<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/NCT03038633</url>
  </required_header>
  <id_info>
    <org_study_id>34696</org_study_id>
    <nct_id>NCT03038633</nct_id>
  </id_info>
  <brief_title>A Trial to Evaluate the Safety and Tolerability of a Novel Medical Food for Management of Iron Deficiency Anemia</brief_title>
  <official_title>A Trial to Evaluate the Safety and Tolerability of a Novel Medical Food for Management of Iron Deficiency Anemia</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Milton S. Hershey Medical Center</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Milton S. Hershey Medical Center</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>
    <is_us_export>Yes</is_us_export>
  </oversight_info>
  <brief_summary>
    <textblock>
      This is a Phase 1 clinical trial designed to evaluate the safety and tolerability of a novel&#xD;
      medical food utilizing a nutritional strain of yeast for management of Iron Deficiency&#xD;
      Anemia.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      The investigational product is a medical food and is the result of discoveries following&#xD;
      extensive research on two preclinical models carried out by a team of researchers at Penn&#xD;
      State University. This study will utilize the standard &quot;3+3&quot; rule-based dose-escalation&#xD;
      schemes, which use predetermined dose levels and cohorts of three patients. The total time&#xD;
      commitment for each subject is approximately three months.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">May 2014</start_date>
  <completion_date type="Actual">June 2015</completion_date>
  <primary_completion_date type="Actual">June 2015</primary_completion_date>
  <phase>Phase 1</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Non-Randomized</allocation>
    <intervention_model>Crossover Assignment</intervention_model>
    <intervention_model_description>Individuals will receive 300 mg capsules of the novel medical food. The iron content of these capsules is 7.4mg. Our initial dose is set at 22.2 mg of iron. This dose was chosen as the initial starting dose because it is within the range of the RDA for iron for adult women (18mg). Thus this dose is considered the minimum that would be utilized in any setting for maintenance of a healthy iron status. The dosing increases to a maximum of 88.8mg of iron per day which is significantly below the standard amount of iron (165-195mg per day) recommended for an iron deficient individual or the amount of iron given intravenously (1000 mg) for some iron deficient conditions. As mentioned, the total element iron given a patient treated with FAS is up to 190mg of iron. Assuming a 10% absorption rate these patients get 19mg of iron. So if our goal is approximately 19mg of iron absorbed per day and we assume a 50% absorption rate then our target dose is 6-9 capsules per day (44.4-66.6 mg iron).</intervention_model_description>
    <primary_purpose>Other</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Dose-limiting toxicity, (DLT)</measure>
    <time_frame>28 Days</time_frame>
    <description>Still evaluating data</description>
  </primary_outcome>
  <number_of_arms>4</number_of_arms>
  <enrollment type="Actual">2</enrollment>
  <condition>Iron Deficiency Anemia</condition>
  <arm_group>
    <arm_group_label>Cohort 1</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>900 mg novel medical food containing 22.2mg iron&#xD;
receive 900 mg by mouth (3 capsules, one at each meal) daily; after Day 28 remain on level for additional 2 months if clinically indicated&#xD;
receive a phone call from coordinator between Day 5-9 to assess side effects and medications&#xD;
return to General Clinical Research Center, (GCRC) for a blood draw at days 14, 28, 60, and 90 while on novel medical food</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Cohort 2</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>1800 mg novel medical food containing 44.4 mg of iron&#xD;
receive 1800 mg by mouth (3 capsules, one at each meal) daily; after Day 28 remain on level for additional 2 months if clinically indicated&#xD;
receive a phone call from coordinator between Day 5-9 to assess side effects and medications&#xD;
return to GCRC for a blood draw at days 14, 28, 60, and 90 while on novel medical food</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Cohort 3</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>2700 mg novel medical food containing 66.6 mg of iron&#xD;
receive 2700 mg by mouth (3 capsules, one at each meal) daily; after Day 28 remain on level for additional 2 months if clinically indicated&#xD;
receive a phone call from coordinator between Day 5-9 to assess side effects and medications&#xD;
return to GCRC for a blood draw at days 14, 28, 60, and 90 while on novel medical food</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Cohort 4</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>3600 mg novel medical food containing 88.8 mg of iron&#xD;
receive 3600 mg by mouth (3 capsules, one at each meal) daily; after Day 28 remain on level for additional 2 months if clinically indicated&#xD;
receive a phone call from coordinator between Day 5-9 to assess side effects and medications&#xD;
return to GCRC for a blood draw at days 14, 28, 60, and 90 while on novel medical food</description>
  </arm_group>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>Novel Medical Food</intervention_name>
