<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/NCT04482049</url>
  </required_header>
  <id_info>
    <org_study_id>REN001-903</org_study_id>
    <nct_id>NCT04482049</nct_id>
  </id_info>
  <brief_title>A Prospective Study of the Disease Characteristics of Adult Patients With Long Chain Fatty Acid Oxidation Disorders</brief_title>
  <acronym>FORWARD</acronym>
  <official_title>A Prospective, Multicenter, Non-interventional Study to Investigate the Disease Characteristics of Adult Patients With Long Chain Fatty Acid Oxidation Disorders (FAOD)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Reneo Pharma Ltd</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Reneo Pharma Ltd</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>
      The purpose of the study is to collect information on disease characteristics from adult&#xD;
      patients diagnosed with fatty acid oxidation disorders.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      In this study there will be no drug intervention. The study will include a Baseline visit and&#xD;
      a follow up visit scheduled at Month 4. At these visits medical history, safety assessments,&#xD;
      concomitant medications, exercise tests and quality of life questionnaire data will be&#xD;
      collected.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Active, not recruiting</overall_status>
  <start_date type="Actual">January 27, 2021</start_date>
  <completion_date type="Anticipated">May 2022</completion_date>
  <primary_completion_date type="Anticipated">May 2022</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Case-Only</observational_model>
    <time_perspective>Prospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>To evaluate change in function as measured by 12 minute walk test in adult subjects with long-chain fatty acid oxidation disorders</measure>
    <time_frame>Week 16</time_frame>
    <description>Change from baseline 12mwt</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>To evaluate change in symptoms related to FAOD using a newly developed muscle symptom questionnaire specifically designed for adult patients with long-chain FAOD</measure>
    <time_frame>Week 16</time_frame>
    <description>Change from baseline in FAOD-Muscle Symptom Inventory</description>
  </secondary_outcome>
  <enrollment type="Anticipated">60</enrollment>
  <condition>Fatty Acid Oxidation Disorder</condition>
  <eligibility>
    <study_pop>
      <textblock>
        Patients will be recruited from centres experienced in the identification and subsequent&#xD;
        management of patients LC-FAOD.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. A diagnosis of one of the following genetically confirmed:&#xD;
&#xD;
               1. Carnitine palmitoyltransferase 2 deficiency&#xD;
&#xD;
               2. Very long-chain Acyl-CoA dehydrogenase deficiency&#xD;
&#xD;
               3. Long-chain 3-hydroxyacyl-CoA dehydrogenase deficiency&#xD;
&#xD;
               4. Trifunctional protein deficiency&#xD;
&#xD;
          2. A stable treatment regimen for at least 30 days&#xD;
&#xD;
          3. Ambulatory and able to perform the study exercise test, using walking aids if&#xD;
             necessary&#xD;
&#xD;
          4. Willing and able to personally sign and date an informed consent document indicating&#xD;
             that the subject has been informed of all pertinent aspects of the study.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Unstable or poorly controlled disease as determined by one or more of the following:&#xD;
&#xD;
               1. Presence of symptoms of acute rhabdomyolysis with clinically significant&#xD;
                  elevations in serum CK&#xD;
&#xD;
               2. Evidence of acute crisis from their underlying disease&#xD;
&#xD;
          2. Currently taking a PPAR agonist&#xD;
&#xD;
          3. Have motor abnormalities other than those related to the fatty acid oxidation disorder&#xD;
             that could interfere with the study procedures, as determine by the investigator&#xD;
&#xD;
          4. Evidence of significant concomitant medical or psychiatric disease that in the opinion&#xD;
             of the Investigator may interfere with the conduct or safety of this study&#xD;
&#xD;
          5. Pregnant or nursing females&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Melanie Gillingham, PhD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Dept of Molecular and Medical Genetics, Oregon Health and Science University</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Oregon Health &amp; Science University (OHSU)</name>
      <address>
        <city>Portland</city>
        <state>Oregon</state>
        <zip>97239</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Pittsburgh</name>
      <address>
        <city>Pittsburgh</city>
        <state>Pennsylvania</state>
        <zip>15261</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Vanderbuilt University Medical Center</name>
