<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/NCT01701531</url>
  </required_header>
  <id_info>
    <org_study_id>RBC100312</org_study_id>
    <nct_id>NCT01701531</nct_id>
  </id_info>
  <brief_title>Red Blood Cell Precursor Formulation to Determine Increased Production</brief_title>
  <acronym>RBCPF</acronym>
  <official_title>An Open-Label, Pilot Study of a Red Blood Cell Precursor Formulation to Determine Increased Production in Subjects With Mild to Moderate Anemia</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Targeted Medical Pharma</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Targeted Medical Pharma</source>
  <brief_summary>
    <textblock>
      The objective of this study is to measure the change in hemoglobin levels after the&#xD;
      administration of an amino acid based, RBC precursor formulation.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Medical foods are a distinct FDA regulatory category different from single molecule chemical&#xD;
      pharmaceuticals, and from dietary supplements. The FDA has regulated amino acid preparations&#xD;
      as drugs since the 1940s as they can elicit pharmacologic effects similar to conventional&#xD;
      single molecule pharmaceuticals. The best known amino acid preparations are used to treat&#xD;
      conditions such as maple syrup disease and phenylketonuria (PKU). An official definition and&#xD;
      categorization of medical foods was made in 1988 as part of the Orphan Drug Act. Medical&#xD;
      foods are regulated similarly to drugs except they do not require pre-approval because all&#xD;
      ingredients are found on the FDA's generally recognized as safe (GRAS) (Sections 201(s) and&#xD;
      409 of the Federal Food, Drug, and Cosmetic Act) list and claims are confined to the&#xD;
      nutritional management of a specific disease. Medical food claims must be supported by&#xD;
      recognized scientific data as determined by medical evaluation. A GRAS substance is&#xD;
      distinguished from a food additive on the basis of the common knowledge about the safety of&#xD;
      the substance for its intended use. The standard for an ingredient to achieve GRAS status&#xD;
      requires not only technical demonstration of non-toxicity and safety, but also general&#xD;
      recognition of safety through widespread usage and agreement of that safety by experts in the&#xD;
      field. Many ingredients have been determined by the FDA to be GRAS, and are listed as such by&#xD;
      regulation, in Volume 21 Code of Federal Regulations (CFR) Sections 182, 184, and 186.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Withdrawn</overall_status>
  <why_stopped>
    Funding not received&#xD;
  </why_stopped>
  <start_date>October 2012</start_date>
  <completion_date type="Actual">May 2014</completion_date>
  <primary_completion_date type="Actual">May 2014</primary_completion_date>
  <phase>Phase 2/Phase 3</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 hemoglobin level 2 hours after administration of the first dose.</measure>
    <time_frame>Baseline, 2 hours and 7 days</time_frame>
    <description>Subjects will have blood drawn at baseline and 2 hours after the administration of the treatment intervention. Then again, after 7 days.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>CBC w/ differential</measure>
    <time_frame>Baseline, 2 hours and 7 days</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Erythropoietin level</measure>
    <time_frame>Baseline, 2 hours and 7 days</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Reticulocytes</measure>
    <time_frame>Baseline, 2 hours and 7 days</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Iron levels</measure>
    <time_frame>Baseline, 2 hours and 7 days</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Ferritin</measure>
    <time_frame>Baseline, 2 hours and 7 days</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>IGG</measure>
    <time_frame>Baseline, 2 hours and 7 days</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>B12</measure>
    <time_frame>Baseline, 2 hours and 7 days</time_frame>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">0</enrollment>
  <condition>Anemia</condition>
  <arm_group>
    <arm_group_label>RBCPF</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Treatment intervention arm</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>RBCPF</intervention_name>
    <arm_group_label>RBCPF</arm_group_label>
    <other_name>Red blood cell precursor formulation</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. M/F patients 18 to 75 years old, non-pregnant/lactating&#xD;
&#xD;
          2. Male patients with &lt; Hemoglobin of 12.5&#xD;
&#xD;
