Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

  • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).


    The EU Clinical Trials Register currently displays   43976   clinical trials with a EudraCT protocol, of which   7312   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2019-003370-35
    Sponsor's Protocol Code Number:MCT8-2019-2
    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:2021-05-24
    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 number2019-003370-35
    A.3Full title of the trial
    Tiratricol treatment of children with Monocarboxylate Transporter 8 deficiency: Triac Trial II
    Trattamento con Tiratricol nei bambini con deficit del trasportatore 8 dei monocarbossilati: Triac Trial II
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Tiratricol treatment of children with Monocarboxylate Transporter 8 deficiency: Triac Trial II
    Trattamento con Tiratricol nei bambini con deficit del trasportatore 8 dei monocarbossilati: Triac Trial II
    A.3.2Name or abbreviated title of the trial where available
    Triac Trial II
    Triac Trial II
    A.4.1Sponsor's protocol code numberMCT8-2019-2
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02396459
    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 SponsorRare Thyroid Therapeutics International
    B.1.3.4CountrySweden
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportEurostars
    B.4.2CountryBelgium
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationRare Thyroid Therapeutics International AB
    B.5.2Functional name of contact pointMedical Director
    B.5.3 Address:
    B.5.3.1Street AddressTeatergatan 3
    B.5.3.2Town/ cityStoccolma
    B.5.3.3Post code11148
    B.5.3.4CountrySweden
    B.5.6E-mailmedical@rarethyroid.com
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/17/1945
    D.3 Description of the IMP
    D.3.1Product nameemcitate
    D.3.2Product code [emcitate]
    D.3.4Pharmaceutical form Lozenge
    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 INNTiratricol
    D.3.9.1CAS number 51-24-1
    D.3.9.2Current sponsor codeTiratricol
    D.3.9.3Other descriptive nameTiratricol
    D.3.9.4EV Substance CodeSUB11116MIG
    D.3.10 Strength
    D.3.10.1Concentration unit µg microgram(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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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.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 Yes
    D.2.5.1Orphan drug designation numberEU/3/17/1945
    D.3 Description of the IMP
    D.3.1Product nameEmcitate
    D.3.2Product code [Emcitate]
    D.3.4Pharmaceutical form Lozenge
    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 INNTiratricol
    D.3.9.1CAS number 21-24-1
    D.3.9.2Current sponsor codeTiratricol
    D.3.9.3Other descriptive nameTiratricol
    D.3.9.4EV Substance CodeSUB11116MIG
    D.3.10 Strength
    D.3.10.1Concentration unit µg microgram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number350
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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
    Monocarboxylate Transporter 8 (MCT8) deficiency
    deficit del trasportatore 8 dei monocarbossilati (MCT8)
    E.1.1.1Medical condition in easily understood language
    MCT 8 deficiency or AHDS (Allan-Herndon-Dudley syndrome)
    deficit MCT8 o sindrome di Allan-Herndon-Dudley
    E.1.1.2Therapeutic area Diseases [C] - Hormonal diseases [C19]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10078564
    E.1.2Term Thyroid stimulating hormone deficiency
    E.1.2System Organ Class 10014698 - Endocrine disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the effects of tiratricol treatment on neurodevelopment in young MCT8 deficiency patients as measured by the Gross Motor Function Measure 88 (GMFM-88).
    Valutare gli effetti di tiratricol sul neurosviluppo in pazienti in età evolutiva con deficit di MCT8, misurata in base alla scala Gross Motor Function Measure (GMFM-88).
    E.2.2Secondary objectives of the trial
    1) To evaluate the effect of tiratricol treatment on specific Motor Development milestones by individual item scores in the Gross Motor Function Measure test.

    2) To evaluate the effect of tiratricol treatment on neurodevelopment in young MCT8 deficient patients as measured by the Bayley Scales of Infant Development (BSID-III).

