A Phase 2 Study to Assess Efficacy and Safety of LB54640 in Patients With Genetic Obesity

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.

The purpose of the study was to assess the efficacy of LB54640 in participants with rare genetic disorders of obesity

Study Overview

• Status: Not yet recruiting
• Conditions: POMC Deficiency Obesity; PCSK1 Deficiency Obesity; LEPR Deficiency Obesity
• Intervention / Treatment: Drug: LB54640

Detailed Description

Patients with obesity due to POMC, PCSK1 or LEPR deficiency will be enrolled to receive LB54640 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.

• Study Type: Interventional
• Enrollment (Estimated): 4
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: LG CHEM; Phone Number: +82-2-3777-1114; Email: pathway@lgchem.com

Study Locations

• France
  • Paris, France
    • Pitié Salpêtrière hospital and Sorbonne Université
• United Kingdom
  • Cambridge, United Kingdom
    • Cambridge University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: POMC/PCSK1/LEPR cohort; LB54640 once daily by oral administration	Drug: LB54640; LB54640 QD Oral

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Change of BMI	From baseline to Week 16

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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).	From baseline up to Week 52
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	From baseline up to Week 52
Frequency and severity of adverse events (AE)		From first dose up to Week 52
Frequency and severity of adverse events of special interest (AESI)		From first dose up to Week 52
Mean change and mean percentage change from baseline in body weight		From baseline up to Week 52
Mean change and mean percentage change from baseline in waist circumference.		From baseline up to Week 52

Collaborators and Investigators

• Sponsor: LG Chem

Study record dates

Study Major Dates

• Study Start (Estimated): December 1, 2023
• Primary Completion (Estimated): December 1, 2024
• Study Completion (Estimated): December 1, 2025

Study Registration Dates

• First Submitted: September 11, 2023
• First Submitted That Met QC Criteria: September 11, 2023
• First Posted (Actual): September 18, 2023

Study Record Updates

• Last Update Posted (Actual): November 2, 2023
• Last Update Submitted That Met QC Criteria: October 31, 2023
• Last Verified: October 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Overnutrition; Nutrition Disorders; Overweight; Body Weight; Obesity
• Other Study ID Numbers: LG-MCCL004

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No