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    Summary
    EudraCT Number:2021-002011-61
    Sponsor's Protocol Code Number:77267
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-06-21
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2021-002011-61
    A.3Full title of the trial
    The glutamate/GABA balance as novel therapeutic target for psychotic and cognitive symptoms in 22q11.2 deletion syndrome
    De glutamaat/GABA balans als nieuw therapeutisch target voor psychotische en cognitieve symptomen in 22q11.2 deletie syndroom.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Riluzole as treatment for psychotic and cognitive symptoms in 22q11.2 deletion syndrome
    Riluzol als behandeling voor psychotische en cognitieve symptomen in 22q11.2 deletie syndroom.
    A.3.2Name or abbreviated title of the trial where available
    Riluzole in 22q11.2DS
    Riluzol in 22q11.2DS
    A.4.1Sponsor's protocol code number77267
    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 SponsorAcademisch ziekenhuis Maastricht
    B.1.3.4CountryNetherlands
    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 supportAcademisch ziekenhuis Maastricht
    B.4.2CountryNetherlands
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAcademisch ziekenhuis Maastricht
    B.5.2Functional name of contact pointTherese van Amelsvoort
    B.5.3 Address:
    B.5.3.1Street AddressP. Debyelaan 25
    B.5.3.2Town/ cityMAASTRICHT
    B.5.3.3Post code6229 HX
    B.5.3.4CountryNetherlands
    B.5.6E-mailt.vanamelsvoort@maastrichtuniversity.nl
    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 Rilutek
    D.2.1.1.2Name of the Marketing Authorisation holderSanofi Mature IP
    D.2.1.2Country which granted the Marketing AuthorisationNetherlands
    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 nameRiluzole
    D.3.2Product code EMEA/H/C/000109
    D.3.4Pharmaceutical form Capsule
    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
    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
    22q11.2 deletion syndrome
    22q11.2 deletie syndroom
    E.1.1.1Medical condition in easily understood language
    22q11.2DS
    22q11.2DS
    E.1.1.2Therapeutic area Psychiatry and Psychology [F] - Mental Disorders [F03]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The objective of this study is to examine the efficacy of riluzole, a modulator of the glutamate /GABA balance, for treatment of psychotic symptoms and cognitive impairment in 22q11.2DS patients.
    Het doel van deze studie is om de werkzaamheid te onderzoeken van riluzol, een glutamaat / GABA modulator, voor de behandeling van psychotische symptomen en cognitieve stoornissen bij 22q11.2DS-patiënten.
    E.2.2Secondary objectives of the trial
    The secondary objective is to examine effects of riluzole on the glutamate/GABA-balance in order to increase insight in the neurobiological underpinnings of these symptoms.
    Het secundaire doel is om de effecten van riluzol op de glutamaat / GABA-balans in de hersenen te onderzoeken om meer inzicht te krijgen in de neurobiologische mechanisme die ten grondslag liggen aan deze symptomen.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Confirmed diagnosis of 22q11.2 deletion syndrome established by FISH, microarray or MLPA analysis.
    • 16 year or older of age and mentally competent (determined by an experienced physician) to give informed consent.
    • 16 years, incompetent to provide written informed consent. In these cases consent will be obtained from the legal representative of the subject.
    • Presence of psychotic and/or cognitive symptoms (defined as a score of ≥4, moderately ill, on the Clinical Global Impression-Schizophrenia Scale (CGI-SCH)).
    Bevestigde 22q11.2 deletie syndroom diagnose middels FISH, microarray of MLPA.
    • 16 jaar of ouder en wilsbekwaam ter zake om informed consent te geven. Dit zal bepaald worden door een ervaren arts.
    • 16 jaar of ouder en wilsonbekwaam om informed consent te geven. In dit geval zal informed consent worden gegeven door de wettelijk vertegenwoordiger.
    • Aanwezigheid van psychotische en/of cognitieve symptomen (gedefinieerd als een score ≥4 op de Clinical Global Impression-Schizophrenia Scale (CGI-SCH).
    E.4Principal exclusion criteria
    - Other chromosomal abnormalities.
    - Current substance abuse / dependence.
    - Use of first-generation antipsychotics or clozapine. Use of second-generation antipsychotics is allowed.
    - Contraindications for MRI.
    - Contraindications for riluzole.
    - Pregnancy.
    • Andere chromosoom afwijkingen.
    • Huidig middelenmisbruik of afhankelijkheid.
    • Gebruik van typische antipsychotica of clozapine. Gebruik van atypische antipsychotica is toegestaan.
    • Contra-indicaties voor MRI.
    • Contra-indicaties voor riluzol.
    • Zwangerschap
    E.5 End points
    E.5.1Primary end point(s)
    The main endpoint will be the change in psychotic and cognitive symptom severity.
    De primaire uitkomstmaat is de verandering in de ernst van psychotische en cognitieve symptomen.
    E.5.1.1Timepoint(s) of evaluation of this end point
    At the end of each intervention period
    Na iedere interventie periode
    E.5.2Secondary end point(s)
    The secondary study endpoint will be the change in glutamate and GABA concentrations in the anterior cingulate cortex.
    De secundaire uitkomstmaat is de verandering in glutamaat en GABA concentraties in de anterior cingulate cortex.
    E.5.2.1Timepoint(s) of evaluation of this end point
    At the end of each intervention period
    Na iedere interventie periode
    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 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 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 Yes
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Partieel geblindeerd
    Partially blind
    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 trial1
    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 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
    LVLS
    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 months0
    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 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: 46
    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 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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    In case patients are unable to provide consent personally, consent will be obtained from their legal representative. Incapacitated subjects who show resistance to actions, will not be included according to existing guidelines.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state46
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 46
    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)
    After completing the study, participants can continue treatment (off-label) in consultation with the treating physician.
    Na het afronden van de studie kunnen deelnemers de behandeling (off-label) voortzetten in overleg met de behandelend arts.
    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 Decision2021-06-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-08-13
    P. End of Trial
    P.End of Trial StatusOngoing
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