<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/NCT02979249</url>
  </required_header>
  <id_info>
    <org_study_id>GCO 08-0959-1001</org_study_id>
    <secondary_id>U54DK083909</secondary_id>
    <nct_id>NCT02979249</nct_id>
  </id_info>
  <brief_title>Oral Iron for Erythropoietic Protoporphyrias</brief_title>
  <acronym>EPP</acronym>
  <official_title>Effect of Oral Iron Therapy on Erythrocyte Protoporphyrin Levels in the Erythropoietic Protoporphyrias</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Icahn School of Medicine at Mount Sinai</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)</agency>
      <agency_class>NIH</agency_class>
    </collaborator>
  </sponsors>
  <source>Icahn School of Medicine at Mount Sinai</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      In the medical literature there are conflicting reports on whether iron improves symptoms in&#xD;
      patients with EPP and XLP. Giving iron to people who are iron deficient is thought to improve&#xD;
      EPP symptoms. However, this has never been systematically tested. Therefore, the purpose of&#xD;
      this study is to determine the effect of oral iron for EPP and XLP patients.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This is a clinical trial, which means its purpose is to study an intervention or treatment.&#xD;
      In this study all patients with EPP or XLP will be given a standard dose of iron pills and&#xD;
      monitored for one year. There is currently no effective Food and Drug Administration (FDA)&#xD;
      approved treatment for EPP or XLP in the US. Giving iron to patients with low ferritin (a&#xD;
      measure of body iron stores) levels may help improve their EPP symptoms by decreasing&#xD;
      erythrocyte protoporphyrin levels.&#xD;
&#xD;
      Patients will be asked come to the study site once every three months over the course of a&#xD;
      year for a total of five visits. At these visits the study doctors will check in with&#xD;
      participants and some blood and urine samples will be taken. Participants will not be charged&#xD;
      for any of the lab tests that are being done for part of this study alone. In between these&#xD;
      study visits there will be phone call to check in and see how participants are doing. All&#xD;
      patients in this study will receive iron pills at no cost to them.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date>December 2016</start_date>
  <completion_date type="Actual">July 19, 2019</completion_date>
  <primary_completion_date type="Actual">July 19, 2019</primary_completion_date>
  <phase>N/A</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Change in Erythrocyte Protoporphyrin Levels</measure>
    <time_frame>Baseline and at 12 months</time_frame>
    <description>Relative difference in erythrocyte protoporphyrin levels between baseline and 12 months after treatment</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Mean Change in EPP-specific Quality of Life Questionnaire</measure>
    <time_frame>Baseline and 12 months</time_frame>
    <description>Quality of life tool designed to capture the specific issues that patients with EPP experience. Score range is 0-100, higher scores represent higher levels or satisfaction, lower scores represent more impacted quality of life. Mean change at 12 months as compared to baseline.</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">16</enrollment>
  <condition>Erythropoietic Protoporphyria</condition>
  <condition>EPP</condition>
  <condition>X-linked Protoporphyria</condition>
  <condition>XLP</condition>
  <arm_group>
    <arm_group_label>Oral Iron</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>standard dose of iron pills</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Oral Iron</intervention_name>
    <description>for one year</description>
    <arm_group_label>Oral Iron</arm_group_label>
    <other_name>Ferrous sulfate USP</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Enrollment in the Longitudinal Study of the Porphyrias&#xD;
&#xD;
          -  Male or female age ≥18 years&#xD;
&#xD;
          -  History of nonblistering cutaneous photosensitivity&#xD;
&#xD;
          -  Biochemical findings - A marked increase in erythrocyte protoporphyrin (total&#xD;
             erythrocyte protoporphyrin &gt;400 ug/dL) with a predominance of metal-free&#xD;
             protoporphyrin&#xD;
&#xD;
          -  Serum ferritin ≤30 ng/mL at baseline&#xD;
&#xD;
          -  Able to tolerate oral iron&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  History of liver or bone marrow transplant or clinically significant liver dysfunction&#xD;
             as determined by the investigator&#xD;
&#xD;
          -  Known or suspected allergy to oral iron based on patient report&#xD;
&#xD;
          -  Clinical evidence of active and ongoing GI bleeding&#xD;
&#xD;
          -  Use of any other clinical or experimental therapy in the past 3 months&#xD;
&#xD;
          -  Individuals with elevations of porphyrins in plasma or erythrocytes due to other&#xD;
             diseases (i.e. secondary porphyrinemia) such as liver and bone marrow diseases&#xD;
&#xD;
          -  Patients with any clinically significant comorbid conditions, which in the opinion of&#xD;
             the investigator, precludes participation&#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>Manisha Balwani, MD, MS</last_name>
    <role>Study Chair</role>
    <affiliation>Icahn School of Medicine at Mount Sinai</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>University of Alabama, Birmingham</name>
      <address>
        <city>Birmingham</city>
        <state>Alabama</state>
        <zip>35294</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of California</name>
      <address>
        <city>San Francisco</city>
        <state>California</state>
        <zip>94143</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Icahn School of Medicine at Mount Sinai</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 Health Sciences</name>
      <address>
        <city>Winston-Salem</city>
        <state>North Carolina</state>
        <zip>27106</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Texas Medical Branch</name>
      <address>
        <city>Galveston</city>
        <state>Texas</state>
        <zip>77555</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Utah</name>
      <address>
        <city>Salt Lake City</city>
        <state>Utah</state>
        <zip>84132</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <link>
    <url>https://www.rarediseasesnetwork.org/cms/porphyrias/Studies</url>