    <description>This medical food takes advantage of the stability and high level of solubility of the human H-ferritin protein to be absorbed by the body as an iron source; indeed H-ferritin is enriched in breast milk as the mechanism for transferring iron from mother to infants. Once the ferritin has been absorbed from the gut, uptake into each organ is regulated by a specific extracellular receptor pathway to deliver iron in the body's preferred bioavailable form.</description>
    <arm_group_label>Cohort 1</arm_group_label>
    <arm_group_label>Cohort 2</arm_group_label>
    <arm_group_label>Cohort 3</arm_group_label>
    <arm_group_label>Cohort 4</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Hemoglobin, (Hgb) 7-10 g/dL; transferrin saturation &lt;20%; and serum ferritin &lt;20ng/mL&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Pregnant or sexually-active female subjects who are of childbearing potential and who&#xD;
             are not willing to use an acceptable form of contraception (tubal ligation or&#xD;
             otherwise be incapable of pregnancy, hormonal contraceptives, spermicide plus barrier&#xD;
             or intrauterine device).&#xD;
&#xD;
          -  Present consumption of iron supplements or multivitamins must be switched to vitamins&#xD;
             not containing iron, such as the multivitamin Centrum Silver. No washout period is&#xD;
             necessary since it will be apparent from the ongoing anemia that any current&#xD;
             supplements are ineffective.&#xD;
&#xD;
          -  Current anemia not attributed to immune deficiency, (ID) (e.g. other microcytic anemia&#xD;
             or hemolytic, macrocytic, sideroblastic or myelosuppression or chemotherapy or&#xD;
             radiotherapy induced anemia).&#xD;
&#xD;
          -  Active malignancy within 1 year. Basal or squamous cell skin cancer is not&#xD;
             exclusionary.&#xD;
&#xD;
          -  Aspartate Aminotransferase, (AST) or Alanine Aminotransferase, (ALT) at screening&#xD;
             greater than 1.5 times the upper limit of normal.&#xD;
&#xD;
          -  Known positive hepatitis B with evidence of active hepatitis.&#xD;
&#xD;
          -  Known positive HIV-1/HIV-2 antibodies (anti-HIV).&#xD;
&#xD;
          -  Patient has a current diagnosis of asthma and is actively using an anti-asthmatic&#xD;
             therapy.&#xD;
&#xD;
          -  Received an investigational drug within 30 days of screening.&#xD;
&#xD;
          -  Hemochromatosis or other iron storage disorders.&#xD;
&#xD;
          -  Unregulated hypertension&#xD;
&#xD;
          -  Chronic kidney disease.&#xD;
&#xD;
          -  Chronic inflammatory condition including but not limited to Lupus and Rheumatoid&#xD;
             Arthritis.&#xD;
&#xD;
          -  Significant cardiovascular disease, including but not limited to myocardial infarction&#xD;
             or unstable angina within 6 months prior to study inclusion or current history of New&#xD;
             York Heart Association, (NYHA) Class III or IV congestive heart failure.&#xD;
&#xD;
          -  Smoking&#xD;
&#xD;
          -  Irritable Bowel Syndrome&#xD;
&#xD;
          -  Any other laboratory abnormality, medical condition or psychiatric disorder which in&#xD;
             the opinion of the investigator puts the subject's disease management at risk or may&#xD;
             result in the subject being unable to comply with study requirements.&#xD;
&#xD;
          -  Breastfeeding planned on or after enrolling in the study.&#xD;
&#xD;
          -  Known allergy to yeast&#xD;
&#xD;
          -  Currently on Monoamine oxidase inhibitors, (MAOI's) or Demerol&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>80 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>Penn State Milton S. Hershey Medical Center</name>
      <address>
        <city>Hershey</city>
        <state>Pennsylvania</state>
        <zip>17033</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>January 2017</verification_date>
  <study_first_submitted>October 27, 2015</study_first_submitted>
  <study_first_submitted_qc>January 30, 2017</study_first_submitted_qc>
  <study_first_posted type="Estimate">January 31, 2017</study_first_posted>
  <last_update_submitted>January 30, 2017</last_update_submitted>
  <last_update_submitted_qc>January 30, 2017</last_update_submitted_qc>
  <last_update_posted type="Estimate">January 31, 2017</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Milton S. Hershey Medical Center</investigator_affiliation>
    <investigator_full_name>Jeffrey J. Pu, MD, PhD</investigator_full_name>
    <investigator_title>Assistant Proffesor of Medicine</investigator_title>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Anemia</mesh_term>
    <mesh_term>Anemia, Iron-Deficiency</mesh_term>
    <mesh_term>Iron Deficiencies</mesh_term>
    <mesh_term>Deficiency Diseases</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>Undecided</sharing_ipd>
    <ipd_description>Data under review</ipd_description>
  </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/NCT03038633
org study id: 34696
nct id: NCT03038633
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
is us export: Yes
This is a Phase 1 clinical trial designed to evaluate the safety and tolerability of a novel
      medical food utilizing a nutritional strain of yeast for management of Iron Deficiency
      Anemia.
The investigational product is a medical food and is the result of discoveries following
      extensive research on two preclinical models carried out by a team of researchers at Penn
      State University. This study will utilize the standard "3+3" rule-based dose-escalation
      schemes, which use predetermined dose levels and cohorts of three patients. The total time
      commitment for each subject is approximately three months.
allocation: Non-Randomized
intervention model: Crossover Assignment
intervention model description: Individuals will receive 300 mg capsules of the novel medical food. The iron content of these capsules is 7.4mg. Our initial dose is set at 22.2 mg of iron. This dose was chosen as the initial starting dose because it is within the range of the RDA for iron for adult women (18mg). Thus this dose is considered the minimum that would be utilized in any setting for maintenance of a healthy iron status. The dosing increases to a maximum of 88.8mg of iron per day which is significantly below the standard amount of iron (165-195mg per day) recommended for an iron deficient individual or the amount of iron given intravenously (1000 mg) for some iron deficient conditions. As mentioned, the total element iron given a patient treated with FAS is up to 190mg of iron. Assuming a 10% absorption rate these patients get 19mg of iron. So if our goal is approximately 19mg of iron absorbed per day and we assume a 50% absorption rate then our target dose is 6-9 capsules per day (44.4-66.6 mg iron).
primary purpose: Other
masking: None (Open Label)
measure: Dose-limiting toxicity, (DLT)
time frame: 28 Days
description: Still evaluating data
arm group label: Cohort 1
arm group type: Experimental
description: 900 mg novel medical food containing 22.2mg iron
receive 900 mg by mouth (3 capsules, one at each meal) daily; after Day 28 remain on level for additional 2 months if clinically indicated
receive a phone call from coordinator between Day 5-9 to assess side effects and medications
return to General Clinical Research Center, (GCRC) for a blood draw at days 14, 28, 60, and 90 while on novel medical food
arm group label: Cohort 2
arm group type: Experimental
description: 1800 mg novel medical food containing 44.4 mg of iron
receive 1800 mg by mouth (3 capsules, one at each meal) daily; after Day 28 remain on level for additional 2 months if clinically indicated
receive a phone call from coordinator between Day 5-9 to assess side effects and medications
return to GCRC for a blood draw at days 14, 28, 60, and 90 while on novel medical food
arm group label: Cohort 3
arm group type: Experimental
description: 2700 mg novel medical food containing 66.6 mg of iron
receive 2700 mg by mouth (3 capsules, one at each meal) daily; after Day 28 remain on level for additional 2 months if clinically indicated
receive a phone call from coordinator between Day 5-9 to assess side effects and medications
return to GCRC for a blood draw at days 14, 28, 60, and 90 while on novel medical food
arm group label: Cohort 4
arm group type: Experimental
description: 3600 mg novel medical food containing 88.8 mg of iron
receive 3600 mg by mouth (3 capsules, one at each meal) daily; after Day 28 remain on level for additional 2 months if clinically indicated
receive a phone call from coordinator between Day 5-9 to assess side effects and medications
return to GCRC for a blood draw at days 14, 28, 60, and 90 while on novel medical food
intervention type: Other
intervention name: Novel Medical Food
description: This medical food takes advantage of the stability and high level of solubility of the human H-ferritin protein to be absorbed by the body as an iron source; indeed H-ferritin is enriched in breast milk as the mechanism for transferring iron from mother to infants. Once the ferritin has been absorbed from the gut, uptake into each organ is regulated by a specific extracellular receptor pathway to deliver iron in the body's preferred bioavailable form.
arm group label: Cohort 1
arm group label: Cohort 2
arm group label: Cohort 3
arm group label: Cohort 4
criteria: 
gender: All
minimum age: 18 Years
maximum age: 80 Years
healthy volunteers: No
facility: 
country: United States
responsible party type: Principal Investigator
investigator affiliation: Milton S. Hershey Medical Center
investigator full name: Jeffrey J. Pu, MD, PhD
investigator title: Assistant Proffesor of Medicine
mesh term: Anemia
mesh term: Anemia, Iron-Deficiency
mesh term: Iron Deficiencies
mesh term: Deficiency Diseases
sharing ipd: Undecided
ipd description: Data under review