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    Summary
    EudraCT Number:2017-000825-11
    Sponsor's Protocol Code Number:P160914J
    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:2018-06-06
    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 number2017-000825-11
    A.3Full title of the trial
    Lithium effect in patients with Autism Spectrum Disorder and Phelan-McDermid Syndrome (SHANK3 haploinsuffisance): pilot study.
    Effet du Lithium chez les patients ayant un Trouble du Spectre Autistique et Syndrome de Phelan-McDermid (SHANK3 haploinsuffisance) : étude pilote.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Effect of lithium on autistic children
    Effet du lithium sur enfants autistes
    A.3.2Name or abbreviated title of the trial where available
    LISPHEM
    A.4.1Sponsor's protocol code numberP160914J
    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-maillaura.blanchet@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 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 namecarbonate de lithium 62,5mg
    D.3.4Pharmaceutical form Capsule
    D.3.4.1Specific paediatric formulation Yes
    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 INNcarbonate de lithium
    D.3.9.1CAS number 554-13-2
    D.3.9.2Current sponsor code5005635
    D.3.9.3Other descriptive namelithium carbonate
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number250
    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 Yes
    D.IMP: 2
    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 namecarbonate de lithium 125mg
    D.3.4Pharmaceutical form Capsule
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    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 INNCarbonate de Lithium
    D.3.9.1CAS number 554-13-2
    D.3.9.2Current sponsor code5005635
    D.3.9.3Other descriptive nameLITHIUM CARBONATE
    D.3.9.4EV Substance CodeSUB14375MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number to 250
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.13.1Other medicinal product typePlacebo
    D.IMP: 3
    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 namecarbonate de Lithium 250mg
    D.3.4Pharmaceutical form Capsule
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    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 INNcarbonate de lithium
    D.3.9.1CAS number 554-13-2
    D.3.9.2Current sponsor code5005635
    D.3.9.3Other descriptive nameLITHIUM CARBONATE
    D.3.9.4EV Substance CodeSUB14375MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number to 250
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.13.1Other medicinal product typePlacebo
    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 placeboCapsule
    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
    Autistic Spectrum Disorder and Phelan-McDermid Syndrome
    Trouble du Spectre Autistique et Syndrome de Phelan-McDermid
    E.1.1.1Medical condition in easily understood language
    Autism
    Autisme
    E.1.1.2Therapeutic area Psychiatry and Psychology [F] - Behaviours [F01]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10063844
    E.1.2Term Autism spectrum disorder
    E.1.2System Organ Class 10037175 - Psychiatric disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the effect of Li + at 12 weeks as a new treatment for social communication deficit in patients with Phelan-McDermid Syndrome (PMS)
    Evaluer l'effet du Li + à 12 semaines comme un nouveau traitement pour le déficit de communication sociale chez les patients souffrant de Syndrome de Phelan-McDermid Syndrome (SPM).
    E.2.2Secondary objectives of the trial
    - To evaluate the effect of Li + at 12 weeks on all cardinal and main symptoms in patients with Phelan-McDemid Syndrome (PMS).
    - To evaluate the tolerance of Li + for 12 weeks in children with PMS.
    - Demonstrate the feasibility of a randomized controlled phase III study
    -Evaluer l'effet du Li + à 12 semaines sur l'ensemble des symptômes cardinaux et principaux chez les patients souffrant de Syndrome de Phelan-McDemid (SPM).
    -Evaluer la tolérance du Li + pendant 12 semaines chez les enfants souffrant de SPM.
    -Démontrer la faisabilité d'un projet de phase III, contrôlé randomisé
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria

    -Children aged 7-17 years and 8 months;
    Nosological criteria:
    -Patient with haplo SHANK3 deficiency, ie carrying a deletion (CNV) SHANK3 or a de novo truncating mutation in SHANK3 (Phelan McDermid syndrome);
    -Total score to Social Responsiveness Scale - T score (SRS) of at least 66
    -Affiliation to a social security scheme (no MEA)
    -Consentement of the holders of the parental authority
    -Non-participation in another clinical trial

    -Enfants âgés de 7-17 ans et 8 mois;
    -Critères nosologiques :
    -Patient avec une haplo insuffisance SHANK3, à savoir porteur d'une délétion (CNV) SHANK3 ou d'une mutation de novo tronquante dans SHANK3 (syndrome de Phelan McDermid) ;
    -Score total à la Social Responsiveness Scale - T score (SRS) d'au moins 66
    -Affiliation à un régime de sécurité sociale (pas d'AME)
    -Consentement des titulaires de l'autorité parentale
    -Non-participation à un autre essai clinique
    E.4Principal exclusion criteria

