Actively Recruiting
A Phase 3, Double Blind, Randomized, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Setmelanotide in Patients With Acquired Hypothalamic Obesity
Led by Rhythm Pharmaceuticals, Inc. · Updated on 2026-02-12
39
Participants Needed
11
Research Sites
52 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying how well setmelanotide works to reduce weight, hunger, and improve quality of life in patients aged 4 years and older with congenital Hypothalamic Obesity (cHO). This phase 3 trial evaluates the safety and effectiveness of setmelanotide compared to placebo in this population. Participants have specific diagnoses related to hypothalamic injury and hormone deficiencies, with elevated body mass index (BMI) or BMI percentiles as criteria. Participants will receive a daily subcutaneous injection of either setmelanotide or a placebo in a randomized 2:1 ratio. The treatment period lasts for 26 weeks on a therapeutic regimen. The study is double-blind and placebo-controlled to objectively assess the effects of setmelanotide on weight and hunger measures. During the trial, participants will complete assessments measuring changes in BMI, hunger scores, weight, and quality of life related to weight impact. Safety will be monitored by tracking adverse events throughout the 26-week period. Additional evaluations include changes in BMI z-scores for pediatric patients and physical functioning scores. The trial is sponsored by Rhythm Pharmaceuticals, Inc. and will follow participants from baseline through the 26-week treatment duration.
CONDITIONS
Brief Title
A Trial of Setmelanotide in Patients With Congenital Hypothalamic Obesity (Sub-study of NCT05774756)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of multiple pituitary hormone deficiency, septo-optic dysplasia, optic nerve hypoplasia, childhood-onset combined pituitary hormone deficiency, or pituitary stalk interruption syndrome with at least one pituitary deficiency
- Body mass index (BMI) of 30 kg/m2 or higher for patients 18 years and older
- BMI at or above the 95th percentile for age and sex for patients aged 4 to under 18 years
- Age 4 years and older
- Weight gain associated with hypothalamic injury and meeting BMI criteria above
- Agree to use a highly effective form of contraception during the study and for 90 days after the study
You will not qualify if you...
- Diagnosis of Prader-Willi syndrome or ROHHADNET syndrome
- Weight loss greater than 2% in the past 3 months for adults or BMI reduction greater than 2% for children
- Bariatric surgery or weight loss procedure within the last 2 years
- Diagnosis of severe psychiatric disorders or any suicidal thoughts or behavior
- Current significant pulmonary, cardiac, metabolic, or cancer-related disease
- Significant skin findings related to melanoma or pre-melanoma lesions (excluding certain non-invasive types)
- Personal or close family history of skin cancer or melanoma
- Participation in any clinical trial with investigational drug/device within 3 months before first dose
- Previous exposure to setmelanotide or participation in related clinical trials
- Inability to follow once daily injection regimen
- Pregnant or breastfeeding females
- Receiving hormone replacement therapy with unstable dose less than 2 months prior to screening
- Other protocol-defined inclusion or exclusion criteria may apply
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 26 weeks
Participants receive daily subcutaneous injections of either setmelanotide or placebo as part of the study treatment.
Regular visits throughout the 26 weeks for treatment administration and monitoring
Trial Site Locations
Total: 11 locations
1
University of Alabama
Birmingham, Alabama, United States, 35233
Actively Recruiting
2
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
3
Lurie Children's Hospital
Chicago, Illinois, United States, 60611
Actively Recruiting
4
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
5
Children's Minnesota
Saint Paul, Minnesota, United States, 55102
Actively Recruiting
6
Columbia University Irving Medical Center
New York, New York, United States, 10032
Actively Recruiting
7
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
8
Seattle Children's Research Institute
Seattle, Washington, United States, 98105
Actively Recruiting
9
UCL Great Ormond Street Institute of Child Health
London, Greater London, United Kingdom, WC1N 1EH
Actively Recruiting
10
Birmingham Women and Children's Hospital NHS Trust
Birmingham, West Midlands, United Kingdom, B46NH
Actively Recruiting
11
Hull Royal Infirmary
Hull, United Kingdom, HU32RW
Actively Recruiting
Research Team
R
Rhythm Clinical Trials
P
Physician Inquiry Clinical Trials
How is the study designed?
Study Type
INTERVENTIONAL
Masking
TRIPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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