    <description>Porphyrias Consortium website</description>
  </link>
  <verification_date>August 2020</verification_date>
  <study_first_submitted>November 29, 2016</study_first_submitted>
  <study_first_submitted_qc>November 30, 2016</study_first_submitted_qc>
  <study_first_posted type="Estimate">December 1, 2016</study_first_posted>
  <results_first_submitted>August 24, 2020</results_first_submitted>
  <results_first_submitted_qc>August 24, 2020</results_first_submitted_qc>
  <results_first_posted type="Actual">September 11, 2020</results_first_posted>
  <last_update_submitted>August 24, 2020</last_update_submitted>
  <last_update_submitted_qc>August 24, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">September 11, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Icahn School of Medicine at Mount Sinai</investigator_affiliation>
    <investigator_full_name>Manisha C Balwani</investigator_full_name>
    <investigator_title>Associate Professor</investigator_title>
  </responsible_party>
  <keyword>Interventional</keyword>
  <keyword>open-label</keyword>
  <keyword>EPP</keyword>
  <keyword>XLP</keyword>
  <keyword>porphyria</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Protoporphyria, Erythropoietic</mesh_term>
  </condition_browse>
  <provided_document_section>
    <provided_document>
      <document_type>Study Protocol and Statistical Analysis Plan</document_type>
      <document_has_protocol>Yes</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>Yes</document_has_sap>
      <document_date>June 14, 2017</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/49/NCT02979249/Prot_SAP_000.pdf</document_url>
    </provided_document>
  </provided_document_section>

  <clinical_results>

    <participant_flow>
      <group_list>
        <group group_id="P1">
          <title>Oral Iron</title>
          <description>standard dose of oral iron pills for one year</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="16"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="10"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="6"/>
              </participants_list>
            </milestone>
          </milestone_list>
          <drop_withdraw_reason_list>
            <drop_withdraw_reason>
              <title>Lost to Follow-up</title>
              <participants_list>
                <participants group_id="P1" count="2"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Withdrawal by Subject</title>
              <participants_list>
                <participants group_id="P1" count="4"/>
              </participants_list>
            </drop_withdraw_reason>
          </drop_withdraw_reason_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <group_list>
        <group group_id="B1">
          <title>Oral Iron</title>
          <description>standard dose of oral iron pills for one year</description>
        </group>
      </group_list>
      <analyzed_list>
        <analyzed>
          <units>Participants</units>
          <scope>Overall</scope>
          <count_list>
            <count group_id="B1" value="16"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age</title>
          <units>years</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="38.8" spread="14.5"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Sex: Female, Male</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Female</title>
                  <measurement_list>
                    <measurement group_id="B1" value="11"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="5"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Ethnicity (NIH/OMB)</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="2"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Not Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="14"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Race (NIH/OMB)</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>American Indian or Alaska Native</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Asian</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Native Hawaiian or Other Pacific Islander</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Black or African American</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>White</title>
                  <measurement_list>
                    <measurement group_id="B1" value="16"/>
                  </measurement_list>
                </category>
                <category>
                  <title>More than one race</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Change in Erythrocyte Protoporphyrin Levels</title>
        <description>Relative difference in erythrocyte protoporphyrin levels between baseline and 12 months after treatment</description>
        <time_frame>Baseline and at 12 months</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Oral Iron</title>
            <description>standard dose of oral iron pills for one year</description>
          </group>
        </group_list>
        <measure>
          <title>Change in Erythrocyte Protoporphyrin Levels</title>
          <description>Relative difference in erythrocyte protoporphyrin levels between baseline and 12 months after treatment</description>
          <units>µg/dL</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="10"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Baseline</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="2314.6" spread="1820.3"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>12 months</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="2240.4" spread="2312.6"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Mean Change in EPP-specific Quality of Life Questionnaire</title>
        <description>Quality of life tool designed to capture the specific issues that patients with EPP experience. Score range is 0-100, higher scores represent higher levels or satisfaction, lower scores represent more impacted quality of life. Mean change at 12 months as compared to baseline.</description>
        <time_frame>Baseline and 12 months</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Oral Iron</title>
            <description>standard dose of oral iron pills for one year</description>
          </group>
        </group_list>
        <measure>
          <title>Mean Change in EPP-specific Quality of Life Questionnaire</title>
          <description>Quality of life tool designed to capture the specific issues that patients with EPP experience. Score range is 0-100, higher scores represent higher levels or satisfaction, lower scores represent more impacted quality of life. Mean change at 12 months as compared to baseline.</description>
