Real-World Effects of MC4R Agonist Therapy in BBS and Severe Genetic Obesity

Recruiting now · Phase 4

Conditions studied: Bardet Biedl Syndrome (BBS), Bardet Biedl Syndrome, Bardet-Biedl Syndrome (BBS), Alstrom Syndrome

In brief

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.

Key facts

Study ID

NCT07674290

Run by

Tom Hühne

People needed

200

Starts

2023-01-01

Expected to finish

2030-12-31

Last updated by the study team

2026-06-29

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• clinical phenotype corresponding to Bardet-Biedl Syndrome
• genetic testing with notable finding

You may not qualify if…

• patients younger than the age approved for treatment with setmelanotide

Where it is running

• University Hospital Essen, Deparment of Pediatrics II — Essen, Germany (enrolling)

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.