines à M0, M6 et M12
    - Psycho-Educative Profile (PEP-3) ou tests de Wechsler à M0 et M12
    - Echelles de Conners (version parents et version enseignants) et l'Aberrant Behaviour Checklist (ABC) à M0, M6 et M12

    Critères biochimiques:
    Quantification des métabolites urinaire et plasmatique SAICAr et S-Ado avant (M0) et pendant le traitement à M3, M6 et M12.

    Critères d'amélioration de l'épilepsie:
    Evaluation du nombre de crises convulsives, des traitements antiépileptiques et des tracés des électroencéphalogrammes (EEG) pour les patients épileptiques à M0, M6 et M12
    E.5.2.1Timepoint(s) of evaluation of this end point
    12 month
    12 mois
    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 Yes
    E.6.6Pharmacokinetic Yes
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    cohorte
    cohort
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    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 concerned2
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days0
    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: 2
    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: 1
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 1
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 6
    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 Yes
    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
    all
    tous
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state8
    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
    aucun
    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 Decision2018-08-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-08-30
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2022-06-17
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