Clinical trial NCT06041841: 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
Overall status: NOT_YET_RECRUITING
Phase: PHASE2
Conditions: POMC Deficiency Obesity
PCSK1 Deficiency Obesity
LEPR Deficiency Obesity
Lead sponsor: LG Chem
Sponsor class: INDUSTRY
First posted: 2023-09-18
Enrollment: 4
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: This is a study where each participant participates for up to 56 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
Primary purpose: TREATMENT

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 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.

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

Primary outcomes:
measure: Change of BMI
timeFrame: From baseline to Week 16
Secondary outcomes:
measure: Frequency and severity of adverse events (AE)
timeFrame: From first dose up to Week 52
measure: Frequency and severity of adverse events of special interest (AESI)
timeFrame: From first dose up to Week 52
measure: Mean change and mean percentage change from baseline in body weight
timeFrame: From baseline up to Week 52
measure: Mean change and mean percentage change from baseline in waist circumference.
timeFrame: From baseline up to Week 52
measure: Mean change and mean percentage change from baseline in Hunger Questionnaire Scores
timeFrame: From baseline up to Week 52
measure: Mean change and mean percentage change from baseline in body composition assessed by dual energy x-ray absorptiometry
timeFrame: From baseline up to Week 52
Other outcomes:
Locations:
city: Paris
country: France
facility: Pitié Salpêtrière hospital and Sorbonne Université
city: Cambridge
country: United Kingdom
facility: Cambridge university
Study officials:
References: