A Trial of Setmelanotide in Acquired Hypothalamic Obesity
Study Details
Study Description
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 up to 60 weeks.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 3 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Setemelanotide Randomized 2:1 (Setmelanotide: Placebo) |
Drug: Setmelanotide
Solution for daily subcutaneous injection
Other Names:
|
| Placebo Comparator: Placebo Randomized 2:1 (Setmelanotide: Placebo) |
Drug: Placebo
Placebo matched to setmelanotide for daily subcutaneous injection
|
Outcome Measures
Primary Outcome Measures
- Mean % change in BMI [From Baseline to Week 60]
Secondary Outcome Measures
- 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 to Week 60]
- Mean change in the weekly average of the daily most hunger score in patients ≥12 years old [From Baseline to Week 60]
- Proportion of patients with a ≥2 point reduction in the weekly average of the daily most hunger score [From Baseline to Week 60]
- Mean change in Symptoms of Hyperphagia total score [From Baseline to Week 60]
- Proportion of patients with a ≥10% reduction in BMI [From Baseline to Week 60]
- Mean percent change in weight in patients ≥18 years [From Baseline to Week 60]
- Mean BMI-z score and BMI percentile reduction in patients <18 (using combined height and weight to report BMI in kg/m2) [From Baseline to Week 60]
- Proportion of patients aged ≥4 to <18 years with ≥0.2-point reduction of BMI Z-score [From Baseline to Week 60]
- Proportion of patients with BMI <30 kg/m2 (patients aged ≥18 years) or <95th percentile (patients aged <18 years) [From Baseline to Week 60]
- Mean change in physical functioning score and total score for the Impact of Weight on Quality of Life-Lite (IWQOL) [From Baseline to Week 60]
- Safety and tolerability of setmelanotide compared to placebo assessed by frequency and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs) [From Baseline to Week 60]
Eligibility Criteria
Criteria
Key Inclusion Criteria:
-
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 Exclusion Criteria:
-
Diagnosis of Prader-Willi syndrome (PWS) or Rapid-onset obesity with hypoventilation, hypothalamic, autonomic dysregulation, neuroendocrine tumor syndrome (ROHHADNET)
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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
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Bariatric surgery or procedure within last 2 years
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Diagnosis of severe psychiatric disorders; any suicidal ideation, attempt or behavior
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Current, clinically significant pulmonary, cardiac, metabolic, or oncologic disease
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Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions (excluding non-invasive basal or squamous cell lesion)
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History or close family history of skin cancer or melanoma
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Participation in any clinical trial with an investigational drug/device within 3 months prior to the first trial dose
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Previously enrolled in a clinical trial involving setmelanotide or any previous exposure to setmelanotide
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Inability to comply with once daily (QD) injection regimen
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If female, pregnant and/or breastfeeding
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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.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | University of Florida | Gainesville | Florida | United States | 32610-0296 |
Sponsors and Collaborators
- Rhythm Pharmaceuticals, Inc.
Investigators
- Study Chair: David Meeker, MD, Rhythm Pharmaceuticals, Inc.
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- RM-493-040