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    Summary
    EudraCT Number:2022-001478-78
    Sponsor's Protocol Code Number:MCT8-2021-3
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2022-10-25
    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 number2022-001478-78
    A.3Full title of the trial
    Withdrawal of Tiratricol Treatment in Males with Monocarboxylate Transporter 8 Deficiency (MCT8 Deficiency): A Double blind, Randomized, Placebo controlled Study
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Withdrawal of Tiratricol treatment of children with Monocarboxylate Transporter 8 deficiency: ReTRIACt
    A.3.2Name or abbreviated title of the trial where available
    ReTRIACt
    A.4.1Sponsor's protocol code numberMCT8-2021-3
    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 IMPIntragastric use (Noncurrent)
    Oral 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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    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
    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
    To evaluate the effects of withdrawal of tiratricol treatment (placebo group) on serum total triiodothyronine (T3) concentrations, measured by liquid chromatography with tandem mass spectrometry (LC/MS/MS), and the requirement for rescue treatment with tiratricol as compared to continuing tiratricol treatment (tiratricol group), in males diagnosed with MCT8 deficiency and on a stable maintenance dose of tiratricol
    E.2.2Secondary objectives of the trial
    1. To evaluate the safety and tolerability of tiratricol treatment
    2. To evaluate the effect of tiratricol treatment upon serum thyroid hormone measurements, sex hormone binding globulin (SHBG), and cardiovascular measurements
    3. To evaluate the serum concentrations of tiratricol
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male participants diagnosed with a pathogenic mutation in the MCT8 gene, confirmed with a genetic test.
    2. Serum total T3 concentration above the ULN of the age specific normal range :
    a) at the time of diagnosis (or the closest sample taken prior to first ever treatment with tiratricol) for participants who are currently treated with tiratricol (if serum total T3 concentration is not available, free T3 results from standard of care samples may be used).
    b) In the Screening Visit sample:
    i. For participants who have never received and/or are currently not receiving tiratricol.
    ii. For participants who stopped prohibited medications per exclusion criterion #6
    3. Participants will be aged 4 years or older at the time of randomization.
    Participants entering screening who are <4 years of age but expected to be aged 4 years at randomization should be discussed with the medical monitor.
    4. Signed and dated informed consent form from the parents or legal guardian.
    E.4Principal exclusion criteria
    1. Major illness or recent major surgery unrelated to MCT8 deficiency (in the principal investigator’s judgement), defined as:
    • Conditions requiring repeated hospitalizations that are likely to confound ability to participate in the trial.
    • Major illness in the 3 months prior to the Screening Visit that is likely to confound the ability of the participant to participate fully within the trial and/or confound the assessment of serum total T3 and/or safety.
    • Major surgery within the 3 months prior to the Screening Visit or planned to take place during the study, including but not limited to major abdominal/thoracic/neurosurgical procedures.
    • Major/minor abdominal and/or maxillofacial surgery that may inhibit the administration and/or absorption of study drug.
    2. Body weight <10 kg at the Screening Visit.
    3. Patients who are participating, or intend to participate, in other therapeutic and/or interventional clinical studies during the study period.
    4. History of allergic reactions to components of tiratricol or any excipients in the investigational product (IP).
    5. Participants with any contra-indication for treatment with tiratricol or any excipients in the IP.
    6. Participants who have used other T3 analogues, levothyroxine, propylthiouracil, or other antithyroid medications within 6 weeks of screening
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of participants who meet the rescue criterion (serum total T3 > ULN) from samples obtained during the 30 day double-blind Randomized Treatment Period
    E.5.1.1Timepoint(s) of evaluation of this end point
    end of Randomized Treatment period
    E.5.2Secondary end point(s)
    • Change in serum thyroid hormone variables (T3, T4, TSH, fT3, and fT4) from baseline (start of the Randomized Treatment Period) to the end of Randomized Treatment Period (completion or rescue)
    • Change in serum thyroid hormone variables (T3, T4, TSH, fT3, and fT4) from initiation of tiratricol administration at screening to the last measurement prior to randomization (Cohort B only)
    • Change in the cardiovascular variables from extended ECG assessments, 24 hour ABPM, and heart rate assessments from the last measurement prior to the start of the Randomized Treatment Period to the end of the Randomized Treatment Period (completion or rescue)
    • Serum concentrations of tiratricol
    • Change in serum SHBG from baseline (start of the Randomized Treatment Period) to the end of the Randomized Treatment Period (completion or rescue)
    • Time (days) from randomization to the time when the rescue criterion is met or the time of completion of the Randomized Treatment Period (whichever comes first)
    E.5.2.1Timepoint(s) of evaluation of this end point
    end of Randomized Treatment 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 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) 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 Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    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 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 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 EEA1
    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 Kingdom
    United States
    Netherlands
    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.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months12
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial months12
    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: 16
    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: 9
    F.1.1.6Adolescents (12-17 years) No
    F.1.1.6.1Number of subjects for this age range: 4
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 3
    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
    Male participants >4 years old at time of randomization
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 5
    F.4.2.2In the whole clinical trial 16
    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 the end of the study, each participant will be treated according to standard clinical practice. In addition, participants will be invited to receive continuing treatment with tiratricol under a compassionate-use program.
    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 Decision2022-10-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-03-31
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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