    3) Evaluate the effect of tiratricol on clinical and biochemical thyrotoxic features (serum T3 concentrations, tissue specific markers of thyroid hormone action).
    1. Valutare l'effetto del trattamento con tiratricol su specifici traguardi di sviluppo motorio in base ai punteggi individuali nel test Gross Motor Function Measure
    2. Valutare l'effetto del trattamento con tiratricol sul neurosviluppo in base alle Bayley Scales of Infant Development (BSID-III)
    3. Valutare l'effetto di tiratricol sulle caratteristiche tireotossiche cliniche e biochimiche (concentrazioni sieriche di T3, marcatori tessutali specifici dell'azione dell'ormone tiroideo).
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Signed and dated informed consent form from the parents or legal guardian.
    2. Parents stated willingness to comply with all study procedures and availability for the duration of the study.
    3. The participant should be aged between 0 and 30 months on the day of inclusion.
    4. The participant should have a pathogenic mutation in the MCT8 gene.
    1. Modulo di consenso informato firmato e datato dai genitori o dal tutore legale.
    2. I genitori hanno volontariamente dichiarato di rispettare tutte le procedure di studio e di essere disponibili per tutta la durata dello studio.
    3. Il partecipante deve avere un'età compresa tra 0 e 30 mesi, al giorno dell'inclusione.
    4. Il partecipante dovrebbe presentare una mutazione patogena nel gene MCT8.
    E.4Principal exclusion criteria
    1. Previous treatment with tiratricol.
    2. Previous treatment with a combination of Propylthiouracil (PTU) and Levothyroxine (LT4).
    3. Previous treatment with LT4 for a longer period than three months.
    4. Treatment with LT4 within three months of baseline visit.
    5. Major illness or recent major surgery (within four weeks of baseline visit 1) unrelated to MCT8 deficiency.
    6. Known allergic reactions to components of the IMP.
    7. Treatment with another investigational drug or participation in other interventional trial within three months prior to baseline visit 1.
    8. Patients that have any contra-indication for tiratricol treatment as stated in the Investigators Brochure.
    1. Trattamento precedente con tiratricol.
    2. Trattamento precedente con una combinazione di propiltiouracile (PTU) e levotiroxina (LT4).
    3. Trattamento precedente con LT4 per un periodo più lungo di tre mesi.
    4. Trattamento con LT4 entro tre mesi dalla visita basale.
    5. Malattia grave o recente intervento chirurgico maggiore (entro quattro settimane dalla visita basale 1) non correlato al deficit di MCT8.
    6. Reazioni allergiche note ai componenti dell'IMP.
    7. Trattamento con un altro farmaco sperimentale o partecipazione ad altri studi interventistici entro tre mesi prima della visita basale 1.
    8. Pazienti che hanno controindicazioni per il trattamento con tiratricol come indicato nell'Investigators brochure
    E.5 End points
    E.5.1Primary end point(s)
    GMFM-88 score compared to natural history GMFM-88 scores from untreated patients.
    Punteggio GMFM-88 rispetto allo storico dei punteggi GMFM-88 di pazienti non trattati.
    E.5.1.1Timepoint(s) of evaluation of this end point
    After 24 months of treatment compared to baseline. An interim analysis will be done after 12 months treatment.
    Dopo 24 mesi di trattamento rispetto al basale. Un'analisi intermedia verrà effettuata dopo 12 mesi di trattamento.
    E.5.2Secondary end point(s)
    1) Gross Motor Function Measure test (GMFM-88) individual item score 10 and 24 compared to natural history scores from untreated patients.
    2) Age equivalent (AE) score from the BSID compared to natural history AE scores from untreated patients.
    3) Serum T3 (efficacy), peripheral thyroid hormone TH status (serum SHBG; serum creatine kinase, creatinine, blood pressure and body weight) (efficacy).
    1. Punteggi individuali GMFM-88 per le voci 10 e 24 rispetto allo storico dei punteggi di pazienti non trattati.
    2. Punteggio equivalente di età (AE) dal BSID rispetto allo storico dei punteggi AE di pazienti non trattati
    3. T3 sierico (efficacia), stato dell'ormone tiroideo periferico (SHBG sierico; creatina chinasi sierica, creatinina, pressione sanguigna e peso corporeo) (efficacia).
    E.5.2.1Timepoint(s) of evaluation of this end point
    After 24 months of treatment compared to baseline. An interim analysis will be done after 12 months treatment.
    Dopo 24 mesi di trattamento rispetto al basale. Un'analisi intermedia verrà effettuata dopo 12 mesi di trattamento.
    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 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 No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Tiratricol will be administered to all patients, orally in an individual dose titrated based on seru
    Tiratricol will be administered to all patients, orally in an individual dose titrated based on seru
    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
    Tiratricol verrà somministrato a tutti i pazienti, per via orale in una singola dose titolata in bas
    Tiratricol will be administered to all patients, orally in an individual dose titrated based on seru
    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 Yes
    E.8.5.1Number of sites anticipated in the EEA8
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    United States
    France
    Germany
    Italy
    Netherlands
    United Kingdom
    Czechia
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    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 years3
    E.8.9.2In all countries concerned by the trial months0
    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.1.1.1In 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) Yes
    F.1.1.4.1Number of subjects for this age range: 12
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 6
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    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
    Children 0-30 months of age.
    Bambini da 0 a 30 mesi
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 16
    F.4.2.2In the whole clinical trial 18
    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)
    Patients will be offered treatment in a follow up protocol after completion of the trial.
    Ai pazienti verrà offerto un trattamento in un protocollo di follow-up dopo il completamento della sperimentazione.
    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 Decision2020-04-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-04-30
    P. End of Trial
    P.End of Trial StatusOngoing
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA