<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/NCT03817528</url>
  </required_header>
  <id_info>
    <org_study_id>7716</org_study_id>
    <nct_id>NCT03817528</nct_id>
  </id_info>
  <brief_title>ITI-007 (Lumateperone Tosylate) for Schizophrenia</brief_title>
  <official_title>ITI-007 (Lumateperone Tosylate) for Schizophrenia</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>New York State Psychiatric Institute</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Intra-Cellular Therapies, Inc.</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
  </sponsors>
  <source>New York State Psychiatric Institute</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this study is to offer open label ITI-007 treatment to patients who poorly&#xD;
      respond or poorly tolerate approved medications.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Patients will be started on ITI-007 and current medication will be slowly discontinued within&#xD;
      the first 7 days of starting ITI-007, with some flexibility allowed if clinically indicated.&#xD;
      No patients will be left unmedicated because of this study.&#xD;
&#xD;
      Patients will be seen weekly for the first 4 weeks, biweekly for the second month and then&#xD;
      monthly for six months. Patients will be monitored by clinical and safety rating scales, and&#xD;
      will be required to show improvement after 3 months to remain in this study. Patients not&#xD;
      improving at this time will be assessed for the risks/benefits of continuing.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Terminated</overall_status>
  <why_stopped>
    ITI-007 approved by FDA&#xD;
  </why_stopped>
  <start_date type="Actual">March 1, 2019</start_date>
  <completion_date type="Actual">September 23, 2020</completion_date>
  <primary_completion_date type="Actual">September 23, 2020</primary_completion_date>
  <phase>Phase 2</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>
    <intervention_model_description>open-label</intervention_model_description>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
    <masking_description>None (open label)</masking_description>
  </study_design_info>
  <primary_outcome>
    <measure>Schizophrenia Symptoms</measure>
    <time_frame>Change from baseline in Total PANSS score after 6 month treatment</time_frame>
    <description>schizophrenia symptoms will be measures using the Positive and Negative Symptom Scale (PANSS) Total PANSS score (range: 30-210). Individual items scored from 1(absent) to 7 (extremely severe). Total PANSS score is sum of the 30 individual items with lowest score (30) indicating all symptoms absent and the maximum score (210) indicating all symptoms rated as extremely severe.</description>
  </primary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">4</enrollment>
  <condition>Schizophrenia</condition>
  <arm_group>
    <arm_group_label>ITI-007</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Open-Label ITI-007 40-60 mg</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>ITI-007</intervention_name>
    <description>ITI-007 (Lumateperone tosylate)dosed 40-60 mg based on efficacy/adverse events</description>
    <arm_group_label>ITI-007</arm_group_label>
    <other_name>Lumateperone tosylate</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Diagnostic and Statistical Manual of Mental Disorders, 5th edition (DSM-5) diagnosis&#xD;
             of schizophrenia or schizoaffective disorder&#xD;
&#xD;
          -  Has capacity to provide informed consent&#xD;
&#xD;
          -  Medically stable for study participation&#xD;
&#xD;
          -  Judged clinically not to be at significant suicide or violence risk&#xD;
&#xD;
          -  Inadequate response or tolerability to previously antipsychotic therapy, as defined by&#xD;
             at least one of the following: prior clozapine failure, a PANSS&gt;80 despite at least&#xD;
             six weeks of a current antipsychotic therapy, a Clinical Global Impressions&#xD;
             scale-Improvement (CGI-I) of 4 after at least two six week trials of antipsychotics&#xD;
             (retrospective assessment) or failure to tolerate an adequate dose of at least&#xD;
             antipsychotics (as defined by the Physicians Desk Reference)&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Substance abuse within last 90 days&#xD;
&#xD;
          -  ECG abnormality that is clinically significant&#xD;
&#xD;
          -  Pregnancy, lactation, or lack of use of effective birth control&#xD;
&#xD;
          -  Presence or positive history of significant unstable medical or neurological illness&#xD;
             (including any history of seizure disorder, hepatitis, renal insufficiency or mental&#xD;
             retardation), history of HIV&#xD;
&#xD;
          -  Clinically significant abnormal laboratory tests, positive for hepatitis B or C or&#xD;
             liver function tests (LFTs) &gt; 2x Upper Limit of Normal, use of strong CYP3A4&#xD;
             inhibitors or inducers&#xD;
&#xD;
          -  History or presence of concomitant major psychiatric illness.&#xD;
&#xD;
          -  Use of other antipsychotic medications at baseline.&#xD;
&#xD;
          -  Use of another investigational medication in the previous 4 weeks&#xD;
      </textblock>
    </criteria>
    <gender>Male</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>60 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Jeffrey A Lieberman, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>New York State Psychiatric Institute</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>New York State Psychiatric Institute</name>
      <address>
        <city>New York</city>
        <state>New York</state>
        <zip>10032</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>November 2021</verification_date>
  <study_first_submitted>November 30, 2018</study_first_submitted>
  <study_first_submitted_qc>January 23, 2019</study_first_submitted_qc>
  <study_first_posted type="Actual">January 25, 2019</study_first_posted>
  <results_first_submitted>September 9, 2021</results_first_submitted>
  <results_first_submitted_qc>October 5, 2021</results_first_submitted_qc>
  <results_first_posted type="Actual">November 2, 2021</results_first_posted>
  <last_update_submitted>November 2, 2021</last_update_submitted>
  <last_update_submitted_qc>November 2, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">November 4, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>New York State Psychiatric Institute</investigator_affiliation>
    <investigator_full_name>Jeffrey A. Lieberman, MD</investigator_full_name>
    <investigator_title>Director, New York State Psychiatric Institute</investigator_title>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Schizophrenia</mesh_term>
  </condition_browse>
  <provided_document_section>
    <provided_document>
      <document_type>Study Protocol</document_type>
      <document_has_protocol>Yes</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>No</document_has_sap>
      <document_date>October 16, 2019</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/28/NCT03817528/Prot_000.pdf</document_url>
    </provided_document>
  </provided_document_section>

  <clinical_results>

    <participant_flow>
      <recruitment_details>Enrollment reflects the number of participants were deemed eligible and received study drug</recruitment_details>
      <pre_assignment_details>Participants underwent a screening phase to determine eligibility to receive study medication</pre_assignment_details>
      <group_list>
        <group group_id="P1">
          <title>ITI-007</title>
          <description>Open-Label ITI-007 40-60 mg&#xD;
ITI-007: ITI-007 (Lumateperone tosylate)dosed 40-60 mg based on efficacy/adverse events</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="4"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="3"/>
              </participants_list>
            </milestone>
          </milestone_list>
          <drop_withdraw_reason_list>
            <drop_withdraw_reason>
              <title>Withdrawal by Subject</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Adverse Event</title>
              <participants_list>
                <participants group_id="P1" count="2"/>
              </participants_list>
            </drop_withdraw_reason>
          </drop_withdraw_reason_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <population>Baseline population is participants who received at least one dose of study drug</population>
      <group_list>
        <group group_id="B1">
          <title>ITI-007</title>
          <description>Open-Label ITI-007 40-60 mg&#xD;
ITI-007: ITI-007 (Lumateperone tosylate)dosed 40-60 mg based on efficacy/adverse events</description>
        </group>
      </group_list>
      <analyzed_list>
        <analyzed>
          <units>Participants</units>
          <scope>Overall</scope>
          <count_list>
            <count group_id="B1" value="4"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>&lt;=18 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Between 18 and 65 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="4"/>
                  </measurement_list>
                </category>
                <category>
                  <title>&gt;=65 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </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="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="4"/>
                  </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="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Not Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="4"/>
                  </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="1"/>
                  </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="3"/>
                  </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>
          <title>Region of Enrollment</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <title>United States</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="4"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Schizophrenia Symptoms</title>
        <description>schizophrenia symptoms will be measures using the Positive and Negative Symptom Scale (PANSS) Total PANSS score (range: 30-210). Individual items scored from 1(absent) to 7 (extremely severe). Total PANSS score is sum of the 30 individual items with lowest score (30) indicating all symptoms absent and the maximum score (210) indicating all symptoms rated as extremely severe.</description>
        <time_frame>Change from baseline in Total PANSS score after 6 month treatment</time_frame>
        <population>Completed participants (6 months of treatment). Outcome measure reported as change in PANSS score from baseline to end of treatment</population>
        <group_list>
          <group group_id="O1">
            <title>ITI-007</title>
            <description>Open-Label ITI-007 40-60 mg&#xD;
