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    Summary
    EudraCT Number:2017-000748-16
    Sponsor's Protocol Code Number:MT-2-01
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2017-06-22
    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 number2017-000748-16
    A.3Full title of the trial
    A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTINATIONAL, MULTICENTER STUDY WITH OPEN-LABEL TREATMENT EXTENSION TO ASSESS THE EFFECT OF MIN-102 ON THE PROGRESSION OF ADRENOMYELONEUROPATHY IN MALE PATIENTS WITH X-LINKED ADRENOLEUKODYSTROPHY
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to evaluate effect of MIN-102 compared to Placebo after 96 weeks of randomized, double-blind Treatment followed by open-label extension study to evaluate effect of long term treatment with MIN-102 on the progression of adrenomyeloneuropathy (AMN) in male patients
    A.3.2Name or abbreviated title of the trial where available
    ADVANCE
    A.4.1Sponsor's protocol code numberMT-2-01
    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 pointSílvia Pascual
    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 number034935441466
    B.5.5Fax number034930160119
    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/3/16/1770
    D.3 Description of the IMP
    D.3.1Product name5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride
    D.3.2Product code MIN-102
    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 INN5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride
    D.3.9.1CAS number 146062-44-4
    D.3.9.2Current sponsor codeMIN-102
    D.3.9.4EV Substance CodeSUB16466MIG
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboOral suspension
    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
    ADRENOMYELONEUROPATHY IN MALE PATIENTS WITH X-LINKED ADRENOLEUKODYSTROPHY
    E.1.1.1Medical condition in easily understood language
    ADRENOMYELONEUROPATHY
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    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
    Main Study:
    Efficacy of MIN-102 on the progression of adrenomyeloneuropathy (AMN) in male patients as determined by the change from baseline in Six-Minute Walk Test (6MWT) compared with placebo after 96 weeks of treatment.

    Extension Study:
    To assess the safety and tolerability of MIN-102 upon long-term treatment.
    E.2.2Secondary objectives of the trial
    Main Study:
    To evaluate the effects of MIN-102 after 96 weeks of treatment on:
    • Change from baseline in body sway amplitude (in four states: eyes closed/feet apart, eyes open/feet apart, eyes closed/feet together, eyes open/feet together)
    • Comprehensive clinical rating scales (Severity Score System for Progressive Myelopathy [SSPROM] and Expanded Disability Status Scale [EDSS])
    • Clinician and patient global impression of symptom severity and change
    • Muscle strength
    • Quality of life
    • Incidence of progression of cerebral lesions.
    • Loes severity score

    Extension Study:
    Secondary objectives- To evaluate the long-term effects of MIN-102 on:
    • Change from baseline in 6MWT and body sway (in four states: eyes closed/feet apart, eyes open/feet apart, eyes closed/feet together, eyes open/feet together)
    • Comprehensive clinical rating scales (SSPROM and EDSS)
    • Quality of life
    • Incidence of progression of cerebral lesions.
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Optional CSF Sampling Sub-Study (integral part of Protocol Version 2.0 dated 05 Oct 2017)
    Objective: To evaluate the effects of MIN-102 on various biochemical markers in plasma and cerebrospinal fluid (CSF).

    Optional Spinal Cord MRI scan Sub-study (integral part of Protocol Version 2.0 dated 05 Oct 2017)
    Objective: To evaluate the effects of MIN-102 on spinal cord imaging parameters.
    E.3Principal inclusion criteria
    Main Study:

    1. Provision of written informed consent to participate in the main study.
    2. Male patients aged ≥18 to ≤65 years.
    3. Diagnosis of ALD based on genetic testing.
    4. Clinical evidence of spinal cord involvement, with an EDSS score between 2 and 6.
    5. Ability to walk for 6 minutes, without or with rest, with usual walking aids (e.g. lag braces, cane or crutch).
    6. Ability to stand on a force plate with closed eyes and with feet apart for a minimum of 20 seconds.
    7. Either a normal brain MRI or a type-1 through type-5 pattern MRI abnormality in which the abnormality does not show presence of inflammation. Note: MRI is not required at V-1 if an MRI was obtained within the 6 months prior to the first day of screening.
    8. Normal adrenal function or appropriate steroid replacement if adrenal insufficiency is present.
    9. Patients who are surgically sterilized. If not surgically sterilized, patients should be willing to use adequate contraception and not donate sperm from the first dose of the study medication until 90 days after the follow-up visit. Adequate contraception for the male patient (and his female partner, if of childbearing potential) is defined as hormonal contraceptives or an intrauterine device combined with at least 1 of the following forms of contraception: a diaphragm or cervical cap, or a condom. Total abstinence, in accordance with the lifestyle of the patient, is also acceptable.