          3. Female Patients with &lt; Hemoglobin of 11&#xD;
&#xD;
          4. Diagnosis of mild to moderate anemia by study physician&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Pregnant or unwilling to use adequate birth control for the duration of the study.&#xD;
&#xD;
          2. Unwilling or unable to sign informed consent.&#xD;
&#xD;
          3. Myocardial infarction within the last 6 months.&#xD;
&#xD;
          4. Patients currently taking an erythropoietin medication and unable to discontinue for&#xD;
             the duration of the study.&#xD;
&#xD;
          5. GI bleed in the last 6 months.&#xD;
&#xD;
          6. Inflammatory bowel disease.&#xD;
&#xD;
          7. Chronic liver disease.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>75 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Lawrence May, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Lawrence May MD, Inc</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Lawrence May, MD, Inc.</name>
      <address>
        <city>Tarzana</city>
        <state>California</state>
        <zip>91356</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>May 2016</verification_date>
  <study_first_submitted>October 3, 2012</study_first_submitted>
  <study_first_submitted_qc>October 3, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">October 5, 2012</study_first_posted>
  <last_update_submitted>May 4, 2016</last_update_submitted>
  <last_update_submitted_qc>May 4, 2016</last_update_submitted_qc>
  <last_update_posted type="Estimate">May 5, 2016</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>Anemia</mesh_term>
  </condition_browse>
  <!-- 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/NCT01701531
org study id: RBC100312
nct id: NCT01701531
lead sponsor: 
The objective of this study is to measure the change in hemoglobin levels after the
      administration of an amino acid based, RBC precursor formulation.
Medical foods are a distinct FDA regulatory category different from single molecule chemical
      pharmaceuticals, and from dietary supplements. The FDA has regulated amino acid preparations
      as drugs since the 1940s as they can elicit pharmacologic effects similar to conventional
      single molecule pharmaceuticals. The best known amino acid preparations are used to treat
      conditions such as maple syrup disease and phenylketonuria (PKU). An official definition and
      categorization of medical foods was made in 1988 as part of the Orphan Drug Act. Medical
      foods are regulated similarly to drugs except they do not require pre-approval because all
      ingredients are found on the FDA's generally recognized as safe (GRAS) (Sections 201(s) and
      409 of the Federal Food, Drug, and Cosmetic Act) list and claims are confined to the
      nutritional management of a specific disease. Medical food claims must be supported by
      recognized scientific data as determined by medical evaluation. A GRAS substance is
      distinguished from a food additive on the basis of the common knowledge about the safety of
      the substance for its intended use. The standard for an ingredient to achieve GRAS status
      requires not only technical demonstration of non-toxicity and safety, but also general
      recognition of safety through widespread usage and agreement of that safety by experts in the
      field. Many ingredients have been determined by the FDA to be GRAS, and are listed as such by
      regulation, in Volume 21 Code of Federal Regulations (CFR) Sections 182, 184, and 186.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Change in hemoglobin level 2 hours after administration of the first dose.
time frame: Baseline, 2 hours and 7 days
description: Subjects will have blood drawn at baseline and 2 hours after the administration of the treatment intervention. Then again, after 7 days.
measure: CBC w/ differential
time frame: Baseline, 2 hours and 7 days
measure: Erythropoietin level
time frame: Baseline, 2 hours and 7 days
measure: Reticulocytes
time frame: Baseline, 2 hours and 7 days
measure: Iron levels
time frame: Baseline, 2 hours and 7 days
measure: Ferritin
time frame: Baseline, 2 hours and 7 days
measure: IGG
time frame: Baseline, 2 hours and 7 days
measure: B12
time frame: Baseline, 2 hours and 7 days
arm group label: RBCPF
arm group type: Experimental
description: Treatment intervention arm
intervention type: Drug
intervention name: RBCPF
arm group label: RBCPF
other name: Red blood cell precursor formulation
criteria: 
gender: All
minimum age: 18 Years
maximum age: 75 Years
healthy volunteers: No
last name: Lawrence May, MD
role: Principal Investigator
affiliation: Lawrence May MD, Inc
facility: 
country: United States
responsible party type: Sponsor
mesh term: Anemia