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

Study Overview
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Brief Summary
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The goal of this trial is to determine the effect of LB54640 on weight reduction, hunger, and quality of life in patients 12 years of age and older with Hypothalamic Obesity (HO). To determine how well LB54640 works and how safe it is, patients with HO will take a daily dosing of either LB54640 or placebo and complete trial assessments for up to 56 weeks.

Official Title
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A Randomized, Placebo-controlled, Double-Blind Phase 2 Study to Assess Efficacy and Safety of LB54640 in Patients With Hypothalamic Obesity, With an Open-Label Extension

Conditions
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Hypothalamic Obesity

Intervention / Treatment
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* Drug: LB54640
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Documented evidence of acquired hypothalamic obesity (HO) Age 12 years and older Weight gain associated with the hypothalamic injury and a BMI of ≥30 kg/m2 for patients ≥18 years of age or BMI ≥95th percentile for age and sex for patients 12 to <18 years of age Agree to use a highly effective form of contraception throughout the study and for 90 days after the study Exclusion Criteria: Weight loss >2% in the previous 2 months for patients Use of any medication that is approved to treat obesity within 3 months of first dose of study drug. History of major surgical procedure within 30 days HbA1c >10.9% Fasting glucose level >270 mg/dL Diagnosis of severe psychiatric disorders; any suicidal ideation, attempt or behavior History or close family history of skin cancer or melanoma

Ages Eligible for Study
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Minimum Age: 12 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: LB54640 Low dose<br>Participants will be randomized to receive LB54640 low dose, middle dose, or high dose, or placebo by oral administration in a 1:1:1:1 ratio. | Drug: LB54640<br>* Oral daily administration<br>|
| Experimental: LB54640 Middle dose<br>Participants will be randomized to receive LB54640 low dose, middle dose, or high dose, or placebo by oral administration in a 1:1:1:1 ratio. | Drug: LB54640<br>* Oral daily administration<br>|
| Experimental: LB54640 High dose<br>Participants will be randomized to receive LB54640 low dose, middle dose, or high dose, or placebo by oral administration in a 1:1:1:1 ratio. | Drug: LB54640<br>* Oral daily administration<br>|
| Placebo Comparator: Placebo<br>Participants will be randomized to receive LB54640 low dose, middle dose, or high dose, or placebo by oral administration in a 1:1:1:1 ratio. | Drug: Placebo<br>* Placebo matched to LB54640<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change of BMI |  | From baseline to Week 14 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 first dose up to Week 52 | 
| Mean change and mean percentage change from baseline in waist circumference. |  | From first dose up to Week 52 | 
| 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 first dose 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 first dose up to Week 52 |