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

• Sponsor: LG Chem (Industry)
• Overall Status: Not yet recruiting
• CT.gov ID: NCT06041841
• Collaborator: (none)
• Enrollment: 4
• Locations: 2
• Arm: 1
• Anticipated Duration (Months): 24
• Patients Per Site: 2
• Patients Per Site Per Month: 0.1

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
Genetic Obesity; POMC Deficiency Obesity; PCSK1 Deficiency Obesity; LEPR Deficiency Obesity	Drug: LB54640	Phase 2

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.

Study Design

• Study Type: Interventional
• Anticipated Enrollment: 4 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Intervention 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 periodThis 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)
• Primary Purpose: Treatment
• 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.
• Anticipated Study Start Date: Dec 1, 2023
• Anticipated Primary Completion Date: Dec 1, 2024
• Anticipated Study Completion Date: Dec 1, 2025

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures

1. Change of BMI [From baseline to Week 16]

   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)

Secondary Outcome Measures

1. Frequency and severity of adverse events (AE) [From first dose up to Week 52]

   This will be measured frequency and severity of AEs

2. Frequency and severity of adverse events of special interest (AESI) [From first dose up to Week 52]

   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.

3. Mean change and mean percentage change from baseline in body weight [From baseline up to Week 52]

   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.

4. 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. [From baseline up to Week 52]

   Waist circumference (cm) will be measured with a tape measure in a standing position. The waist circumference should be measured twice.

5. Mean change and mean percentage change from baseline in Hunger Questionnaire Scores [From baseline up to Week 52]

   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).

6. Mean change and mean percentage change from baseline in body composition assessed by dual energy x-ray absorptiometry [From baseline up to Week 52]

   Fat mass and lean mass will be measured through dual energy x-ray absorptiometry

Eligibility Criteria

Criteria

• Ages Eligible for Study: 12 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Pitié Salpêtrière hospital and Sorbonne Université	Paris		France
2	Cambridge university	Cambridge		United Kingdom

Sponsors and Collaborators

• LG Chem

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: LG Chem
• ClinicalTrials.gov Identifier: NCT06041841
• Other Study ID Numbers: LG-MCCL004
• First Posted: Sep 18, 2023
• Last Update Posted: Sep 22, 2023
• Last Verified: Sep 1, 2023

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Obesity

Study Results

No Results Posted as of Sep 22, 2023