<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT03231878</url>
  </required_header>
  <id_info>
    <org_study_id>MT-2-01</org_study_id>
    <nct_id>NCT03231878</nct_id>
  </id_info>
  <brief_title>A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients.</brief_title>
  <acronym>Advance</acronym>
  <official_title>A Randomized, Double-blind, Placebo-controlled, Multinational, Multicenter Study With Open-label Treatment Extension to Assess the Effect of MIN-102 (IMP) on the Progression of Adrenomyeloneuropathy in Male Patients With X-linked Adrenoleukodystrophy</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Minoryx Therapeutics, S.L.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Minoryx Therapeutics, S.L.</source>
  <oversight_info>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      This is a Phase II/III, randomized, double-blind, placebo-controlled, multicenter, two&#xD;
      parallel-group study in male patients with the AMN phenotype of X-linked adrenoleukodystrophy&#xD;
      (X-ALD) to assess the efficacy and safety of MIN-102 treatment. Study sites will consist of&#xD;
      specialist referral centers experienced in the management of adrenoleukodystrophy (ALD).&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Unknown status</overall_status>
  <last_known_status>Active, not recruiting</last_known_status>
  <start_date type="Actual">December 8, 2017</start_date>
  <completion_date type="Anticipated">June 2023</completion_date>
  <primary_completion_date type="Actual">June 25, 2021</primary_completion_date>
  <phase>Phase 2/Phase 3</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>Triple (Participant, Care Provider, Investigator)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>To evaluate the efficacy of MIN-102 on the progression of adrenomyeloneuropathy (AMN) in male patients as determined by a motor function test.</measure>
    <time_frame>in 96 weeks</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>To evaluate the efficacy of MIN-102 in terms of patient reported outcomes.</measure>
    <time_frame>in 96 weeks</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>SSPROM (Severity Score System for Progressive Myelopathy )</measure>
    <time_frame>in 96 weeks</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>EDSS (Expanded Disability Status Scale )</measure>
    <time_frame>in 96 weeks</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Quality of life scales (Euroqol)</measure>
    <time_frame>in 96 weeks</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Incidence of cerebral inflammatory lesions</measure>
    <time_frame>in 96 weeks</time_frame>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Anticipated">105</enrollment>
  <condition>Adrenoleukodystrophy</condition>
  <arm_group>
    <arm_group_label>Active</arm_group_label>
    <arm_group_type>Active Comparator</arm_group_type>
  </arm_group>
  <arm_group>
    <arm_group_label>Placebo</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>MIN-102</intervention_name>
    <description>MIN-102 treatment</description>
    <arm_group_label>Active</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Placebos</intervention_name>
    <description>Placebo</description>
    <arm_group_label>Placebo</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Male and between 18-65 years of age.&#xD;
&#xD;
          -  Diagnosed with X-linked adrenoleukodystrophy (X-ALD) based on elevated VLCFA and&#xD;
             genetic testing.&#xD;
&#xD;
          -  Clinical evidence of spinal cord involvement.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Any other chronic neurological disease with signs of spastic paraplegia, such as&#xD;
             hereditary spastic paraplegia, multiple sclerosis, etc.&#xD;
&#xD;
          -  Presence of inflammatory (Gd-enhancing) MRI lesions or any abnormality other than&#xD;
             those mentioned in the inclusion criteria.&#xD;
&#xD;
          -  Known type 1 or type 2 diabetes.&#xD;
&#xD;
          -  Known intolerance to pioglitazone or any other thiazolidinedione.&#xD;
&#xD;
          -  Taking or have taken honokiol, pioglitazone or other thiazolidinediones within the 6&#xD;
             months prior to screening.&#xD;
&#xD;
          -  Previous bone marrow transplantation.&#xD;
&#xD;
          -  Previous or current history of cancer (other than treated basal cell carcinoma).&#xD;
&#xD;
          -  Previous or current history of congestive heart failure.&#xD;
      </textblock>
    </criteria>
    <gender>Male</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>65 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>Stanford University Medical Center</name>
      <address>
        <city>Stanford</city>
        <state>California</state>
        <zip>94304</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Kennedy Krieger Institute</name>
      <address>
        <city>Baltimore</city>
        <state>Maryland</state>
        <zip>21205</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Massachusetts General Hospital</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02114</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital de la Pitié-Salpêtrière</name>
      <address>
        <city>Paris</city>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Universitat Leipzig Klinik and Poliklinik für Neurologie</name>
      <address>
        <city>Leipzig</city>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Institute of Genomic Medicine and Rare Disorders</name>
      <address>
        <city>Budapest</city>
        <country>Hungary</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Instituto Neurologico Carlo Besta</name>
      <address>
        <city>Milano</city>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Academish Medisch Centrum</name>
      <address>
        <city>Amsterdam</city>
        <country>Netherlands</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital Universitari Vall d'Hebrón</name>
      <address>
        <city>Barcelona</city>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>National hospital for Neurology and Neurosurgery Charles Dent Metabolic Unit)</name>
      <address>
        <city>London</city>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>France</country>
    <country>Germany</country>
    <country>Hungary</country>
    <country>Italy</country>
    <country>Netherlands</country>
    <country>Spain</country>
    <country>United Kingdom</country>
    <country>United States</country>
  </location_countries>
  <verification_date>November 2020</verification_date>
  <study_first_submitted>July 24, 2017</study_first_submitted>
  <study_first_submitted_qc>July 26, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">July 27, 2017</study_first_posted>
  <last_update_submitted>October 29, 2021</last_update_submitted>
  <last_update_submitted_qc>October 29, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">November 1, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Adrenoleukodystrophy</mesh_term>
  </condition_browse>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT03231878
org study id: MT-2-01
nct id: NCT03231878
lead sponsor: 
is fda regulated drug: Yes
is fda regulated device: No
This is a Phase II/III, randomized, double-blind, placebo-controlled, multicenter, two
      parallel-group study in male patients with the AMN phenotype of X-linked adrenoleukodystrophy
      (X-ALD) to assess the efficacy and safety of MIN-102 treatment. Study sites will consist of
      specialist referral centers experienced in the management of adrenoleukodystrophy (ALD).
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Triple (Participant, Care Provider, Investigator)
measure: To evaluate the efficacy of MIN-102 on the progression of adrenomyeloneuropathy (AMN) in male patients as determined by a motor function test.
time frame: in 96 weeks
measure: To evaluate the efficacy of MIN-102 in terms of patient reported outcomes.
time frame: in 96 weeks
measure: SSPROM (Severity Score System for Progressive Myelopathy )
time frame: in 96 weeks
measure: EDSS (Expanded Disability Status Scale )
time frame: in 96 weeks
measure: Quality of life scales (Euroqol)
time frame: in 96 weeks
measure: Incidence of cerebral inflammatory lesions
time frame: in 96 weeks
arm group label: Active
arm group type: Active Comparator
arm group label: Placebo
arm group type: Placebo Comparator
intervention type: Drug
intervention name: MIN-102
description: MIN-102 treatment
arm group label: Active
intervention type: Drug
intervention name: Placebos
description: Placebo
arm group label: Placebo
criteria: 
gender: Male
minimum age: 18 Years
maximum age: 65 Years
healthy volunteers: No
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
country: France
country: Germany
country: Hungary
country: Italy
country: Netherlands
country: Spain
country: United Kingdom
country: United States
responsible party type: Sponsor
mesh term: Adrenoleukodystrophy