          <units>mean change in score on a scale</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="10"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="3.3" spread="18.3"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>12 months</time_frame>
      <group_list>
        <group group_id="E1">
          <title>Oral Iron</title>
          <description>standard dose of oral iron pills for one year</description>
        </group>
      </group_list>
      <serious_events>
        <default_assessment>Systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, all-cause mortality</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Eye disorders</title>
            <event_list>
              <event>
                <sub_title>Conjunctivitis</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Diarrhea</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Pregnancy, puerperium and perinatal conditions</title>
            <event_list>
              <event>
                <sub_title>Miscarriage</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>0</frequency_threshold>
        <default_assessment>Systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, other adverse events</sub_title>
                <counts group_id="E1" subjects_affected="9" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Blood and lymphatic system disorders</title>
            <event_list>
              <event>
                <sub_title>Elevated Protoporphyrin</sub_title>
                <description>greater than 35% of baseline</description>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Abdominal Pain</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Nausea</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Dyspepsia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Constipation</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Sinusitis</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Mucosal Infection</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Metabolism and nutrition disorders</title>
            <event_list>
              <event>
                <sub_title>Elevated Protoporphyrin</sub_title>
                <description>greater than 35% of baseline</description>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Renal and urinary disorders</title>
            <event_list>
              <event>
                <sub_title>Urinary Tract Pain</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Respiratory, thoracic and mediastinal disorders</title>
            <event_list>
              <event>
                <sub_title>Productive cough</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Skin and subcutaneous tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Photosensitivity</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </other_events>
    </reported_events>

    <certain_agreements>
      <pi_employee>Principal Investigators are NOT employed by the organization sponsoring the study.</pi_employee>
      <restrictive_agreement>There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed. </restrictive_agreement>
    </certain_agreements>
    <point_of_contact>
      <name_or_title>Dr. Hetanshi Naik</name_or_title>
      <organization>Icahn School of Medicine at Mount Sinai</organization>
      <phone>212-659-8512</phone>
      <email>hetanshi.naik@mssm.edu</email>
    </point_of_contact>
  </clinical_results>
</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/NCT02979249
org study id: GCO 08-0959-1001
secondary id: U54DK083909
nct id: NCT02979249
lead sponsor: 
collaborator: 
has dmc: Yes
In the medical literature there are conflicting reports on whether iron improves symptoms in
      patients with EPP and XLP. Giving iron to people who are iron deficient is thought to improve
      EPP symptoms. However, this has never been systematically tested. Therefore, the purpose of
      this study is to determine the effect of oral iron for EPP and XLP patients.
This is a clinical trial, which means its purpose is to study an intervention or treatment.
      In this study all patients with EPP or XLP will be given a standard dose of iron pills and
      monitored for one year. There is currently no effective Food and Drug Administration (FDA)
      approved treatment for EPP or XLP in the US. Giving iron to patients with low ferritin (a
      measure of body iron stores) levels may help improve their EPP symptoms by decreasing
      erythrocyte protoporphyrin levels.

      Patients will be asked come to the study site once every three months over the course of a
      year for a total of five visits. At these visits the study doctors will check in with
      participants and some blood and urine samples will be taken. Participants will not be charged
      for any of the lab tests that are being done for part of this study alone. In between these
      study visits there will be phone call to check in and see how participants are doing. All
      patients in this study will receive iron pills at no cost to them.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Change in Erythrocyte Protoporphyrin Levels
time frame: Baseline and at 12 months
description: Relative difference in erythrocyte protoporphyrin levels between baseline and 12 months after treatment
measure: Mean Change in EPP-specific Quality of Life Questionnaire
time frame: Baseline and 12 months
description: Quality of life tool designed to capture the specific issues that patients with EPP experience. Score range is 0-100, higher scores represent higher levels or satisfaction, lower scores represent more impacted quality of life. Mean change at 12 months as compared to baseline.
arm group label: Oral Iron
arm group type: Experimental
description: standard dose of iron pills
intervention type: Drug
intervention name: Oral Iron
description: for one year
arm group label: Oral Iron
other name: Ferrous sulfate USP
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Manisha Balwani, MD, MS
role: Study Chair
affiliation: Icahn School of Medicine at Mount Sinai
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
country: United States
url: https://www.rarediseasesnetwork.org/cms/porphyrias/Studies
description: Porphyrias Consortium website
responsible party type: Principal Investigator
investigator affiliation: Icahn School of Medicine at Mount Sinai
investigator full name: Manisha C Balwani
investigator title: Associate Professor
mesh term: Protoporphyria, Erythropoietic
provided document: 
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
point of contact: 