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    Summary
    EudraCT Number:2019-003370-35
    Sponsor's Protocol Code Number:MCT8-2019-2
    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:2020-03-17
    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 number2019-003370-35
    A.3Full title of the trial
    Tiratricol treatment of children with Monocarboxylate Transporter 8 deficiency: 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
    A.3.2Name or abbreviated title of the trial where available
    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 AB
    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 AddressKlara Norra Kyrkogata 26
    B.5.3.2Town/ cityStockholm
    B.5.3.3Post code11122
    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.3.4Pharmaceutical 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 INNTIRATRICOL
    D.3.9.1CAS number 51-24-1
    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 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.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.3.4Pharmaceutical 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 INNTIRATRICOL
    D.3.9.1CAS number 51-24-1
    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 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
    Monocarboxylate Transporter 8 (MCT8) deficiency
    E.1.1.1Medical condition in easily understood language
    MCT 8 deficiency or AHDS (Allan-Herndon-Dudley syndrome)
    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
    Part I: Evaluate the effects of tiratricol treatment on neurodevelopment in young MCT8 deficiency patients, as measured by the Gross Motor Function Measure (GMFM-88) and Bayley Scales of Infant Development (BSID)-III Gross Motor Skill Domain.

    Part II: Evaluate the effects of long-term treatment (up to 4 years of total treatment) with tiratricol on neurodevelopment in young boys (≤30 months) with MCT8 deficiency, as measured by the Gross Motor Function Measure (GMFM)-88 and Bayley Scales of Infant Development (BSID)-III Gross Motor Skill Domain.
    E.2.2Secondary objectives of the trial
    Part I:
    1. Evaluate the effect of Tiratricol treatment at week 96 on specific motor development milestones.
    2. Evaluate the effect of tiratricol treatment on neurodevelopment in young MCT8 deficient patients as measured by the BSID-III.
    3. Evaluate the effect of tiratricol at week 96 on clinical and biochemical thyrotoxic features (serum T3 concentrations, tissue specific markers of thyroid hormone action).

    Part II:
    1. Evaluate the effect of long-term treatment (up to 4 years of total treatment) with tiratricol on specific motor development milestones.
    2. Evaluate the effect of long-term treatment (up to 4 years of total treatment) with tiratricol as measured by the BSID-III.
    3. Evaluate the long-term treatment effect (up to 4 years of total treatment) of tiratricol on clinical and biochemical thyrotoxic features (serum T3 concentrations, tissue specific markers of thyroid hormone action).
    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 be male and have a pathogenic mutation in the MCT8 gene.
    E.4Principal exclusion criteria
    1. Previous treatment with tiratricol.
    2. Previous treatment with LT4 and/or PTU and/or other anti-thyroid medication for a period longer than three months. Patients previously treated with LT4 for a shorter period than 3 months may be included in the study (baseline visit) six weeks (or longer) after last dose of LT4 if two consecutive analyses show stable TFT*. Patients treated with PTU and/or other anti-thyroid medication for a shorter period than three months may be included in the study (baseline visit) six weeks (or longer) after last dose.
    3. Major illness or recent major surgery (within four weeks of baseline visit 1) unrelated to MCT8 deficiency.
    4. Known allergic reactions to components of the IMP. Patients with galactose intolerance, Lapp lactase deficiency or malabsorption of glucose or galactose (the IMP contains lactose).
    5. Treatment with another investigational drug or participation in other interventional trial within three months prior to baseline visit 1.

    *Stable TFT (T3, T4, fT4), determined as a maximal variation of 20%, should be demonstrated at two separate occasions at least two weeks apart, measured on the same platform.

    E.5 End points
    E.5.1Primary end point(s)
    Part I of the study: GMFM-88 total score and BSID Gross Motor Skill Domain at week 96 compared to natural history scores from the Triac Trial I study
    Part II of the study: GMFM-88 total score and BSID-III Gross Motor Skill Domain at 3 years and 4 years respectively, compared to natural history scores from the Triac Trial I study.*
    E.5.1.1Timepoint(s) of evaluation of this end point
    Part I: At week 96 compared to historical controls
    Part II: After 4 years compared to historical controls
    E.5.2Secondary end point(s)
    Part I:
    1. GMFM-88 individual item score 10 (“lifts head upright”) and item score 24 (“sit on mat”) at week 96 compared to baseline; GMFM Domain B (Sitting) - summary score of all items 18-37 at week 96 compared to baseline; Motor milestone responder analysis of Section 2 of the Hammersmith Infant Neurological Examination (HINE) at week 96.
    2. Age equivalent (AE) score from the BSID-III compared to baseline and historical controls.
    3. Serum T3 (efficacy), peripheral thyroid hormone status (serum SHBG; serum creatine kinase, creatinine, blood pressure and body weight) (efficacy).

    Part II:
    1. GMFM-88 individual item score 10 (“lifts head upright”) and item score 24 (“sit on mat”) at 3 years and 4 years respectively, compared to baseline in Triac Trial II; GMFM Domain B (Sitting) - summary score of all items 18-37 at 3 years and 4 years respectively, compared to baseline in Triac Trial II; Motor milestone responder analysis of Section 2 of the Hammersmith Infant Neurological Examination (HINE) at 3 years and 4 years respectively.
    2. Age equivalent (AE) score from the BSID-III at 3 years and 4 years respectively, compared to baseline and historical controls.
    3. Serum T3 (efficacy) at 3 years and 4 years respectively, peripheral thyroid hormone status (serum SHBG; serum creatine kinase, creatinine, blood pressure and body weight) (efficacy).
    E.5.2.1Timepoint(s) of evaluation of this end point
    Part I: At week 96 compared to historical controls
    Part II: After 4 years compared to historical controls
    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 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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    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 No
    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
    Netherlands
    Czechia
    Germany
    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 study is defined as completion of the last visit for the last patient in the trial globally. The end of Part I is defined as last patient completing visit 13.
    Patients who completed Part I of the study will be offered to continue treatment for another 112 weeks in Part II.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years6
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years6
    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: 22
    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: 14
    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) 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.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 16
    F.4.2.2In the whole clinical trial 22
    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.
    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 Decision2020-03-17
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-06-26
    P. End of Trial
    P.End of Trial StatusOngoing
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