ITI-007: ITI-007 (Lumateperone tosylate)dosed 40-60 mg based on efficacy/adverse events</description>
          </group>
        </group_list>
        <measure>
          <title>Schizophrenia Symptoms</title>
          <description>schizophrenia symptoms will be measures using the Positive and Negative Symptom Scale (PANSS) Total PANSS score (range: 30-210). Individual items scored from 1(absent) to 7 (extremely severe). Total PANSS score is sum of the 30 individual items with lowest score (30) indicating all symptoms absent and the maximum score (210) indicating all symptoms rated as extremely severe.</description>
          <population>Completed participants (6 months of treatment). Outcome measure reported as change in PANSS score from baseline to end of treatment</population>
          <units>change in PANSS score from baseline</units>
          <param>Number</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="1"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="11"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>Adverse events to be reported from time of consent (whether received study drug or not) to 30 days after completion of study treatment (up to 8 months)</time_frame>
      <desc>AE/SAE collection from all individuals who signed informed consent (total: 8)</desc>
      <group_list>
        <group group_id="E1">
          <title>ITI-007</title>
          <description>Open-Label ITI-007 40-60 mg&#xD;
ITI-007: ITI-007 (Lumateperone tosylate)dosed 40-60 mg based on efficacy/adverse events</description>
        </group>
      </group_list>
      <serious_events>
        <default_assessment>Non-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="8"/>
              </event>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="8"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Psychiatric disorders</title>
            <event_list>
              <event>
                <sub_title>worsening of symptoms</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="8"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>0</frequency_threshold>
        <default_assessment>Non-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="1" subjects_at_risk="8"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>vomiting</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="8"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Psychiatric disorders</title>
            <event_list>
              <event>
                <sub_title>hypomania</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="8"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </other_events>
    </reported_events>

    <certain_agreements>
      <pi_employee>All Principal Investigators ARE 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>Marlene Carlson, MPH</name_or_title>
      <organization>New York State Psychiatric Institute</organization>
      <phone>646-774-5340</phone>
      <email>marlene.carlson@nyspi.columbia.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/NCT03817528
org study id: 7716
nct id: NCT03817528
lead sponsor: 
collaborator: 
has dmc: No
is fda regulated drug: Yes
is fda regulated device: No
The purpose of this study is to offer open label ITI-007 treatment to patients who poorly
      respond or poorly tolerate approved medications.
Patients will be started on ITI-007 and current medication will be slowly discontinued within
      the first 7 days of starting ITI-007, with some flexibility allowed if clinically indicated.
      No patients will be left unmedicated because of this study.

      Patients will be seen weekly for the first 4 weeks, biweekly for the second month and then
      monthly for six months. Patients will be monitored by clinical and safety rating scales, and
      will be required to show improvement after 3 months to remain in this study. Patients not
      improving at this time will be assessed for the risks/benefits of continuing.
allocation: N/A
intervention model: Single Group Assignment
intervention model description: open-label
primary purpose: Treatment
masking: None (Open Label)
masking description: None (open label)
measure: Schizophrenia Symptoms
time frame: Change from baseline in Total PANSS score after 6 month treatment
description: schizophrenia symptoms will be measures using the Positive and Negative Symptom Scale (PANSS) Total PANSS score (range: 30-210). Individual items scored from 1(absent) to 7 (extremely severe). Total PANSS score is sum of the 30 individual items with lowest score (30) indicating all symptoms absent and the maximum score (210) indicating all symptoms rated as extremely severe.
arm group label: ITI-007
arm group type: Experimental
description: Open-Label ITI-007 40-60 mg
intervention type: Drug
intervention name: ITI-007
description: ITI-007 (Lumateperone tosylate)dosed 40-60 mg based on efficacy/adverse events
arm group label: ITI-007
other name: Lumateperone tosylate
criteria: 
gender: Male
minimum age: 18 Years
maximum age: 60 Years
healthy volunteers: No
last name: Jeffrey A Lieberman, MD
role: Principal Investigator
affiliation: New York State Psychiatric Institute
facility: 
country: United States
responsible party type: Principal Investigator
investigator affiliation: New York State Psychiatric Institute
investigator full name: Jeffrey A. Lieberman, MD
investigator title: Director, New York State Psychiatric Institute
mesh term: Schizophrenia
provided document: 
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
point of contact: 