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Real-World Effects of MC4R Agonist Therapy in BBS and Severe Genetic Obesity
Sponsor: Tom Hühne
Summary
Bardet-Biedl syndrome (BBS) and other rare disorders associated with impairment of the melanocortin-4 receptor (MC4R) pathway are characterized by severe early-onset obesity, hyperphagia, and substantial morbidity. Setmelanotide, an MC4R agonist, is approved in Europe for selected genetic obesity disorders and reimbursed in Germany for eligible patients. This study aims to evaluate the effectiveness, safety, treatment persistence, metabolic outcomes, and patient-reported outcomes of Setmelanotide under real-world conditions. The registry is designed to allow future inclusion of additional MC4R agonists as they become approved and clinically available. The study will primarily be conducted at University Hospital Essen and will collect longitudinal routine clinical data from pediatric and adult patients receiving MC4R agonist therapy according to approved indications.
Official title: Real-World Effectiveness, Safety and Patient-reported Outcomes of Setmelanotide in Patients With Bardet-Biedl Syndrome: A Prospective Mono Centric Observational Interventional Study
Key Details
Gender
All
Age Range
Any - Any
Study Type
INTERVENTIONAL
Enrollment
200
Start Date
2023-01-01
Completion Date
2030-12-31
Last Updated
2026-06-29
Healthy Volunteers
No
Conditions
Interventions
Setmelanotide
Administration according to approved product labeling and treating physician discretion
Locations (1)
University Hospital Essen, Deparment of Pediatrics II
Essen, Germany