xml: <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/NCT05994534</url>
    </required_header>
    <id_info>
      <org_study_id>C-23-01</org_study_id>
      <nct_id>NCT05994534</nct_id>
    </id_info>
    <brief_title>PK and PD Study of NPI-001 and Cysteamine Bitartrate</brief_title>
    <acronym>INCA</acronym>
    <official_title>A Phase 1/2 Pharmacokinetic and Pharmacodynamic Study of NPI-001 Oral Solution Compared to Cysteamine Bitartrate in Cystinosis Patients</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>Nacuity Pharmaceuticals, Inc.</agency>
        <agency_class>Industry</agency_class>
      </lead_sponsor>
    </sponsors>
    <source>Nacuity Pharmaceuticals, Inc.</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>
        Safety, pharmacokinetics, and pharmacodynamics of NPI-001 oral solution in cystinosis&#xD;
        patients compared with cysteamine.&#xD;
      </textblock>
    </brief_summary>
    <detailed_description>
      <textblock>
        This study will examine the safety, pharmacokinetics, and pharmacodynamics of NPI-001 oral&#xD;
        solution in cystinosis patients, aged ≥ 10 years. The ability of NPI-001 to reduce white&#xD;
        blood cell (WBC) cystine will be assessed and compared with cysteamine.&#xD;
      </textblock>
    </detailed_description>
    <overall_status>Not yet recruiting</overall_status>
    <start_date type="Anticipated">October 1, 2023</start_date>
    <completion_date type="Anticipated">April 30, 2024</completion_date>
    <primary_completion_date type="Anticipated">December 21, 2023</primary_completion_date>
    <phase>Phase 1/Phase 2</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>
      <primary_purpose>Treatment</primary_purpose>
      <masking>None (Open Label)</masking>
    </study_design_info>
    <primary_outcome>
      <measure>Concentration of Cystine Levels Over Time</measure>
      <time_frame>1 day</time_frame>
      <description>White Blood Cell cystine concentration over 6 hours</description>
    </primary_outcome>
    <number_of_arms>2</number_of_arms>
    <enrollment type="Anticipated">12</enrollment>
    <condition>Cystinosis</condition>
    <arm_group>
      <arm_group_label>cysteamine</arm_group_label>
      <arm_group_type>Active Comparator</arm_group_type>
      <description>Single dose, tablets in current treatment dose</description>
    </arm_group>
    <arm_group>
      <arm_group_label>NPI-001</arm_group_label>
      <arm_group_type>Experimental</arm_group_type>
      <description>Single dose, NPI-001 (N-acetylcysteine amide) oral solution at molar equivalent of current cysteamine dose.</description>
    </arm_group>
    <intervention>
      <intervention_type>Drug</intervention_type>
      <intervention_name>Cysteamine Bitartrate</intervention_name>
      <description>Single dose, tablets at current therapeutic dose</description>
      <arm_group_label>cysteamine</arm_group_label>
      <other_name>Cystagon</other_name>
    </intervention>
    <intervention>
      <intervention_type>Drug</intervention_type>
      <intervention_name>N-Acetylcysteine Amide</intervention_name>
      <description>Single dose, oral solution</description>
      <arm_group_label>NPI-001</arm_group_label>
      <other_name>NPI-001</other_name>
    </intervention>
    <eligibility>
      <criteria>
        <textblock>
          Inclusion Criteria:&#xD;
  &#xD;
            1. Males or females, any race, ≥ 10 years of age.&#xD;
  &#xD;
            2. Diagnosis of nephropathic cystinosis and able to cease cysteamine therapy for 2 days.&#xD;
  &#xD;
            3. Females will be nonpregnant and nonlactating, and females of childbearing potential&#xD;
               and males will agree to use contraception as detailed in the protocol.&#xD;
  &#xD;
            4. Able to comprehend and willing to sign an informed consent /assent form and to abide&#xD;
               by the study restrictions (travel as necessary, clinical phase 1 unit or similar for&#xD;
               up to 3 days).&#xD;
  &#xD;
          Exclusion Criteria:&#xD;
  &#xD;
            1. Have undergone kidney transplantation.&#xD;
  &#xD;
            2. Are receiving dialysis treatment.&#xD;
  &#xD;
            3. History of significant hypersensitivity to NAC or any ingredient of NPI-001 oral&#xD;
               solution.&#xD;
  &#xD;
            4. Participation in a clinical study involving administration of an investigational drug&#xD;
               (new chemical entity) in the 30 days prior to Day 1.&#xD;
  &#xD;
            5. Inability to provide blood samples, including difficulty with venous access.&#xD;
  &#xD;
            6. Subjects who, in the opinion of the Investigator and/or Sponsor (or designee), should&#xD;
               not participate in this study.&#xD;
        </textblock>
      </criteria>
      <gender>All</gender>
      <minimum_age>10 Years</minimum_age>
      <maximum_age>N/A</maximum_age>
      <healthy_volunteers>No</healthy_volunteers>
    </eligibility>
    <overall_official>
      <last_name>Hugh McCarthy, PhD, FRACP</last_name>
      <role>Principal Investigator</role>
      <affiliation>Sydney Children's Health Network</affiliation>
    </overall_official>
    <overall_contact>
      <last_name>Jami Kern, PhD</last_name>
      <phone>+1-817-336-3000</phone>
      <email>info@nacuity.com</email>
    </overall_contact>
    <verification_date>August 2023</verification_date>
    <study_first_submitted>August 8, 2023</study_first_submitted>
    <study_first_submitted_qc>August 8, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">August 16, 2023</study_first_posted>
    <last_update_submitted>August 15, 2023</last_update_submitted>
    <last_update_submitted_qc>August 15, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">August 18, 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>Cystinosis</mesh_term>
    </condition_browse>
    <intervention_browse>
      <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
      <mesh_term>Acetylcysteine</mesh_term>
      <mesh_term>N-monoacetylcystine</mesh_term>
      <mesh_term>Cysteamine</mesh_term>
    </intervention_browse>
    <patient_data>
      <sharing_ipd>No</sharing_ipd>
      <ipd_description>Individual participant data will not be made available to other researchers.</ipd_description>
    </patient_data>
    <!-- Results have not yet been posted for this study                                          -->
  </clinical_study>
  
  
text: 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/NCT05994534
  org study id: C-23-01
  nct id: NCT05994534
  lead sponsor: 
  has dmc: No
  is fda regulated drug: No
  is fda regulated device: No
  Safety, pharmacokinetics, and pharmacodynamics of NPI-001 oral solution in cystinosis
        patients compared with cysteamine.
  This study will examine the safety, pharmacokinetics, and pharmacodynamics of NPI-001 oral
        solution in cystinosis patients, aged ≥ 10 years. The ability of NPI-001 to reduce white
        blood cell (WBC) cystine will be assessed and compared with cysteamine.
  allocation: Non-Randomized
  intervention model: Crossover Assignment
  primary purpose: Treatment
  masking: None (Open Label)
  measure: Concentration of Cystine Levels Over Time
  time frame: 1 day
  description: White Blood Cell cystine concentration over 6 hours
  arm group label: cysteamine
  arm group type: Active Comparator
  description: Single dose, tablets in current treatment dose
  arm group label: NPI-001
  arm group type: Experimental
  description: Single dose, NPI-001 (N-acetylcysteine amide) oral solution at molar equivalent of current cysteamine dose.
  intervention type: Drug
  intervention name: Cysteamine Bitartrate
  description: Single dose, tablets at current therapeutic dose
  arm group label: cysteamine
  other name: Cystagon
  intervention type: Drug
  intervention name: N-Acetylcysteine Amide
  description: Single dose, oral solution
  arm group label: NPI-001
  other name: NPI-001
  criteria: 
  gender: All
  minimum age: 10 Years
  maximum age: N/A
  healthy volunteers: No
  last name: Hugh McCarthy, PhD, FRACP
  role: Principal Investigator
  affiliation: Sydney Children's Health Network
  last name: Jami Kern, PhD
  phone: +1-817-336-3000
  email: info@nacuity.com
  responsible party type: Sponsor
  mesh term: Cystinosis
  mesh term: Acetylcysteine
  mesh term: N-monoacetylcystine
  mesh term: Cysteamine
  sharing ipd: No
  ipd description: Individual participant data will not be made available to other researchers.