    Hepatic or renal insufficiency (disturbed hepatic assessment, abnormal creatinine clearance);
    - Thyroid or unbalanced diabetic pathology;
    - Cardiac pathology: Brugada syndrome or family history of Brugada syndrome, heart failure;
    -Maladie of Addison;
    -Non-stabilized epileptic disease.
    Criteria for contraindications to any of the treatments studied:
    -Patient with concomitant diseases considered for which experimental treatment with Li + could compromise tolerance;
    -A history of allergy to Li +;
    -Lactose allergy, lactose being the only diluent and excipient of the prepared form
    -Start of a co-occurring cognitive-behavioral therapy that is specifically focused on autistic symptoms in the 6 weeks prior to inclusion;
    -Any introduction of psychotropic molecules in the 2 weeks preceding the test, including neuroleptics, monoamine oxidase inhibitors, stimulants, antidepressants. For delayed neuroleptics and Fluoxetine, this time should be 4 weeks prior to the study;
    - Serious behavioral problem or refusal to take medication that does not allow medication compliance;
    -Impossibility to practice blood tests to check the lithemia when the patient is included.

    -Insuffisance hépatique ou rénale (bilan hépatique perturbé, clairance de la créatinine anormale) ;
    -Pathologie thyroïdienne ou diabétique non équilibrée ;
    -Pathologie cardiaque : syndrome de Brugada ou antécédent familial de syndrome de Brugada, insuffisance cardiaque ;
    -Maladie d'Addison ;
    -Maladie épileptique non stabilisée.
    Critères relatifs aux contre-indications à l'un ou l'autre des traitements étudiés :
    -Patient ayant des maladies concomitantes jugées pour lequel le traitement expérimental avec Li + pourrait compromettre la tolérance ;
    -Antécédents d'allergie à Li + ;
    -Allergie au lactose, le lactose étant le diluant et excipient unique de la forme préparée
    -Début d'une thérapie cognitivo-comportementale co-occurrente qui est spécifiquement axée sur les symptômes autistiques dans les 6 semaines précédant l'inclusion ;
    -Toute introduction de molécules psychotropes dans les 2 semaines précédant l'essai, y compris les neuroleptiques, les inhibiteurs de la monoamine oxydase, les stimulants, les antidépresseurs. Pour les neuroleptiques retard et la Fluoxetine, ce délai devra être de 4 semaines précédant l'essai ;
    -Trouble grave du comportement ou refus de prendre le traitement médicamenteux ne permettant pas l'observance médicamenteuse ;
    -Impossibilité de pratiquer les prises de sang permettant de vérifier la lithémie lorsque le patient est inclus.
    E.5 End points
    E.5.1Primary end point(s)
    Main Criterion: Severity of Autistic Symptoms - Social Responsiveness Scale - Total score at 12 weeks (SRS, Constantino 2003).
    Secondary evaluation criteria:
    -Scores at scales exploring aberrant, stereotyped, repetitive and obsessive behaviors, and comorbidities.
    -Scores at the scales evaluating the presence of treatment-related adverse effects.
    -Precision of the efficiency hypothesis for calculating the number of patients and validation of the placebo for blind evaluation.
    Critère principal : Sévérité des Symptômes autistiques - Social Responsiveness Scale - Score total à 12 semaines (SRS; Constantino 2003).

    Critères d'évaluation secondaire :
    -Scores aux échelles explorant les comportements aberrants, stéréotypés, répétitifs et obsessionnels, et les comorbidités.
    -Scores aux échelles évaluant la présence d'effets indésirables liés au traitement.
    -Précision de l'hypothèse d'efficacité pour calcul du nombre de patients et validation du placebo pour évaluation en aveugle.
    E.5.1.1Timepoint(s) of evaluation of this end point
    12 weeks
    12 semaines
    E.5.2Secondary end point(s)
    Secondary evaluation criteria:
    -Scores at scales exploring aberrant, stereotyped, repetitive and obsessive behaviors, and comorbidities.
    -Scores at the scales evaluating the presence of treatment-related adverse effects.
    -Precision of the efficiency hypothesis for calculating the number of patients and validation of the placebo for blind evaluation.
    Critères d'évaluation secondaire :
    -Scores aux échelles explorant les comportements aberrants, stéréotypés, répétitifs et obsessionnels, et les comorbidités.
    -Scores aux échelles évaluant la présence d'effets indésirables liés au traitement.
    -Précision de l'hypothèse d'efficacité pour calcul du nombre de patients et validation du placebo pour évaluation en aveugle.
    E.5.2.1Timepoint(s) of evaluation of this end point
    12 weeks
    12 semaines
    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 No
    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 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 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 No
    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 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
    DVDP
    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 months4
    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: 40
    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: 20
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 20
    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 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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state22
    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)
    Current care
    Soins courants
    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 Decision2020-04-08
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2024-08-26
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