<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/NCT03278899</url>
  </required_header>
  <id_info>
    <org_study_id>NV1205-008</org_study_id>
    <nct_id>NCT03278899</nct_id>
  </id_info>
  <brief_title>A Study to Prospectively Assess Disease Progression in Male Children With X-ALD</brief_title>
  <official_title>A Non-interventional Study to Prospectively Assess Baseline Status and Disease Progression in Male Children With X-Linked Adrenoleukodystrophy</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>NeuroVia, Inc.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>NeuroVia, Inc.</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      This is a non-interventional, multi-center study that follows general principles of periodic&#xD;
      assessment of X-ALD patients in routine practice. No study drug treatment will be given and&#xD;
      no changes to patient treatment are necessary.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This is a non-interventional, multi-center study that follows general principles of periodic&#xD;
      assessment of X-ALD patients in routine practice. No study drug treatment will be given and&#xD;
      no changes to the patient treatment are necessary.&#xD;
&#xD;
      Patients with X-ALD will be recruited and invited to attend a baseline visit. After eligible&#xD;
      patients are enrolled, retrospective and baseline data will be collected. After enrollment,&#xD;
      patients will be seen approximately every 6 months until they meet withdrawal criteria or the&#xD;
      Sponsor terminates the study.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Unknown status</overall_status>
  <last_known_status>Recruiting</last_known_status>
  <start_date type="Actual">February 16, 2018</start_date>
  <completion_date type="Anticipated">June 1, 2021</completion_date>
  <primary_completion_date type="Anticipated">June 1, 2021</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Cohort</observational_model>
    <time_perspective>Prospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Loes score</measure>
    <time_frame>baseline and 24 weeks</time_frame>
    <description>Percent change from baseline in brain lesions assessed as Loes score will be calculated</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Plasma VLCFA levels</measure>
    <time_frame>baseline and 24 weeks</time_frame>
    <description>Change from baseline plasma VLCFA levels</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Neurological symptoms</measure>
    <time_frame>baseline and 24 weeks</time_frame>
    <description>Development of new neurological symptoms throughout the study</description>
  </primary_outcome>
  <enrollment type="Anticipated">100</enrollment>
  <condition>X-Linked Adrenoleukodystrophy</condition>
  <biospec_retention>Samples Without DNA</biospec_retention>
  <biospec_descr>
    <textblock>
      Biospecimen retained: Plasma&#xD;
    </textblock>
  </biospec_descr>
  <eligibility>
    <study_pop>
      <textblock>
        Patients will be selected from participating sites, referring physicians, and outreach&#xD;
        communities&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Signed informed consent by patient's parent(s)/legally acceptable representative(s).&#xD;
             In addition, signed children's assent form according to local requirements.&#xD;
&#xD;
          -  Males patients 2-13 years of age with a confirmed diagnosis of X-ALD that fall into&#xD;
             one of the following:&#xD;
&#xD;
               -  Asymptomatic patients without MRI evidence of cerebral involvement&#xD;
&#xD;
               -  Patients with MRI evidence of cerebral involvement (Loes score ≥0.5) with or&#xD;
                  without clinical symptoms&#xD;
&#xD;
               -  Patient who have HSCT within 3 months from enrollment&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Patients who are 14 years of age or older&#xD;
&#xD;
          -  Patients who are in a vegetative state&#xD;
&#xD;
          -  Patients (or their guardians) who are unwilling or unable to comply with the study&#xD;
             procedures&#xD;
&#xD;
          -  Patients who received HSCT more than 3 months before enrollment&#xD;
      </textblock>
    </criteria>
    <gender>Male</gender>
    <gender_based>Yes</gender_based>
    <gender_description>Males with a confirmed diagnosis of X-ALD with or without MRI evidence of cerebral involvement</gender_description>
    <minimum_age>2 Years</minimum_age>
    <maximum_age>13 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>John Henderson, MD</last_name>
