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/NCT06041841</url>
    </required_header>
    <id_info>
      <org_study_id>LG-MCCL004</org_study_id>
      <nct_id>NCT06041841</nct_id>
    </id_info>
    <brief_title>LB54640 Phase 2 Treatment Trial in Patients With Rare Genetic Disorders of Obesity</brief_title>
    <official_title>A Phase 2, Single-arm, Open-label Study to Access Efficacy and Safety of LB54640 in Patients With Obesity Due to POMC, PCSK1, or LEPR Deficiency.</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>LG Chem</agency>
        <agency_class>Industry</agency_class>
      </lead_sponsor>
    </sponsors>
    <source>LG Chem</source>
    <oversight_info>
      <has_dmc>Yes</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 the study was to assess the efficacy of LB54640 on weight, hunger assessments,&#xD;
        and safety and pharmacokinetics profile in participants with rare genetic disorders of&#xD;
        obesity&#xD;
      </textblock>
    </brief_summary>
    <detailed_description>
      <textblock>
        This is a Phase 2, multicenter, open-label, single-arm study to assess the efficacy, safety,&#xD;
        and PK of LB54640 in participants with obesity due to POMC, PCSK1, or LEPR deficiency.&#xD;
        Participants with obesity and homozygous or compound heterozygous variants in POMC, LEPR, or&#xD;
        PCSK1 genes will be enrolled in the study.&#xD;
  &#xD;
        All participants will initiate treatment with LB54640 and the dose will be escalated up to a&#xD;
        maximum dose of 600 mg QD. Upon completion of treatment phase at Week 16, the participant&#xD;
        will enter a long-term extension phase and receive treatment with LB54640 for an additional&#xD;
        36 weeks.&#xD;
  &#xD;
        The total duration of the study including screening, treatment, long-term extension, and&#xD;
        follow-up periods for a participant will be 64 weeks.&#xD;
      </textblock>
    </detailed_description>
    <overall_status>Not yet recruiting</overall_status>
    <start_date type="Anticipated">December 2023</start_date>
    <completion_date type="Anticipated">December 2025</completion_date>
    <primary_completion_date type="Anticipated">December 2024</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>This is an open-label study where each participant participates for 20 weeks. &quot;Open label&quot; means that the participants and researchers both know which treatment is given. The study consists of 7 visits to study center which include a Screening period of approximately 8 weeks, treatment period of 16 weeks, and a follow-up period of 4 weeks. Eligible participants who are willing to enter the long-term extension period and who are judged by the study doctor to have no safety concerns, will continue to receive LB54640 for 36 weeks (3 additional visits) at the same dose level from the treatment period. The long-term extension period will be followed by a follow-up period of 4 weeks.</intervention_model_description>
      <primary_purpose>Treatment</primary_purpose>
      <masking>None (Open Label)</masking>
    </study_design_info>
    <primary_outcome>
      <measure>Change of BMI</measure>
      <time_frame>From baseline to Week 16</time_frame>
      <description>This will be measured in terms of changes in BMI. Body Mass Index (kg/m2&#xD;
  ) will be calculated from height and weight according to the following formula: Weight (kg)/[height (m)]2 (weight in kilograms divided by height in meters squared)</description>
    </primary_outcome>
    <secondary_outcome>
      <measure>Frequency and severity of adverse events (AE)</measure>
      <time_frame>From first dose up to Week 52</time_frame>
      <description>This will be measured frequency and severity of AEs</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Frequency and severity of adverse events of special interest (AESI)</measure>
      <time_frame>From first dose up to Week 52</time_frame>
      <description>This will be measured frequency and severity of AESIs. Adverse events of special interest are events of scientific and medical interest specific to the further understanding of the safety profile of the study intervention and require close monitoring and rapid communication by the investigators to the Sponsor.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Mean change and mean percentage change from baseline in body weight</measure>
      <time_frame>From baseline up to Week 52</time_frame>
      <description>Body weight must be measured to the nearest 0.1 kg or 0.1 pounds. The same digital scale should be used throughout the study and the scale should be calibrated on a regular basis.&#xD;
  Weight should be measured when participants are in the fasting state and without shoes; participants should have an empty bladder and must wear light clothing for weight assessment.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Mean change and mean percentage change from baseline in waist circumference. Waist circumference was measured when participants were in fasting condition and at approximately the same time at each visit.</measure>
      <time_frame>From baseline up to Week 52</time_frame>
      <description>Waist circumference (cm) will be measured with a tape measure in a standing position. The waist circumference should be measured twice.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Mean change and mean percentage change from baseline in Hunger Questionnaire Scores</measure>
      <time_frame>From baseline up to Week 52</time_frame>
      <description>The question will asked using the Hunger Questionnaire. The questionnaire consists of a new 5-item, patient-reported scale assessing major eating-related factors (worst and average hunger, appetite, craving, and satiety).</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Mean change and mean percentage change from baseline in body composition assessed by dual energy x-ray absorptiometry</measure>
      <time_frame>From baseline up to Week 52</time_frame>
      <description>Fat mass and lean mass will be measured through dual energy x-ray absorptiometry</description>
    </secondary_outcome>
    <number_of_arms>1</number_of_arms>
    <enrollment type="Anticipated">4</enrollment>
    <condition>Obesity Due to Melanocortin 4 Receptor Deficiency</condition>
    <condition>Genetic Obesity</condition>
    <condition>Obesity</condition>
    <arm_group>
      <arm_group_label>POMC/PCSK1/LEPR Homozyous or Compound Heterozygous Cohort</arm_group_label>
      <arm_group_type>Experimental</arm_group_type>
      <description>Participants with Homozyous or Compound Heterozygous POMC/PCSK1/LEPR deficiency will received LB54640 QD oral administration for 16 weeks. All participants initiated treatment with LB54640 and the dose will escalated up to a maximum dose of 600 mg QD. Upon completion of treatment phase at Week 16, the participant will enter a long-term extension phase and receive treatment with LB54640 for an additional 36 weeks.The total treatment period will be up to 52 weeks.</description>
    </arm_group>
    <intervention>
      <intervention_type>Drug</intervention_type>
      <intervention_name>LB54640</intervention_name>
      <description>LB54640 QD Oral</description>
      <arm_group_label>POMC/PCSK1/LEPR Homozyous or Compound Heterozygous Cohort</arm_group_label>
    </intervention>
    <eligibility>
      <criteria>
        <textblock>
          Inclusion Criteria:&#xD;
  &#xD;
          - Participants aged ≥12 years at the time of enrollment and diagnosed with genetic obesity&#xD;
          due to POMC, PCSK1, or LEPR deficiency mutations are eligible.&#xD;
  &#xD;
          Obesity is defined as BMI ≥30 kg/m2 for participants ≥18 years of age or BMI ≥95th&#xD;
          percentile for age and gender for participants &lt;18 years of age.&#xD;
  &#xD;
            -  POMC/PCSK1/LEPR compound heterozygous (two different mutations in gene) or homozygous&#xD;
               deficiency obesity&#xD;
  &#xD;
            -  Mutations were interpreted as pathogenic or likely pathogenic by the American College&#xD;
               of Medical Genetics and Genomics (ACMG) classification.&#xD;
  &#xD;
          Exclusion Criteria:&#xD;
  &#xD;
            -  Participants with recent (within 2 months, prior to Screening and up to enrollment)&#xD;
               intensive diet/exercise regimen that resulted in &gt;2% weight loss, use of medication to&#xD;
               treat obesity (within 3 months of first dose of LB54640 with certain exceptions), or&#xD;
               weight loss surgery within 6 months prior to Screening or prior surgery which resulted&#xD;
               in &gt;10% weight loss with no evidence of weight regain are excluded&#xD;
  &#xD;
            -  History of major surgical procedure&#xD;
  &#xD;
            -  Weight loss surgery within the previous 6 months&#xD;
  &#xD;
            -  Any history of a suicide attempt, or any suicidal behavior&#xD;
  &#xD;
            -  HbA1c &gt;10.9%&#xD;
  &#xD;
            -  Fasting glucose level &gt;270 mg/dL&#xD;
        </textblock>
      </criteria>
      <gender>All</gender>
      <minimum_age>12 Years</minimum_age>
      <maximum_age>N/A</maximum_age>
      <healthy_volunteers>No</healthy_volunteers>
    </eligibility>
    <overall_contact>
      <last_name>LG CHEM</last_name>
      <phone>+82-2-3777-1114</phone>
      <email>pathway@lgchem.com</email>
    </overall_contact>
    <location>
      <facility>
        <name>Pitié Salpêtrière hospital and Sorbonne Université</name>
        <address>
          <city>Paris</city>
          <country>France</country>
        </address>
      </facility>
    </location>
    <location>
      <facility>
        <name>Cambridge university</name>
        <address>
          <city>Cambridge</city>
          <country>United Kingdom</country>
        </address>
      </facility>
    </location>
    <location_countries>
      <country>France</country>
      <country>United Kingdom</country>
    </location_countries>
    <verification_date>August 2023</verification_date>
    <study_first_submitted>September 11, 2023</study_first_submitted>
    <study_first_submitted_qc>September 11, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">September 18, 2023</study_first_posted>
    <last_update_submitted>September 11, 2023</last_update_submitted>
    <last_update_submitted_qc>September 11, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">September 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>Obesity</mesh_term>
    </condition_browse>
    <patient_data>
      <sharing_ipd>No</sharing_ipd>
    </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/NCT06041841
  org study id: LG-MCCL004
  nct id: NCT06041841
  lead sponsor: 
  has dmc: Yes
  is fda regulated drug: Yes
  is fda regulated device: No
  The purpose of the study was to assess the efficacy of LB54640 on weight, hunger assessments,
        and safety and pharmacokinetics profile in participants with rare genetic disorders of
        obesity
  This is a Phase 2, multicenter, open-label, single-arm study to assess the efficacy, safety,
        and PK of LB54640 in participants with obesity due to POMC, PCSK1, or LEPR deficiency.
