A Study to Assess Efficacy and Safety of LB54640 in Patients With Hypothalamic Obesity
- Registration Number
- NCT06046443
- Lead Sponsor
- Rhythm Pharmaceuticals, Inc.
- Brief Summary
The goal of this study is to determine how well LB54640 works and how safe it is in patients with Hypothalamic Obesity (HO). The study will evaluate the effect of LB54640 on safety, weight reduction, hunger, and quality of life in patients 12 years of age and older with HO. Patients will take an oral daily dose of either LB54640 (low, middle, or high dose) or placebo through Week 14. Eligible patients who consent to continue in the study after Week 14 will take an oral daily dose of LB54640 through Week 56.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 28
- 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 <18 years of age
- Male and female participants agree to follow study contraception requirements and use a highly effective form of contraception throughout the study and for 90 days after the study
Key
- Weight loss >2% in the previous 3 months for patients ≥18 years of age or >2% reduction in BMI for patients <18 years of age
- History of major surgical procedure within 30 days
- HbA1c >10.9%
- Fasting glucose level >270 mg/dL
- Previous use of MC4R agonists
- Diagnosis of Prader-Willi syndrome (PWS) or Rapid-onset obesity with hypoventilation, hypothalamic, autonomic dysregulation, neuroendocrine tumor syndrome (ROHHADNET).
- Diagnosis of severe psychiatric disorders; any suicidal ideation, attempt or behavior
- History or close family history of skin cancer or melanoma
- Current clinically significant pulmonary, cardiac, or oncologic disease considered severe enough to interfere with the study and/or confound the results
Other protocol defined Inclusion/Exclusion criteria may apply.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo Placebo 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. LB54640 High dose LB54640 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. LB54640 Middle dose LB54640 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. Open-label LB54640 LB54640 Eligible participants who provide consent to continue into the open-label portion of the study will receive open-label LB54640. LB54640 Low dose LB54640 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.
- Primary Outcome Measures
Name Time Method Mean percentage change in BMI From Baseline to Week 14
- Secondary Outcome Measures
Name Time Method Frequency and severity of adverse events (AEs) From Baseline to Week 52 Mean change in BMI Baseline to Weeks 6, 10, 14 Proportion of patients who achieve ≥5% reduction in body weight Baseline to Week 14 Mean change in the weekly average of the daily most hunger score Baseline to Week 14 Mean change and mean percentage change from baseline in body weight From Baseline to Week 52 Mean change and mean percentage change from baseline in waist circumference. From Baseline to Week 52 Mean change and mean percentage change from baseline in body composition assessed by dual energy x-ray absorptiometry From Baseline to Week 52 Fat mass and lean mass will be measured through dual energy x-ray absorptiometry
Trial Locations
- Locations (10)
Children's Hospital Colorado
🇺🇸Aurora, Colorado, United States
University of Iowa Children's Hospital
🇺🇸Iowa City, Iowa, United States
Tufts Medical Center
🇺🇸Boston, Massachusetts, United States
Columbia University
🇺🇸New York, New York, United States
The University of North Carolina at Chapel Hill
🇺🇸Chapel Hill, North Carolina, United States
Lynn Health Science Institute
🇺🇸Oklahoma City, Oklahoma, United States
St. Jude Children's Research Hospital
🇺🇸Memphis, Tennessee, United States
Birmingham Women's and Children's NHS Foundation Trust
🇬🇧Birmingham, United Kingdom
University of Cambridge
🇬🇧Cambridge, United Kingdom
Great Ormond Street Hospital for Children
🇬🇧London, United Kingdom