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    Summary
    EudraCT Number:2019-000654-59
    Sponsor's Protocol Code Number:MT-2-02
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2019-04-30
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2019-000654-59
    A.3Full title of the trial
    An exploratory, open-label, multicenter study in male pediatric patients with cerebral X-linked Adrenoleukodystrophie (cALD) to assess the effect of MIN-102 treatment on the progression of cerebral lesions.
    Estudio multicéntrico, exploratorio, abierto, en pacientes pediátricos varones con adrenoleucodistrofia cerebral ligada a x (cALD) para evaluar los efectos del tratamiento con min 102 sobre la progresión de las lesiones cerebrales
    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 the progression of brain lesions in boys with cerebral X-linked adrenoleukodystrophy (cALD)
    Un estudio para evaluar los efectos del nuevo medicamento, MIN-02, en la progresión de lesiones cerebrales en niños con adrenoluecodistrofia cerebral ligada a X (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)
    Adrenoleucodistrofia cerebral ligada a x (cALD)
    E.1.1.1Medical condition in easily understood language
    Cerebral adrenoleukodystrophy
    Adrenoleucodistrofia cerebral
    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
    To evaluate the effects of MIN-102 on the progression of cerebral lesions from baseline to immediately prior to hematopoietic stem-cell transplantation (HSCT), as determined by serial magnetic resonance imaging (MRI) in pediatric subjects.
    Evaluar los efectos de MIN-102 sobre la progresión de las lesiones cerebrales respecto al momento basal hasta inmediatamente antes del trasplante de células progenitoras hematopoyéticas (TCPH), determinado mediante resonancia magnética (RM) seriada en sujetos pediátricos.
    E.2.2Secondary objectives of the trial
    • To evaluate the effects of pre-HSCT MIN-102 treatment on:
    − Loes scores
    − Gadolinium intensity score (GIS)
    • To assess the pharmacokinetics (PK), safety, tolerability, and palatability of MIN-102 in pediatric subjects
    • To assess the changes in neurological function from baseline to immediately before decision to HSCT
    • Evaluar los efectos del tratamiento con MIN-102 pre-TCPH sobre:
    - Puntuaciones de Loes
    - Puntuación de intensidad de gadolinio (GIS)
    • Evaluar la farmacocinética (FC), la seguridad, la tolerabilidad y la palatabilidad de MIN-102 en sujetos pediátricos
    • Evaluar los cambios en la función neurológica desde el momento basal hasta inmediatamente antes de la decisión de realizar TCPH.
    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
    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 GIS ≤2
    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
    1. Consentimiento informado por escrito por parte del progenitor/tutor legal o representante legalmente autorizado para participar en el estudio.
    2. Varones de ≥2 y ≤12 años de edad con un diagnóstico de ALD ligada a X basado en pruebas genéticas
    3. Afectación de la sustancia blanca determinada por lesiones en RM cerebral sin realce con Gd en el momento basal (Población 1) o con realce con Gd en el momento basal (Población 2).
    4. Puntuación de Discapacidades Funcionales Mayores (MFD) de 0, determinada por medidas clave en la Escala de Función Neurológica (NFS)
    5. Puntuación de Loes en el momento basal >0 y ≤10
    6. GIS basal ≤2
    7. Sin signos o síntomas de insuficiencia suprarrenal y niveles de cortisol matutino y de aldosterona dentro de los intervalos normales del laboratorio para la edad o sustitución esteroidea adecuada si existe insuficiencia suprarrenal. Los antecedentes de insuficiencia suprarrenal no son motivo de exclusión si actualmente se cumple lo anterior.
