Back to resultsA Phase 2 Study to Assess Efficacy and Safety of LB54640 in Patients With Genetic Obesity
Primary Purpose
POMC Deficiency Obesity, PCSK1 Deficiency Obesity, LEPR Deficiency Obesity
Status
Not yet recruiting
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
LB54640
Sponsored by
About this trial
This is an interventional treatment trial for POMC Deficiency Obesity
Eligibility Criteria
Inclusion Criteria: Participants aged ≥12 years at the time of enrollment and diagnosed with genetic obesity due to POMC, PCSK1, or LEPR deficiency mutations. Obesity is defined as BMI ≥30 kg/m2 for participants ≥18 years of age or BMI ≥95th percentile for age and gender for participants <18 years of age. Exclusion Criteria: Participants with recent (within 2 months, prior to Screening and up to enrollment) intensive diet/exercise regimen that resulted in >2% weight loss, use of medication to treat obesity (within 3 months of first dose of LB54640 with certain exceptions), or weight loss surgery within 6 months prior to Screening or prior surgery which resulted in >10% weight loss with no evidence of weight regain are excluded History of major surgical procedure Weight loss surgery within the previous 6 months Any history of a suicide attempt, or any suicidal behavior HbA1c >10.9% Fasting glucose level >270 mg/dL
Sites / Locations
- Pitié Salpêtrière hospital and Sorbonne Université
- Cambridge university
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
POMC/PCSK1/LEPR cohort
Arm Description
LB54640 once daily by oral administration
Outcomes
Primary Outcome Measures
Change of BMI
Secondary Outcome Measures
Frequency and severity of adverse events (AE)
Frequency and severity of adverse events of special interest (AESI)
Mean change and mean percentage change from baseline in body weight
Mean change and mean percentage change from baseline in waist circumference.
Mean change and mean percentage change from baseline in Hunger Questionnaire Scores
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).
Mean change and mean percentage change from baseline in body composition assessed by dual energy x-ray absorptiometry
Fat mass and lean mass will be measured through dual energy x-ray absorptiometry
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT06041841
Brief Title
A Phase 2 Study to Assess Efficacy and Safety of LB54640 in Patients With Genetic Obesity
Official Title
A Phase 2, Single-arm, Open-label Study to Assess Efficacy and Safety of LB54640 in Patients With Obesity Due to POMC, PCSK1, or LEPR Deficiency.
Study Type
Interventional
2. Study Status
Record Verification Date
September 2023
Overall Recruitment Status
Not yet recruiting
Study Start Date
December 2023 (Anticipated)
Primary Completion Date
December 2024 (Anticipated)
Study Completion Date
December 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
LG Chem
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The purpose of the study was to assess the efficacy of LB54640 in participants with rare genetic disorders of obesity
Detailed Description
Patients with obesity due to POMC, PCSK1 or LEPR deficiency will be enrolled to receive LB54640 once daily by oral administration for 16 weeks. After 16 weeks of treatment, the participant may enter a long term extension phase and receive treatment for up to 1 year.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
POMC Deficiency Obesity, PCSK1 Deficiency Obesity, LEPR Deficiency Obesity
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Model Description
This is a study where each participant participates for 20 weeks. The study consists up to 7 visits to study center which include a Screening period, treatment period, and a follow-up period. 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 at the same dose level from the treatment period. The long-term extension period will be followed by a follow-up period
Masking
None (Open Label)
Allocation
N/A
Enrollment
4 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
POMC/PCSK1/LEPR cohort
Arm Type
Experimental
Arm Description
LB54640 once daily by oral administration
Intervention Type
Drug
Intervention Name(s)
LB54640
Intervention Description
LB54640 QD Oral
Primary Outcome Measure Information:
Title
Change of BMI
Time Frame
From baseline to Week 16
Secondary Outcome Measure Information:
Title
Frequency and severity of adverse events (AE)
Time Frame
From first dose up to Week 52
Title
Frequency and severity of adverse events of special interest (AESI)
Time Frame
From first dose up to Week 52
Title
Mean change and mean percentage change from baseline in body weight
Time Frame
From baseline up to Week 52
Title
Mean change and mean percentage change from baseline in waist circumference.
Time Frame
From baseline up to Week 52
Title
Mean change and mean percentage change from baseline in Hunger Questionnaire Scores
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).
Time Frame
From baseline up to Week 52
Title
Mean change and mean percentage change from baseline in body composition assessed by dual energy x-ray absorptiometry
Description
Fat mass and lean mass will be measured through dual energy x-ray absorptiometry
Time Frame
From baseline up to Week 52
10. Eligibility
Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Participants aged ≥12 years at the time of enrollment and diagnosed with genetic obesity due to POMC, PCSK1, or LEPR deficiency mutations.
Obesity is defined as BMI ≥30 kg/m2 for participants ≥18 years of age or BMI ≥95th percentile for age and gender for participants <18 years of age.
Exclusion Criteria:
Participants with recent (within 2 months, prior to Screening and up to enrollment) intensive diet/exercise regimen that resulted in >2% weight loss, use of medication to treat obesity (within 3 months of first dose of LB54640 with certain exceptions), or weight loss surgery within 6 months prior to Screening or prior surgery which resulted in >10% weight loss with no evidence of weight regain are excluded
History of major surgical procedure
Weight loss surgery within the previous 6 months
Any history of a suicide attempt, or any suicidal behavior
HbA1c >10.9%
Fasting glucose level >270 mg/dL
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
LG CHEM
Phone
+82-2-3777-1114
Email
pathway@lgchem.com
Facility Information:
Facility Name
Pitié Salpêtrière hospital and Sorbonne Université
City
Paris
Country
France
Facility Name
Cambridge university
City
Cambridge
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
A Phase 2 Study to Assess Efficacy and Safety of LB54640 in Patients With Genetic Obesity
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