Hypothalamic Obesity
Conditions
Keywords
melanocortin 4 receptor, MC4R, MC4R agonism
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.
Interventions
DRUGLB54640
Oral daily administration
DRUGPlacebo
Placebo matched to LB54640
Sponsors
Rhythm Pharmaceuticals, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Key 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 \<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
Exclusion criteria
* 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/
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Mean percentage change in BMI | From Baseline to Week 14 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 | — |
| 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 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 |
Countries
United Kingdom, United States
Contacts
STUDY_CHAIRDavid Meeker, MD
Rhythm Pharmaceuticals, Inc.
Outcome results
None listed