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    Summary
    EudraCT Number:2014-000178-20
    Sponsor's Protocol Code Number:MCT8-2014-1
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2015-10-16
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2014-000178-20
    A.3Full title of the trial
    Thyroid hormone analog therapy of patients with severe psychomotor retardation caused by mutations in the MCT8 thyroid hormone transporter: The Triac Trial.
    Terapia sostitutiva ormonale in pazienti con grave ritardo psicomotorio causato da una mutazione del trasportatore MCT8: studio TRIAC.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Therapy of MCT8 patients with the thyroid hormone analog Triac.
    Terapia sostitutiva ormonale in pazienti con mutazione MCT8.
    A.3.2Name or abbreviated title of the trial where available
    Triac Trial in MCT8 patients
    Studio Triac in pazienti con MCT8
    A.4.1Sponsor's protocol code numberMCT8-2014-1
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02060474
    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 SponsorErasmus Medical Centre
    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 supportZonMw
    B.4.2CountryNetherlands
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationErasmus Medical Centre
    B.5.2Functional name of contact pointInformation Triac Trial
    B.5.3 Address:
    B.5.3.1Street AddressDr. Molenwaterplein 50
    B.5.3.2Town/ cityRotterdam
    B.5.3.3Post code3015 GE
    B.5.3.4CountryNetherlands
    B.5.6E-mails.groeneweg@erasmusmc.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 Téatrois (Tiratricol)
    D.2.1.1.2Name of the Marketing Authorisation holderLaboratoires Théranol-Deglaude
    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 nameTéatrois
    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 No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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
    This therapuetical trial will be conducted in patient with the Allan-Herndon-Dudley Syndrome (AHDS), casued by mutations in the thyroid hormone transporter MCT8. This results in the characteristic clinical phenotype of severe psychomotor retardation due to local hypothyroidism in the brain, in combination with high serum T3 and high normal serum TSH levels that lead to local hyperthyroidism in tissues that do not dependent on MCT8, resulting in tachycardia, low body weight and muscle wasting.
    Lo studio sarà condotto su pazienti con la sindrome di Allan-Herndon-Dudley (AHDS) dovuta a mutazione del trasportatore degli ormoni tiroidei MCT8. Il fenotipo clinico della AHDS è caratterizzato da un severo ritardo psicomotorio dovuto al deficit cerebrale di ormoni tiroidei. Ciò si associa alti livelli serici di T3 e normali livelli di TSH tali da determinare una situazione di disfunzione tissutale periferica degli ormoni tiroidei con conseguente tachicardia, basso peso, atrofia muscolare.
    E.1.1.1Medical condition in easily understood language
    Patients with a mutation in the thyroid hormone transporter MCT8, resulting in severe psychomotor retardation and abnormal thyroid hormone values in the blood.
    Pazienti con una mutazione del trasportatore degli ormoni tiroidei (MCT8), tale da determinare un grave ritardo psicomotrio ed un alterato profilo degli ormoni tiroidei nel sangue.
    E.1.1.2Therapeutic area Diseases [C] - Hormonal diseases [C19]
    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
    (1) To determine the effect of Triac treatment on serum T3 and other TH levels.
    (2) To determine the effect of Triac on the toxic effects of the hyperthyroid state in peripheral tissues.
    E.2.2Secondary objectives of the trial
    (1) To determine the effects of Triac treatment on the neurological phenotype.
    (2) The registration of adverse event.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    clinical relevant mutation in the MCT8 gene leading to the clinical phenotype of AHDS
    rilevanti mutazioni cliniche del gene MCT8 caratterizzanti la sindrome di AHDS
    E.4Principal exclusion criteria
    - Major illness or recent major surgery (within 4 weeks) unrelated to AHDS;
    - Patients who are participating in ongoing RCTs of therapeutic interventions (including clinical trials of investigational medicinal products);
    - Patients that have any major contra-indication for Triac treatment (severe cardiac decompensation (NYHA 4), coronary insufficiency, severe cardiac arrhytmias, Galactose intolerance, the Lapp lactose deficiency or glucose-galactose malabsorption).