    Extension Study:
    1. Completion of the entire 96-weeks double-blind period of the study (Part 1).
    2. Provision of written informed consent to participate in the extension study part.
    3. Normal adrenal function or appropriate steroid replacement if adrenal function has changed during the double-blind treatment phase.
    4. The following inclusion criteria of Part 1 will be modified:
    • age ≥18 to ≤65 years will no longer apply.
    • EDSS score between 2 and 6 will no longer apply.
    • Ability to walk for 6 minutes will no longer apply. If a patient is unable to walk for 6 minutes, the maximum time and walking distance will be recorded. If a patient is unable or refuses to walk at all, this will also be recorded.
    • Ability to stand on a force plate with eyes closed and feet apart for a minimum of 20 seconds will no longer apply. If a patient is no longer able to stand on a force plate for 20 seconds, this test will not be conducted.
    • Either a normal brain MRI or a type-1 through type-5 pattern MRI abnormality in which the abnormality does not show presence of inflammation (gadolinium enhancement) will no longer apply.
    In case of brain MRI lesions making the patient eligible for HSCT, he is still eligible for the extension study, but treatment will be discontinued immediately before any transplant-related treatment is initiated. A stable dose of Lorenzo's Oil, botulinum toxin, N-acetylcysteine, baclofen, benzodiazepines, opiates and cannabis preparations, fampidrine and antioxidants will no longer apply.
    All other inclusion criteria of Part 1 remain in place.
    E.4Principal exclusion criteria
    Main Study & Extension Study:
    1. Any other chronic neurological disease with signs of spastic paraplegia
    2. Known type 1 or type 2 diabetes.
    3. Known intolerance to pioglitazone or any other thiazolidinedione.
    4. Current treatment with immunosuppressant medication, except for corticosteroids.
    5. Previous or current history of cancer, bone marrow transplantation, Congestive heart failure, significant liver and renal disorders, anemia, pulmonary or cardiac diseases, Cognitive or behavioral abnormalities, alcohol/drug abuse etc.
    6. 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.
    7. A positive result on laboratory tests for hepatitis B surface antigen, hepatitis C antibody and human immunodeficiency virus antibody.
    E.5 End points
    E.5.1Primary end point(s)
    Main Study:
    1. Efficacy: Change from baseline to week 96 in the total walking distance in the 6MWT

    Extension Study:
    1. Long-term safety and tolerability assessed in terms of AE, SAE, SUSARs, Vitals signs, ECG, Clinical Laboratory tests
    E.5.1.1Timepoint(s) of evaluation of this end point
    During treatment period
    E.5.2Secondary end point(s)
    Main Study:Secondary efficacy endpoints are changes from baseline to week 96 in the following:
    • Body sway amplitude (in four states: eyes closed/feet apart, eyes open/feet apart, eyes closed/feet together, eyes open/feet together)
    • SSPROM
    • EDSS
    • Clinical Global Impressions - Severity (CGI-S)
    • Clinical Global Impressions - Improvement (CGI-I)
    • Patient Global Impressions - Improvement (PGI-I)
    • Dynamometry
    • Quality of Life Assessments
    - European Quality of Life 5 Dimensions (EQ-5D-5L)
    - Multiple Sclerosis Walking Scale (MSWS-12)
    - Qualiveen Short Form quality of life questionnaire for urinary disorders (Qualiveen-SF)
    - International Index of Erectile Function questionnaire (IIEF)
    • Cerebral MRI (incidence of inflammatory lesions).

    Extension Study:
    The efficacy endpoints are the changes from the baseline of Part 2 (V6) to the last scheduled on-study assessment for the following variables:
    • 6MWT
    • Body sway amplitude (in four states: eyes closed/feet apart, eyes open/feet apart, eyes closed/feet together, eyes open/feet together)
    • SSPROM
    • EDSS
    • Quality of Life Assessments (EQ-5D-5L, MSWS-12, Qualiveen-SF and IIEF).
    • Cerebral MRI (incidence of inflammatory lesions).
    E.5.2.1Timepoint(s) of evaluation of this end point
    During 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 No
    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) 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 Yes
    E.8.1.7.1Other trial design description
    Main Study is Randomized, Controlled, Double-Blind and Extension Study is Open-Label
    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 EEA7
    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
    France
    Germany
    Hungary
    Italy
    Netherlands
    Spain
    United Kingdom
    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
    Main Study: LVLS (expected duration of trial is 3 years after which Clinical Study Report (CSR) will be written)

    Extension Study: LVLS (expected duration of trial cannot be estimated since it will be for unlimited time in principle)
    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 months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    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) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 105
    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 No
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state23
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 88
    F.4.2.2In the whole clinical trial 105
    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)
    Extension study is intended to be continued for unlimited time until one of the termination criteria according to the protocol is met
    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 Decision2017-10-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-01-02
    P. End of Trial
    P.End of Trial StatusOngoing
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