A Trial of Setmelanotide in Acquired Hypothalamic Obesity
- Conditions
- Hypothalamic Obesity
- Interventions
- Drug: Placebo
- Registration Number
- NCT05774756
- Lead Sponsor
- Rhythm Pharmaceuticals, Inc.
- Brief Summary
The goal of this trial is to learn how well Setmelanotide works to improve weight reduction, hunger, and quality of life in patients 4 years of age and older with acquired Hypothalamic Obesity (HO). To determine how well setmelanotide works and how safe it is, patients with HO will take a daily injection of either setmelanotide or placebo and complete trial assessments for 52 weeks on a therapeutic regimen.
A separate sub-study in patients with congenital HO is detailed under NCT06760546.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 120
- Documented evidence of acquired hypothalamic obesity (HO)
- Age 4 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 4 to <18 years of age
- Agree to use a highly effective form of contraception throughout the study and for 90 days after the study
Key
- Diagnosis of Prader-Willi syndrome (PWS) or Rapid-onset obesity with hypoventilation, hypothalamic, autonomic dysregulation, neuroendocrine tumor syndrome (ROHHADNET)
- Weight loss >2% in the previous 3 months for patients aged ≥18 years or >2% reduction in BMI for patients aged 4 to <18 years
- Bariatric surgery or procedure within last 2 years
- Diagnosis of severe psychiatric disorders; any suicidal ideation, attempt or behavior
- Current, clinically significant pulmonary, cardiac, metabolic, or oncologic disease
- Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions (excluding non-invasive basal or squamous cell lesion)
- History or close family history of skin cancer or melanoma
- Participation in any clinical trial with an investigational drug/device within 3 months prior to the first trial dose
- Previously enrolled in a clinical trial involving setmelanotide or any previous exposure to setmelanotide
- Inability to comply with once daily (QD) injection regimen
- If female, pregnant and/or breastfeeding
- Patients with obesity attributable to other genetic or syndromic conditions (eg, PPL [POMC, PCSK1, LEPR, collectively], BBS) prior to the hypothalamic injury.
- If receiving hormone replacement therapy, dose has remained stable for at least 2 months before Screening
Other protocol defined Inclusion/Exclusion criteria may apply.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo Placebo Randomized 2:1 (Setmelanotide: Placebo) Setemelanotide Setmelanotide Randomized 2:1 (Setmelanotide: Placebo)
- Primary Outcome Measures
Name Time Method Mean % change in BMI From Baseline after 52 weeks on a therapeutic regimen
- Secondary Outcome Measures
Name Time Method Proportion of patients with ≥5% reduction in BMI in adult patients (≥18 years of age) or a BMI Z-score reduction of ≥0.2 points in pediatric patients (<18 years of age) From Baseline after 52 weeks on a therapeutic regimen Mean change in the weekly average of the daily most hunger score in patients ≥12 years old From Baseline after 52 weeks on a therapeutic regimen Proportion of patients with a ≥2 point reduction in the weekly average of the daily most hunger score From Baseline after 52 weeks on a therapeutic regimen Mean change in Symptoms of Hyperphagia total score From Baseline after 52 weeks on a therapeutic regimen Proportion of patients with a ≥10% reduction in BMI From Baseline after 52 weeks on a therapeutic regimen Mean percent change in weight in patients ≥18 years From Baseline after 52 weeks on a therapeutic regimen Mean BMI-z score and BMI percentile reduction in patients <18 (using combined height and weight to report BMI in kg/m2) From Baseline after 52 weeks on a therapeutic regimen Proportion of patients aged ≥4 to <18 years with ≥0.2-point reduction of BMI Z-score From Baseline after 52 weeks on a therapeutic regimen Proportion of patients with BMI <30 kg/m2 (patients aged ≥18 years) or <95th percentile (patients aged <18 years) From Baseline after 52 weeks on a therapeutic regimen Mean change in physical functioning score and total score for the Impact of Weight on Quality of Life-Lite (IWQOL) From Baseline after 52 weeks on a therapeutic regimen Safety and tolerability of setmelanotide compared to placebo assessed by frequency and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs) From Baseline after 52 weeks on a therapeutic regimen
Trial Locations
- Locations (28)
Birmingham Women and Children's Hospital NHS Trust
🇬🇧Birmingham, United Kingdom
Ohio State Wexner Medical Center
🇺🇸Columbus, Ohio, United States
Children's Hospital Colorado
🇺🇸Aurora, Colorado, United States
University of Florida
🇺🇸Gainesville, Florida, United States
Ann and Robert H. Lurie Children's Hospital
🇺🇸Chicago, Illinois, United States
University of Iowa Stead Family Department of Pediatrics
🇺🇸Iowa City, Iowa, United States
Vanderbilt University School of Medicine
🇺🇸Nashville, Tennessee, United States
Seattle Children's Hospital, Research and Foundation - Center for Integrative Brain Research
🇺🇸Seattle, Washington, United States
Hospital for Sick Children
🇨🇦Toronto, Ontario, Canada
Universitaetsklinikum Hamburg-Eppendorf (UKE) - Ambulanzzentrum des UKE GmbH
🇩🇪Hamburg, Germany
Medicover Neuroendokrinologie
🇩🇪München, Germany
Universitaetsklinikum Ulm - Klinik fuer Kinder- und Jugendmedizin
🇩🇪Ulm, Germany
Nagoya City University Hospital
🇯🇵Nagoya, Aichi, Japan
Nagano Children's Hospital
🇯🇵Azumino City, Nagano, Japan
Toranomon Hospital
🇯🇵Minato City, Tokyo, Japan
Prinses Maxima Center for Pediatric Oncology
🇳🇱Utrecht, Netherlands
Icahn School of Medicine at Mount Sinai
🇺🇸New York, New York, United States
Columbia University Irving Medical Center
🇺🇸New York, New York, United States
Lynn Health Science Institute
🇺🇸Oklahoma City, Oklahoma, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Hull University Teaching Hospital
🇬🇧Hull, United Kingdom
UCL Great Ormond Street Institute of Child Health
🇬🇧London, United Kingdom
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
Brigham and Women's Hospital
🇺🇸Boston, Massachusetts, United States
Children's Minnesota
🇺🇸Saint Paul, Minnesota, United States
University Children's Hospital, Klinikum Oldenburg
🇩🇪Oldenburg, Germany
UAN Pediatric Endocrinology
🇺🇸Birmingham, Alabama, United States
Rady Children's Hospital
🇺🇸San Diego, California, United States