Clinical trial NCT06046443: A Study to Assess Efficacy and Safety of LB54640 in Patients With Hypothalamic Obesity
Official title: 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
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: Hypothalamic Obesity
Keywords: melanocortin 4 receptor, MC4R
MC4R agonism
Lead sponsor: Rhythm Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2023-09-21
Enrollment: 28
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility 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/Exclusion criteria may apply.

Primary outcomes:
measure: Mean percentage change in BMI
timeFrame: From Baseline to Week 14
Secondary outcomes:
measure: Frequency and severity of adverse events (AEs)
timeFrame: From Baseline to Week 52
measure: Mean change in BMI
timeFrame: Baseline to Weeks 6, 10, 14
measure: Proportion of patients who achieve ≥5% reduction in body weight
timeFrame: Baseline to Week 14
measure: Mean change in the weekly average of the daily most hunger score
timeFrame: Baseline to Week 14
measure: Mean change and mean percentage change from baseline in body weight
timeFrame: From Baseline to Week 52
measure: Mean change and mean percentage change from baseline in waist circumference.
timeFrame: From Baseline 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 to Week 52
Other outcomes:
Locations:
city: Aurora
country: United States
facility: Children's Hospital Colorado
state: Colorado
city: Iowa City
country: United States
facility: University of Iowa Children's Hospital
state: Iowa
city: Boston
country: United States
facility: Tufts Medical Center
state: Massachusetts
city: New York
country: United States
facility: Columbia University
state: New York
city: Chapel Hill
country: United States
facility: The University of North Carolina at Chapel Hill
state: North Carolina
city: Oklahoma City
country: United States
facility: Lynn Health Science Institute
state: Oklahoma
city: Memphis
country: United States
facility: St. Jude Children's Research Hospital
state: Tennessee
city: Birmingham
country: United Kingdom
facility: Birmingham Women's and Children's NHS Foundation Trust
city: Cambridge
country: United Kingdom
facility: University of Cambridge
city: London
country: United Kingdom
facility: Great Ormond Street Hospital for Children
Study officials:
affiliation: Rhythm Pharmaceuticals, Inc.
name: David Meeker, MD
role: STUDY_CHAIR
References: