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    Summary
    EudraCT Number:2019-000654-59
    Sponsor's Protocol Code Number:MT-2-02
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2019-07-19
    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 ConcernedGermany - BfArM
    A.2EudraCT number2019-000654-59
    A.3Full title of the trial
    An open-label, multicenter study in male pediatric patients with cerebral X-linked Adrenoleukodystrophy (cALD) to assess the effect of MIN-102 treatment on disease progression prior to human stem cell transplant (HSCT)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to evaluate the effects of a new drug called MIN-102 on disease
    progression in boys with cerebral X-linked adrenoleukodystrophy (cALD)
    A.4.1Sponsor's protocol code numberMT-2-02
    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 SponsorMinoryx Therapeutics S.L.
    B.1.3.4CountrySpain
    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 supportMinoryx Therapeutics S.L.
    B.4.2CountrySpain
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMinoryx Therapeutics S.L.
    B.5.2Functional name of contact pointClinical Operations
    B.5.3 Address:
    B.5.3.1Street AddressAv. Ernest Lluch 32, TCM3
    B.5.3.2Town/ cityMataró (Barcelona)
    B.5.3.3Post code08302
    B.5.3.4CountrySpain
    B.5.4Telephone number+34 935 441 466
    B.5.6E-mailspascual@minoryx.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/3/16/177
    D.3 Description of the IMP
    D.3.1Product name5-[4-[2-(5-(1-hydroxyethyl)-2- pyridinyl)ethoxy]benzyl]-2,4- thiazolidinedione hydrochloride
    D.3.4Pharmaceutical form Oral suspension
    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 INNLeriglitazone
    D.3.9.1CAS number 146062-46-6
    D.3.9.2Current sponsor codeMIN-102
    D.3.9.3Other descriptive name5-[[4-[2-[5-(1-HYDROXYETHYL)-2-PYRIDINYL]ETHOXY]PHENYL]METHYL]-2,4-THIAZOLIDINEDIONE HYDROCHLORIDE
    D.3.9.4EV Substance CodeSUB196366
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number15
    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
    Cerebral X-linked Adrenoleukodystrophy (cALD)
    E.1.1.1Medical condition in easily understood language
    cerebral adrenoleukodystrophy
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10051260
    E.1.2Term Adrenoleukodystrophy
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of the study is to evaluate whether MIN-102 can
    halt disease progression of cALD at week 96, as determined by serial clinical and magnetic resonance imaging (MRI) investigations in pediatric subjects.
    E.2.2Secondary objectives of the trial
    To determine the effects of MIN-102 treatment on further clinical and imaging parameters.
    • To assess the changes in neurological function.
    • To evaluate the effects of pre-HSCT MIN-102 treatment on:
    - Loes scores
    - Gadolinium intensity score (GIS)
    - Overall survival of patients who have not undergone HSCT
    - Number of patients meeting HSCT criteria
    • To assess the pharmacokinetics (PK), safety, tolerability, and
    palatability of MIN-102 in pediatric subjects
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Written informed consent by parent/legal guardian, or authorized
    legal representative to participate in the study
    2. Males aged ≥2 and ≤12 years with a diagnosis of X-linked ALD based
    on genetic testing; or, in absence of genetic testing, elevation of VLCFA
    and confirmed by family history of X-ALD with clinical symptoms and
    elevation of VLCFA or by genetic testing of a family member.
    3. White matter involvement as determined by cerebral MRI lesions
    without Gd enhancement at baseline (Population 1), or with Gd
    enhancement at baseline (Population 2).
    4. Major Functional Disabilities (MFD) score of 0, as determined by key
    measures in the Neurological Function Scale (NFS)
    5. Baseline Loes score >0 and ≤10
    6. Baseline Gadolinium Intensity Score (GIS) ≤3
    7. No signs or symptoms of adrenal insufficiency and morning cortisol
    and aldosterone levels within normal laboratory ranges for age, or
    appropriate steroid replacement if adrenal insufficiency is present. A
    history of adrenal insufficiency is not exclusionary if the foregoing is
    currently met.
    8. Glycated hemoglobin (HbA1c) within normal range
    E.4Principal exclusion criteria
    1. Other chronic neurological disease
    2. Known intolerance to pioglitazone or other thiazolidinediones
    3. Use of pioglitazone or other thiazolidinediones within the past 6 months prior to screening
    4. Use of biotin at a daily dose of >50 mg per day within the past 3 months prior to screening
    5. Current participation in another interventional clinical study or participation in such a study within 6 months prior to screening
    6. Previous HSCT
    7. Requirement for a prohibited concomitant medication (section 3.4.10)
    8. Previous or current history of bladder polyps, bladder cell hyperplasia, or cancer (other than successfully treated basal cell carcinoma)
    9. Previous or current history of congestive heart failure
    10. Clinically significant anemia with hemoglobin <10 g/dL
    11. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) level >2 times the ULN or total bilirubin >1.5 times the ULN (unless due to Gilbert's syndrome)
    12. Moderate or severe hepatic impairment (groups B and C according to Child-Pugh classification)
    13. eGFR < 90 ml/min or any evidence of renal disease or impairment,including proteinuria or hematuria
    14. Pulmonary disease or cardiac disease of sufficient severity to limit participation in the study and/or completion of study procedures
    15. Reduced left-ventricular ejection fraction or other clinically significant cardiac abnormalities
    on echocardiogram that, in the investigator's opinion, could predispose the subject to volume
    overload or its attendant consequences
    16. Hereditary Fructose Intolerance
    17. History of diabetes, or glycated hemoglobin (HbA1c) levels >6.4%
    and fasting blood glucose levels ≤ 0.9 times the lower limit of normal
    and ≥ 1.1 times the upper limit of normal at Screening
    18. Contraindication to MRI procedure, such as presence of
    ferromagnetic materials (aneurysm clips, pacemaker, intraocular metal,
    cochlear implant) in the body
    19. Conditions that could modify the absorption of the study drug
    20. Inability or unwillingness of parent/legal guardian or subject to
    comply with the study procedures
    21. Inability or unwillingness of parent/legal guardian or subject to
    resume standard of care at a local center once study is complete or
    criteria for HSCT is met and treatment available.
    22. Other medical, neurologic, psychiatric, or social condition that, in the
    opinion of the investigator, is likely to unfavorably alter risk-benefit of
    study participation, confound interpretation of safety or efficacy results,
    or interfere with the satisfactory completion of study requirements
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint will be the number of patients meeting
    "arrested disease" criteria at Visit 8 and Visit 12.
    E.5.1.1Timepoint(s) of evaluation of this end point
    From Baseline to Visit 8 and Visit 12
    E.5.2Secondary end point(s)
    • Sustained change from Baseline in the score composed of NFS items 1 (hearing/auditory processing), 2 (aphasia/apraxia), 4 (vision
    impairment), 10 (spastic gait) and 13 (incontinence)
    • Sustained change from Baseline in total NFS score
    "Sustained change" is defined as the same total score for NFS items 1, 2,
    4, 10, and 13 and no change >1 in NFS total score observed in two the
    consecutive Visits 6–8, and Visits 11-12, respectively. If "sustained
    change" definition is not met, the average of the two scores of Visit 6
    and 8, and Visit 11 and 12, respectively, will be used. If NFS total score
    differs by 1 point between V6-8, or V11-12, the higher of the two scores
    is used.
    • Change from baseline in Loes MRI severity score
    • Change from baseline in Gadolinium Intensity Score (GIS)
    • Overall survival of patients who have not undergone HSCT
    • Number of patients meeting HSCT criteria
    E.5.2.1Timepoint(s) of evaluation of this end point
    Change from Baseline
    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 Yes
    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.8.2.4Number 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 Yes
    E.8.5.1Number of sites anticipated in the EEA3
    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
    Argentina
    United States
    France
    Germany
    Spain
    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 years4
    E.8.9.1In the Member State concerned months9
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months9
    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: 13
    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: 12
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 1
    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 No
    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
    In the study male children between 2-12 years will be enroled.
    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 8
    F.4.2.2In the whole clinical trial 13
    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 a subject discontinues MIN-102 treatment after a decision not to undergo HSCT, or discontinues treatment per the end-of treatment criteria, he will have a Follow-Up Visit 28 (± 5) days after the last administration of study drug. If a subject drops out of the study before planned HSCT, the Follow-up Visit should be performed 28 days after last administration of study drug, or immediately before initiation of HSCT procedures if these occur earlier than 28 days after last dose of study drug.
    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 Decision2019-08-05
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-08-13
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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    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.

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