    <role>Study Director</role>
    <affiliation>NeuroVia, Inc.</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Program Director</last_name>
    <phone>415-870-6967</phone>
    <email>patients@neurovia-inc.com</email>
  </overall_contact>
  <location>
    <facility>
      <name>Stanford University</name>
      <address>
        <city>Palo Alto</city>
        <state>California</state>
        <zip>94304</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Dalia Pena-Solorzano</last_name>
      <phone>650-498-9732</phone>
      <email>dpena25@stanford.edu</email>
    </contact>
    <contact_backup>
      <last_name>Sweta Patnaik</last_name>
      <phone>650-721-1458</phone>
      <email>sweta@stanford.edu</email>
    </contact_backup>
    <investigator>
      <last_name>Maura Ruzhnikov, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Massachusetts General Hospital</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02114</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Camille Corre</last_name>
      <phone>617-724-6374</phone>
      <email>ccorre@partners.org</email>
    </contact>
    <investigator>
      <last_name>Florian S Eichler, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>University of Pittsburgh</name>
      <address>
        <city>Pittsburgh</city>
        <state>Pennsylvania</state>
        <zip>15224</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Jodi Martin</last_name>
      <phone>412-692-6351</phone>
    </contact>
    <contact_backup>
      <last_name>Mary Branamann</last_name>
      <phone>412-692-6350</phone>
    </contact_backup>
    <investigator>
      <last_name>Maria L Escolar, MD, MS</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>May 2018</verification_date>
  <study_first_submitted>August 30, 2017</study_first_submitted>
  <study_first_submitted_qc>September 7, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">September 12, 2017</study_first_posted>
  <last_update_submitted>August 7, 2018</last_update_submitted>
  <last_update_submitted_qc>August 7, 2018</last_update_submitted_qc>
  <last_update_posted type="Actual">August 8, 2018</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Adrenoleukodystropy</keyword>
  <keyword>X-ALD</keyword>
  <keyword>ALD</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Adrenoleukodystrophy</mesh_term>
    <mesh_term>Disease Progression</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <!-- 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/NCT03278899
org study id: NV1205-008
nct id: NCT03278899
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
This is a non-interventional, multi-center study that follows general principles of periodic
      assessment of X-ALD patients in routine practice. No study drug treatment will be given and
      no changes to patient treatment are necessary.
This is a non-interventional, multi-center study that follows general principles of periodic
      assessment of X-ALD patients in routine practice. No study drug treatment will be given and
      no changes to the patient treatment are necessary.

      Patients with X-ALD will be recruited and invited to attend a baseline visit. After eligible
      patients are enrolled, retrospective and baseline data will be collected. After enrollment,
      patients will be seen approximately every 6 months until they meet withdrawal criteria or the
      Sponsor terminates the study.
observational model: Cohort
time perspective: Prospective
measure: Loes score
time frame: baseline and 24 weeks
description: Percent change from baseline in brain lesions assessed as Loes score will be calculated
measure: Plasma VLCFA levels
time frame: baseline and 24 weeks
description: Change from baseline plasma VLCFA levels
measure: Neurological symptoms
time frame: baseline and 24 weeks
description: Development of new neurological symptoms throughout the study
Biospecimen retained: Plasma
study pop: 
sampling method: Probability Sample
criteria: 
gender: Male
gender based: Yes
gender description: Males with a confirmed diagnosis of X-ALD with or without MRI evidence of cerebral involvement
minimum age: 2 Years
maximum age: 13 Years
healthy volunteers: No
last name: John Henderson, MD
role: Study Director
affiliation: NeuroVia, Inc.
last name: Program Director
phone: 415-870-6967
email: patients@neurovia-inc.com
facility: 
status: Recruiting
contact: 
contact backup: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
contact backup: 
investigator: 
country: United States
responsible party type: Sponsor
mesh term: Adrenoleukodystrophy
mesh term: Disease Progression
sharing ipd: No