      <address>
        <city>Nashville</city>
        <state>Tennessee</state>
        <zip>37232</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Medizinische Universität Innsbruck</name>
      <address>
        <city>Innsbruck</city>
        <zip>A-6020</zip>
        <country>Austria</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>UZ Leuven University Hospitals Leuven</name>
      <address>
        <city>Leuven</city>
        <zip>3000</zip>
        <country>Belgium</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Vseobecna fakultni nemocnice v Praze, Klinika pediatrie a dedicnych poruch metabolizmu</name>
      <address>
        <city>Prague</city>
        <zip>12808</zip>
        <country>Czechia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Rigshospitalet, Klinik for nerve- og muskelsygdomme</name>
      <address>
        <city>København</city>
        <zip>2100</zip>
        <country>Denmark</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hôpital Pitié-Salpêtrière</name>
      <address>
        <city>Paris</city>
        <zip>75013</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Pest Megyei Flór Ferenc Kórház</name>
      <address>
        <city>Kistarcsa</city>
        <zip>H-2143</zip>
        <country>Hungary</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Universita di Brescia</name>
      <address>
        <city>Brescia</city>
        <zip>25123</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Azienda Ospedaliera Universitaria Policlinico &quot;G. Martino&quot; di Messina</name>
      <address>
        <city>Messina</city>
        <zip>98124</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Uniwersyteckie Centrum Kliniczne</name>
      <address>
        <city>Gdańsk</city>
        <zip>80-952</zip>
        <country>Poland</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital 12 de Octubre</name>
      <address>
        <city>Madrid</city>
        <zip>28041</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Unidad de Diagnóstico y Tratamiento de Enfermedades Metabólicas Complejo Hospitalario Universitario de Santiago Travesía de Choupana s/n</name>
      <address>
        <city>Santiago De Compostela</city>
        <zip>15706</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Salford Royal NHS Trust</name>
      <address>
        <city>Salford</city>
        <zip>M6 8HD</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Austria</country>
    <country>Belgium</country>
    <country>Czechia</country>
    <country>Denmark</country>
    <country>France</country>
    <country>Hungary</country>
    <country>Italy</country>
    <country>Poland</country>
    <country>Spain</country>
    <country>United Kingdom</country>
    <country>United States</country>
  </location_countries>
  <removed_countries>
    <country>Germany</country>
  </removed_countries>
  <verification_date>January 2022</verification_date>
  <study_first_submitted>July 14, 2020</study_first_submitted>
  <study_first_submitted_qc>July 17, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">July 22, 2020</study_first_posted>
  <last_update_submitted>January 11, 2022</last_update_submitted>
  <last_update_submitted_qc>January 11, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">January 12, 2022</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <!-- 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/NCT04482049
org study id: REN001-903
nct id: NCT04482049
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
The purpose of the study is to collect information on disease characteristics from adult
      patients diagnosed with fatty acid oxidation disorders.
In this study there will be no drug intervention. The study will include a Baseline visit and
      a follow up visit scheduled at Month 4. At these visits medical history, safety assessments,
      concomitant medications, exercise tests and quality of life questionnaire data will be
      collected.
observational model: Case-Only
time perspective: Prospective
measure: To evaluate change in function as measured by 12 minute walk test in adult subjects with long-chain fatty acid oxidation disorders
time frame: Week 16
description: Change from baseline 12mwt
measure: To evaluate change in symptoms related to FAOD using a newly developed muscle symptom questionnaire specifically designed for adult patients with long-chain FAOD
time frame: Week 16
description: Change from baseline in FAOD-Muscle Symptom Inventory
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Melanie Gillingham, PhD
role: Principal Investigator
affiliation: Dept of Molecular and Medical Genetics, Oregon Health and Science University
facility: 
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country: Austria
country: Belgium
country: Czechia
country: Denmark
country: France
country: Hungary
country: Italy
country: Poland
country: Spain
country: United Kingdom
country: United States
country: Germany
responsible party type: Sponsor