Real-World Effects of MC4R Agonist Therapy in BBS and Severe Genetic Obesity
Recruiting · NCT07674290 · Interventional (participants receive a specific treatment) · Lead sponsor: Tom Hühne
View the official record on ClinicalTrials.gov →Interventions studied
What this trial is about
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.
Who can take part
Inclusion criteria
- clinical phenotype corresponding to Bardet-Biedl Syndrome
- genetic testing with notable finding
Exclusion criteria
- patients younger than the age approved for treatment with setmelanotide
Where it is running
1 location listed.
- University Hospital Essen, Deparment of Pediatrics II - Essen, Germany