<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/NCT04402489</url>
  </required_header>
  <id_info>
    <org_study_id>MT-7117-G01</org_study_id>
    <nct_id>NCT04402489</nct_id>
  </id_info>
  <brief_title>Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria or X-Linked Protoporphyria</brief_title>
  <official_title>A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Adults and Adolescents With Erythropoietic Protoporphyria or X-Linked Protoporphyria</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Mitsubishi Tanabe Pharma America Inc.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Mitsubishi Tanabe Pharma America Inc.</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>
      The primary objective of this study is to investigate the efficacy of MT-7117 on time to&#xD;
      onset and severity of first prodromal symptoms (burning, tingling, or stinging) associated&#xD;
      with sunlight exposure in subjects with EPP or XLP aged 12-75.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">June 1, 2020</start_date>
  <completion_date type="Actual">July 26, 2022</completion_date>
  <primary_completion_date type="Actual">December 14, 2021</primary_completion_date>
  <phase>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>Change from baseline in average daily sunlight exposure time (minutes) to first prodromal symptom (burning, tingling, itching, or stinging) associated with sunlight exposure between 1 hour post sunrise and 1 hour pre-sunset at Week 26.</measure>
    <time_frame>Baseline (Week 0) and 26 weeks</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>Patient Global Impression of Change (PGIC).</measure>
    <time_frame>Week 26</time_frame>
    <description>PGIC: Scale from 1 to 7, where 7 is worse.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Total number of sunlight-induced pain events with pain rating of 1-10 on the Likert scale during the 26-week double-blind treatment period.</measure>
    <time_frame>Baseline (Week 0) and Week 26</time_frame>
  </secondary_outcome>
  <other_outcome>
    <measure>Change from baseline for total score in the domain of pain intensity in the PROMIS-57.</measure>
    <time_frame>Baseline (Week 0) and Week 26</time_frame>
    <description>Pain intensity: 0 to 10, where 10 is worst pain imaginable.</description>
  </other_outcome>
  <other_outcome>
    <measure>The percentage of subjects who are responders based on average daily sunlight exposure time to first prodromal symptom associated with sunlight exposure between 1 hour post sunrise and 1 hour pre-sunset defined by within-subject meaningful change.</measure>
    <time_frame>Week 26</time_frame>
  </other_outcome>
  <other_outcome>
    <measure>Change from baseline for total score in the domain of physical function in the PROMIS-57.</measure>
    <time_frame>Baseline (Week 0) and Week 26</time_frame>
    <description>Physical function: 1-5, where 5 is without any difficulty.</description>
  </other_outcome>
  <number_of_arms>3</number_of_arms>
  <enrollment type="Actual">184</enrollment>
  <condition>EPP</condition>
  <condition>XLP</condition>
  <arm_group>
    <arm_group_label>Placebo Comparator</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>Oral tablet of placebo once a day.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>MT-7117 Low Dose</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Oral tablet of MT-7117 Low Dose once a day.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>MT-7117 High Dose</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Oral tablet of MT-7117 High Dose once a day.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Placebo</intervention_name>
    <description>Placebo</description>
    <arm_group_label>Placebo Comparator</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>MT-7117 Low Dose</intervention_name>
    <description>MT-7117 Low Dose</description>
    <arm_group_label>MT-7117 Low Dose</arm_group_label>
    <other_name>Dersimelagon</other_name>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>MT-7117 High Dose</intervention_name>
    <description>MT-7117 High Dose</description>
    <arm_group_label>MT-7117 High Dose</arm_group_label>
    <other_name>Dersimelagon</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Additional screening criteria check may apply for qualification.&#xD;
&#xD;
        Inclusion Criteria:&#xD;
&#xD;
          1. Subjects provided written informed consent to participate. For minor subjects, both&#xD;
             minor assent and parental consent will be provided.&#xD;
&#xD;
          2. Male and female subjects with a confirmed diagnosis of EPP or XLP based on medical&#xD;
             history, aged 12 years to 75 years, inclusive, at Screening.&#xD;
&#xD;
          3. Subjects have a body weight of ≥30 kg.&#xD;
&#xD;
          4. Subjects are willing and able to travel to the study sites for all scheduled visits.&#xD;
&#xD;
          5. In the Investigator's opinion, subject is able to understand the nature of the study&#xD;
             and any risks involved in participation, and willing to cooperate and comply with the&#xD;