        Participants with obesity and homozygous or compound heterozygous variants in POMC, LEPR, or
        PCSK1 genes will be enrolled in the study.
  
        All participants will initiate treatment with LB54640 and the dose will be escalated up to a
        maximum dose of 600 mg QD. Upon completion of treatment phase at Week 16, the participant
        will enter a long-term extension phase and receive treatment with LB54640 for an additional
        36 weeks.
  
        The total duration of the study including screening, treatment, long-term extension, and
        follow-up periods for a participant will be 64 weeks.
  allocation: N/A
  intervention model: Single Group Assignment
  intervention model description: This is an open-label study where each participant participates for 20 weeks. "Open label" means that the participants and researchers both know which treatment is given. The study consists of 7 visits to study center which include a Screening period of approximately 8 weeks, treatment period of 16 weeks, and a follow-up period of 4 weeks. Eligible participants who are willing to enter the long-term extension period and who are judged by the study doctor to have no safety concerns, will continue to receive LB54640 for 36 weeks (3 additional visits) at the same dose level from the treatment period. The long-term extension period will be followed by a follow-up period of 4 weeks.
  primary purpose: Treatment
  masking: None (Open Label)
  measure: Change of BMI
  time frame: From baseline to Week 16
  description: This will be measured in terms of changes in BMI. Body Mass Index (kg/m2
  ) will be calculated from height and weight according to the following formula: Weight (kg)/[height (m)]2 (weight in kilograms divided by height in meters squared)
  measure: Frequency and severity of adverse events (AE)
  time frame: From first dose up to Week 52
  description: This will be measured frequency and severity of AEs
  measure: Frequency and severity of adverse events of special interest (AESI)
  time frame: From first dose up to Week 52
  description: This will be measured frequency and severity of AESIs. Adverse events of special interest are events of scientific and medical interest specific to the further understanding of the safety profile of the study intervention and require close monitoring and rapid communication by the investigators to the Sponsor.
  measure: Mean change and mean percentage change from baseline in body weight
  time frame: From baseline up to Week 52
  description: Body weight must be measured to the nearest 0.1 kg or 0.1 pounds. The same digital scale should be used throughout the study and the scale should be calibrated on a regular basis.
  Weight should be measured when participants are in the fasting state and without shoes; participants should have an empty bladder and must wear light clothing for weight assessment.
  measure: Mean change and mean percentage change from baseline in waist circumference. Waist circumference was measured when participants were in fasting condition and at approximately the same time at each visit.
  time frame: From baseline up to Week 52
  description: Waist circumference (cm) will be measured with a tape measure in a standing position. The waist circumference should be measured twice.
  measure: Mean change and mean percentage change from baseline in Hunger Questionnaire Scores
  time frame: From baseline up to Week 52
  description: The question will asked using the Hunger Questionnaire. The questionnaire consists of a new 5-item, patient-reported scale assessing major eating-related factors (worst and average hunger, appetite, craving, and satiety).
  measure: Mean change and mean percentage change from baseline in body composition assessed by dual energy x-ray absorptiometry
  time frame: From baseline up to Week 52
  description: Fat mass and lean mass will be measured through dual energy x-ray absorptiometry
  arm group label: POMC/PCSK1/LEPR Homozyous or Compound Heterozygous Cohort
  arm group type: Experimental
  description: Participants with Homozyous or Compound Heterozygous POMC/PCSK1/LEPR deficiency will received LB54640 QD oral administration for 16 weeks. All participants initiated treatment with LB54640 and the dose will escalated up to a maximum dose of 600 mg QD. Upon completion of treatment phase at Week 16, the participant will enter a long-term extension phase and receive treatment with LB54640 for an additional 36 weeks.The total treatment period will be up to 52 weeks.
  intervention type: Drug
  intervention name: LB54640
  description: LB54640 QD Oral
  arm group label: POMC/PCSK1/LEPR Homozyous or Compound Heterozygous Cohort
  criteria: 
  gender: All
  minimum age: 12 Years
  maximum age: N/A
  healthy volunteers: No
  last name: LG CHEM
  phone: +82-2-3777-1114
  email: pathway@lgchem.com
  facility: 
  facility: 
  country: France
  country: United Kingdom
  responsible party type: Sponsor
  mesh term: Obesity
  sharing ipd: No