    - gravi patologie o significativi interventi chirurgici (entro 4 settimane) non correlati con la sindrome di AHDS;
    - pazienti che sono arruolati in altri studi clinici interventistici farmacologici in corso;
    - pazienti che hanno gravi controindicazioni al trattamento con Triac (scompenso cardiaco grave (NYHA 4), insufficienza coronarica, grave aritmia cardiaca, intolleranza al galattosio, carenza di Lapp lattasi o malassorbimento di glucosio-galattosio).
    E.5 End points
    E.5.1Primary end point(s)
    1) Serum TSH, T4, vrij T4, T3, rT3 and Triac levels
    E.5.1.1Timepoint(s) of evaluation of this end point
    These primary endpoints will be evaluated at baseline (t=0) and after 1 year of Triac treatment.
    To optimize titration of the Triac dose and prevent overtreatment, primary end-points will be evaluated with an expected average of 2 weeks during months 1-3 of the trial. During months 4-12 these parameters will be evaluated with an expected average of 6 weeks.
    E.5.2Secondary end point(s)
    1) Body weight, blood pressure and heart rate.
    2) Serum levels of markers that reflect peripheral thyroid hormone action: serum steroid hormone binding globuline (SHBG) and lipids (liver), serum beta Ctx and alkalisch phosphatase (bone) and serum kreatine kinase (muscle).
    3) Motor function, using the Gross Motor Function Measure.
    4) Cognitive function using the Bayley Scales of Infant Development III, or Wechsler Preschool and Primary Scale of Intelligence II.
    5) Adaptive behavior measured by the Vineland adaptive behavior scale.
    6) Basal Metabolic Rate (BMR) using Doubly labeled Water (DLW).
    7) The frequency and nature of adverse events.
    E.5.2.1Timepoint(s) of evaluation of this end point
    The secundary end points 1-3 will will be evaluated at baseline (t=0) and after 1 year of Triac treatment.
    To optimize titration of the Triac dose and prevent overtreatment, these end points will also be evaluated with an expected average of 2 weeks during months 1-3 of the trial. During months 4-12 these parameters will be evaluated with an expected average of 6 weeks.
    End points 3), 4) and 5) will be evaluated at baseline and after 1 year of Triac treatment.
    Point 6) will be evaluated during the whole study period.
    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 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 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 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 No
    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 Yes
    E.8.5.1Number of sites anticipated in the EEA11
    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
    the end of the trial is defined as the date of the last investigational visit for the last patient undergoing protocol treatment.
    Lo studio terminerà con l'ultima visita dell'ultimo paziente.
    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 months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months2
    E.8.9.2In all countries concerned by the trial 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: 20
    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.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 8
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 11
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 3
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    F.2 Gender
    F.2.1Female No
    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
    severe psychomotor retardation is part of the AHDS phenotype. Since this study is specifically conducted to evualute the effectiveness of the IMD in this population, the inclusion of this population is inevitable.
    Il ritardo psicomotorio grave è parte della sindrome di AHDS. Poichè questo studio è specificamente condotto per valutare l'efficacia del trattamento con TRIAC su questi soggetti, la loro inclusione è inevitabile.
    F.3.3.7Others No
    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 40
    F.4.2.2In the whole clinical trial 40
    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)
    If Triac treatment turns out to be successfull, a second international trial will be conducted to assess the effects of Triac on the neurological phenotype in greater detail. Patients that participate in this trial will also be enrolled in this second study.
    In addition, the regular health care will be continued during the study period and will also be continued after the study will be ended.
    Se il trattamento con Triac si dimostrerà efficace, sarà disegnato un secondo studio internazionale per valutare l'effetto del trattamento con Triac sul fenotipo neurologico attraverso scale più dettagliate. I pazienti che parteciperanno a questo studio saranno arruolati anche nel secondo.
    I pazienti saranno trattati secondo normale pratica clinica sia durante lo studio che al termine dello stesso.
    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 Decision2016-02-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-10-28
    P. End of Trial
    P.End of Trial StatusOngoing
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