             protocol restrictions and requirements (including travel).&#xD;
&#xD;
          6. Female subjects who are non-lactating and have a negative urine pregnancy test at&#xD;
             baseline visit prior to receiving the first dose of study drug.&#xD;
&#xD;
          7. Female subjects of childbearing potential and male subjects with partner of&#xD;
             child-bearing potential currently using/willing to use 2 effective methods of&#xD;
             contraception including barrier method as described in the protocol.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. History or presence of photodermatoses other than EPP or XLP.&#xD;
&#xD;
          2. Subjects who are unwilling or unable to go outside during daylight hours most days&#xD;
             (e.g., between 1 hour post sunrise and 1 hour pre-sunset) during the study.&#xD;
&#xD;
          3. Presence of clinically significant hepatobiliary disease based on LFT values at&#xD;
             Screening.&#xD;
&#xD;
          4. Subjects with AST, ALT, ALP ≥3.0 × upper limit of normal (ULN) or total bilirubin &gt;1.5&#xD;
             × ULN at Screening.&#xD;
&#xD;
          5. Subjects with or having a history (in the last 2 years) of excessive alcohol intake in&#xD;
             the opinion of the Investigator.&#xD;
&#xD;
          6. History of melanoma.&#xD;
&#xD;
          7. Presence of melanoma and/or lesions suspicious for melanoma at Screening.&#xD;
&#xD;
          8. History of familial melanoma (defined as having 2 or more first-degree relatives, such&#xD;
             as parents, sibling and/or child).&#xD;
&#xD;
          9. Presence of squamous cell carcinoma, basal cell carcinoma, or other malignant skin&#xD;
             lesions.&#xD;
&#xD;
             Any suspicious lesions or nevi will be evaluated. If the suspicious lesion or nevi&#xD;
             cannot be resolved through biopsy or excision, the subject will be excluded from the&#xD;
             study.&#xD;
&#xD;
         10. History or presence of psychiatric disease judged to be clinically significant by the&#xD;
             Investigator and which may interfere with the study evaluation and/or safety of the&#xD;
             subjects.&#xD;
&#xD;
         11. Presence of clinically significant acute or chronic renal disease based upon the&#xD;
             subject's medical records including hemodialysis; and a serum creatinine level of&#xD;
             greater than 1.2 mg/dL or an estimated glomerular filtration rate (eGFR) &lt;60 ml/min.&#xD;
&#xD;
         12. Presence of any clinically significant disease or laboratory abnormality which, in the&#xD;
             opinion of the Investigator, can interfere with the study objectives and/or safety of&#xD;
             the subjects.&#xD;
&#xD;
         13. Female subjects who are pregnant, lactating, or intending to become pregnant during&#xD;
             the study.&#xD;
&#xD;
         14. Treatment with phototherapy within 3 months before Randomization (Visit 2).&#xD;
&#xD;
         15. Treatment with afamelanotide within 3 months before Randomization (Visit 2).&#xD;
&#xD;
         16. Treatment with cimetidine within 4 weeks before Randomization (Visit 2).&#xD;
&#xD;
         17. Treatment with antioxidant agents within 4 weeks before Randomization (Visit 2), at&#xD;
             doses which, in the opinion of the Investigator, may affect study endpoints (including&#xD;
             but not limited to beta-carotene, cysteine, pyridoxine).&#xD;
&#xD;
         18. Chronic treatment with any scheduled analgesic agents including, but not limited to,&#xD;
             opioids and opioid derivatives such as morphine, hydrocodone, oxycodone, fentanyl, or&#xD;
             their combination with other unscheduled analgesics or non-steroidal anti-inflammatory&#xD;
             drug (Percocet and Vicodin-like prescription drugs) within 4 weeks before&#xD;
             Randomization (Visit 2).&#xD;
&#xD;
             Acute use of scheduled narcotics greater than 3 months prior to randomization, OTCs,&#xD;
             such as NSAIDs or aspirin for analgesia, or prior temporary use of scheduled agents&#xD;
             within 3 months of screening are not excluded.&#xD;
&#xD;
         19. Treatment with any drugs or supplements which, in the opinion of the Investigator, can&#xD;
             interfere with the objectives of the study or safety of the subjects.&#xD;
&#xD;
         20. Previous exposure to MT-7117 (this does not include placebo treated subjects).&#xD;
&#xD;
         21. Previous treatment with any investigational agent within 12 weeks before Screening OR&#xD;
             5 half-lives of the investigational product (whichever is longer).&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>12 Years</minimum_age>
    <maximum_age>75 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Head of Medical Science</last_name>
    <role>Study Director</role>
    <affiliation>Mitsubishi Tanabe Pharma America Inc.</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Marvel Clinical Research, LLC</name>
      <address>
        <city>Huntington Beach</city>
        <state>California</state>
        <zip>92647</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University Of Miami School Of Medicine, Center For Liver Diseases</name>
      <address>
        <city>Miami</city>
        <state>Florida</state>
        <zip>33136</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>MetroBoston Clinical Partners, LLC</name>
      <address>
        <city>Brighton</city>
        <state>Massachusetts</state>
        <zip>02135</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Kansas City Research Institute</name>
      <address>
        <city>Kansas City</city>
        <state>Missouri</state>
        <zip>64131</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Icahn School of Medicine at Mount Sinai (ISMMS) - The Mount Sinai Hospital (MSH)</name>
      <address>
        <city>New York</city>
        <state>New York</state>
        <zip>10029</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Wake Forest University Baptist Health</name>
      <address>
        <city>Winston-Salem</city>
        <state>North Carolina</state>
        <zip>27109</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Remington-Davis Clinical Research</name>
      <address>
        <city>Columbus</city>
        <state>Ohio</state>
        <zip>43215</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Thomas Jefferson University</name>
      <address>
        <city>Philadelphia</city>
        <state>Pennsylvania</state>
        <zip>19107</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The University of Texas Medical Branch (UTMB)</name>
      <address>
        <city>Galveston</city>
        <state>Texas</state>
        <zip>77555</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Washington-Seattle Cancer Care Alliance</name>
      <address>
        <city>Seattle</city>
        <state>Washington</state>
        <zip>19023</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The Wesley Hospital</name>
      <address>
        <city>Brisbane</city>
        <state>Queensland</state>
        <zip>4066</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Royal Melbourne Hospital (RMH)</name>
      <address>
        <city>Melbourne</city>
        <state>Victoria</state>
        <zip>3050</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Alberta</name>
      <address>
        <city>Edmonton</city>
        <state>Alberta</state>
        <zip>AB T6G 2R3</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Westfaelische Wilhelms-Universitaet Muenster</name>
      <address>
        <city>Muenster</city>
        <state>Northrhein Westalien</state>
        <zip>48149</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Charite - Universitaetsmedizin Berlin</name>
      <address>
        <city>Berlin</city>
        <zip>10117</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Azienda Ospedaliera Spedali Civili di Brescia-Universita degli Studi Di Brescia</name>
      <address>
        <city>Brescia</city>
        <zip>1 25123</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano</name>
      <address>
        <city>Milan</city>
        <zip>20122</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>U.O.C. Medicina Interna Azienda ospedaliero Universitaria Policlinico di Modena</name>
      <address>
        <city>Modena</city>
        <zip>41125</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>I.F.O Hospital Centro Porfirie e Malattie Rare</name>
      <address>
        <city>Rome</city>
        <zip>144 Rome RM</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Kobe University Hospital</name>
      <address>
        <city>Kobe</city>
        <state>Hyogo</state>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Sophia Dermatology Clinic</name>
      <address>
        <city>Kanazawa</city>
        <state>Ishikawa</state>
        <zip>921-8035</zip>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigator site</name>
      <address>
        <city>Osakasayama</city>
        <state>Osaka</state>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Osaka Medical College Hospital</name>
      <address>
        <city>Takatsuki</city>
        <state>Osaka</state>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Tokyo Saiseikai Central Hospital</name>
      <address>
        <city>Minato-ku</city>
        <state>Tokyo</state>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Toyama University Hospital</name>
      <address>
        <city>Sugitani</city>
        <state>Toyama</state>
        <zip>930-0194</zip>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Haukeland University Hospital</name>
      <address>
        <city>Bergen</city>
        <country>Norway</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital Clínic de Barcelona</name>
      <address>
        <city>Barcelona</city>
        <zip>08036</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital Universitario 12 de Octubre</name>
      <address>
        <city>Madrid</city>