    8. Hemoglobina glicada (HbA1c) dentro del intervalo normal
    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. Current use of immunosuppressant medication, excluding corticosteroids
    8. Requirement for a prohibited concomitant medication
    9. Previous or current history of bladder polyps, bladder cell hyperplasia, or cancer (other than successfully treated basal cell carcinoma)
    10. Previous or current history of congestive heart failure
    11. Clinically significant anemia with hemoglobin <10 g/dL
    12. 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)
    13. Moderate or severe hepatic impairment (groups B and C according to Child-Pugh classification)
    14. Chronic kidney disease stage 3 or higher (according to chronic kidney disease staging by The Renal Association)
    15. Pulmonary disease or cardiac disease of sufficient severity to limit participation in the study and/or completion of study procedures
    16. 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
    17. Contraindication to MRI procedure, such as presence of paramagnetic materials (aneurysm clips, pacemaker, intraocular metal, cochlear implant) in the body
    18. Conditions that could modify the absorption of the study drug
    19. Inability or unwillingness of parent/legal guardian or subject to comply with the study procedures
    20. 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
    1. Otras enfermedades neurológicas crónicas
    2. Intolerancia conocida a la pioglitazona u otras tiazolidinedionas
    3. Uso de pioglitazona u otras tiazolidinedionas dentro de los 6 meses previos a la selección
    4. Uso de biotina a una dosis diaria de >50 mg al día en los últimos 3 meses antes de la selección
    5. Participación actual en otro estudio clínico intervencionista o participación en un estudio de este tipo dentro de los 6 meses previos a la selección
    6. TCPH previo
    7. Uso actual de medicación inmunodepresora, excepto corticosteroides
    8. Necesidad de una medicación concomitante prohibida
    9. Historia previa o actual de pólipos vesicales, hiperplasia de las células de la vejiga o cáncer (que no sea carcinoma basocelular tratado satisfactoriamente)
    10. Historia previa o actual de insuficiencia cardíaca congestiva
    11. Anemia clínicamente significativa con hemoglobina <10 g/dl
    12. Nivel de alanina aminotransferasa (ALT) o aspartato aminotransferasa (AST) >2 veces el LSN o bilirrubina total >1,5 veces el LSN (a menos que se deba al síndrome de Gilbert)
    13. Insuficiencia hepática moderada o grave (grupos B y C de acuerdo con la clasificación de Child-Pugh)
    14. Nefropatía crónica en estadio 3 o mayor (de acuerdo con la estadificación de la nefropatía crónica de The Renal Association)
    15. Enfermedad pulmonar o enfermedad cardíaca de suficiente intensidad como para limitar la participación en el estudio y/o la terminación de los procedimientos del estudio
    16. Reducción de la fracción de eyección del ventrículo izquierdo u otras anomalías cardíacas clínicamente significativas en el ecocardiograma que, a criterio del investigador, podrían predisponer al sujeto a sobrecarga de volumen o sus consecuencias correspondientes
    17. Contraindicación al procedimiento de RM, como la presencia de materiales paramagnéticos (clips de aneurismas, marcapasos, metales intraoculares, implante coclear) en el cuerpo
    18. Condiciones que podrían modificar la absorción del fármaco del estudio
    19. Incapacidad o falta de disposición del progenitor/tutor legal o sujeto a cumplir los procedimientos del estudio
    20. Otros problemas médicos, neurológicos, psiquiátricos o sociales, que, a criterio del investigador, es probable que alteren desfavorablemente la relación riesgo-beneficio de la participación en el estudio, confundan la interpretación de los resultados de seguridad o eficacia o interfieran con la terminación satisfactoria de los requisitos del estudio
    E.5 End points
    E.5.1Primary end point(s)
    Progression of cerebral lesions, as determined by serial MRI, using the following parameters:
    • First appearance and time to first appearance of Gd-enhancing cerebral lesions in subjects not showing these lesions at baseline in Population 1
    • Change from baseline to immediately prior to HSCT in the T1 and T2/FLAIR volume of Gd-enhancing cerebral lesions in Population 2
    • Progression of FLAIR based overall lesion burden volume, assessed in both Populations
    Progresión de lesiones cerebrales, determinada mediante RM seriada, usando los siguientes parámetros:
    • Primera aparición y tiempo hasta la primera aparición de lesiones cerebrales realzadas con Gd en sujetos que no muestran estas lesiones en el momento basal en la Población 1
    • Cambio entre el momento basal e inmediatamente antes del TCPH en el volumen de T1 y T2/FLAIR de lesiones cerebrales realzadas con Gd en la Población 2
    • Progresión de volumen de carga de lesiones globales basadas en FLAIR, evaluada en ambas poblaciones
    E.5.1.1Timepoint(s) of evaluation of this end point
    From Baseline to immediately prior to HSCT
    Desde el momento basal hasta inmediatamente antes del TCPH
    E.5.2Secondary end point(s)
    • Change from baseline to immediately prior to HSCT, or decision not perform HSCT, in Loes MRI severity score
    • Change from baseline to immediately prior to HSCT, or decision not perform HSCT in GIS
    • Changes from baseline to immediately prior to HSCT, or decision not perform HSCT in:
    − Total score of Neurological Function Score (NFS)
    − Total score of the 6 Major Functional Disabilities (MFD) of the NFS
    • Cambio entre el momento basal e inmediatamente antes del TCPH o la decisión de no realizar TCPH, en la puntuación de intensidad Loes en la RM
    • Cambio entre el momento basal e inmediatamente antes del TCPH o la decisión de no realizar TCPH, en la GIS
    • Cambios entre el momento basal e inmediatamente antes del TCPH o la decisión de no realizar TCPH, en:
    - Puntuación total de la Puntuación de Función Neurológica (NFS)
    - Puntuación total de las 6 Discapacidades Funcionales Mayores (MFD) de la NFS
    E.5.2.1Timepoint(s) of evaluation of this end point
    From Baseline to immediately prior to HSCT
    Desde el momento basal hasta inmediatamente antes del TCPH
    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 EEA2
    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
    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 years1
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months2
    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: 10
    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) 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 10
    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 Decision2019-06-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-06-21
    P. End of Trial
    P.End of Trial StatusOngoing
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