        <zip>28041</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Karolinska University Hospital</name>
      <address>
        <city>Stockholm</city>
        <zip>171 64</zip>
        <country>Sweden</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Manchester</name>
      <address>
        <city>Salford</city>
        <state>Manchester</state>
        <zip>M13 9PL</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Evelina London Children's Hospital - Guy's &amp; St Thomas' NHS Foundation Trust</name>
      <address>
        <city>London</city>
        <zip>SE1 7EH</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Australia</country>
    <country>Canada</country>
    <country>Germany</country>
    <country>Italy</country>
    <country>Japan</country>
    <country>Norway</country>
    <country>Spain</country>
    <country>Sweden</country>
    <country>United Kingdom</country>
    <country>United States</country>
  </location_countries>
  <removed_countries>
    <country>Finland</country>
  </removed_countries>
  <verification_date>May 2023</verification_date>
  <study_first_submitted>May 20, 2020</study_first_submitted>
  <study_first_submitted_qc>May 20, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">May 26, 2020</study_first_posted>
  <disposition_first_submitted>December 5, 2022</disposition_first_submitted>
  <disposition_first_submitted_qc>December 5, 2022</disposition_first_submitted_qc>
  <disposition_first_posted type="Actual">December 12, 2022</disposition_first_posted>
  <last_update_submitted>May 12, 2023</last_update_submitted>
  <last_update_submitted_qc>May 12, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">May 15, 2023</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>Protoporphyria, Erythropoietic</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>Undecided</sharing_ipd>
  </patient_data>
  <!-- Results have not yet been posted, but a results disposition has been submitted           -->
</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/NCT04402489
org study id: MT-7117-G01
nct id: NCT04402489
lead sponsor: 
is fda regulated drug: Yes
is fda regulated device: No
The primary objective of this study is to investigate the efficacy of MT-7117 on time to
      onset and severity of first prodromal symptoms (burning, tingling, or stinging) associated
      with sunlight exposure in subjects with EPP or XLP aged 12-75.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Triple (Participant, Care Provider, Investigator)
measure: Change from baseline in average daily sunlight exposure time (minutes) to first prodromal symptom (burning, tingling, itching, or stinging) associated with sunlight exposure between 1 hour post sunrise and 1 hour pre-sunset at Week 26.
time frame: Baseline (Week 0) and 26 weeks
measure: Patient Global Impression of Change (PGIC).
time frame: Week 26
description: PGIC: Scale from 1 to 7, where 7 is worse.
measure: Total number of sunlight-induced pain events with pain rating of 1-10 on the Likert scale during the 26-week double-blind treatment period.
time frame: Baseline (Week 0) and Week 26
measure: Change from baseline for total score in the domain of pain intensity in the PROMIS-57.
time frame: Baseline (Week 0) and Week 26
description: Pain intensity: 0 to 10, where 10 is worst pain imaginable.
measure: The percentage of subjects who are responders based on average daily sunlight exposure time to first prodromal symptom associated with sunlight exposure between 1 hour post sunrise and 1 hour pre-sunset defined by within-subject meaningful change.
time frame: Week 26
measure: Change from baseline for total score in the domain of physical function in the PROMIS-57.
time frame: Baseline (Week 0) and Week 26
description: Physical function: 1-5, where 5 is without any difficulty.
arm group label: Placebo Comparator
arm group type: Placebo Comparator
description: Oral tablet of placebo once a day.
arm group label: MT-7117 Low Dose
arm group type: Experimental
description: Oral tablet of MT-7117 Low Dose once a day.
arm group label: MT-7117 High Dose
arm group type: Experimental
description: Oral tablet of MT-7117 High Dose once a day.
intervention type: Drug
intervention name: Placebo
description: Placebo
arm group label: Placebo Comparator
intervention type: Drug
intervention name: MT-7117 Low Dose
description: MT-7117 Low Dose
arm group label: MT-7117 Low Dose
other name: Dersimelagon
intervention type: Drug
intervention name: MT-7117 High Dose
description: MT-7117 High Dose
arm group label: MT-7117 High Dose
other name: Dersimelagon
criteria: 
gender: All
minimum age: 12 Years
maximum age: 75 Years
healthy volunteers: No
last name: Head of Medical Science
role: Study Director
affiliation: Mitsubishi Tanabe Pharma America Inc.
facility: 
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country: Australia
country: Canada
country: Germany
country: Italy
country: Japan
country: Norway
country: Spain
country: Sweden
country: United Kingdom
country: United States
country: Finland
responsible party type: Sponsor
mesh term: Protoporphyria, Erythropoietic
sharing ipd: Undecided