Clinical trial

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

Recruiting now · Phase 4 · 1 countries · Registry ID NCT07674290

Recruiting nowPhase 4Interventional

What this study 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.

A promising-looking record is not the same as confirmed eligibility. The study team must review the full criteria and current recruitment status.

Basic eligibility

AgeNot listed to Not listed

SexAll

Healthy volunteersNot accepted

ConditionBardet Biedl Syndrome (BBS), Bardet Biedl Syndrome, Bardet-Biedl Syndrome (BBS), Alstrom Syndrome

Full registry criteria

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

Treatments and study arms

Setmelanotide

Drug

Administration according to approved product labeling and treating physician discretion

Primary outcomes

Percent change in BMI z-scoreBaseline to 12/24/36/48/60/72 months

Relative change in BMI z-Score after initiation of MC4 receptor agonist therapy

Impact on lipid profileBaseline to 12/24/36/48/60/72 months

Changes in lipid profile measured by cholesterol blood levels

Change in Hepatic Fat AttenuationBaseline to 12/24/36/48/60/72 months

Hepatic Fat Attenuation will be measured by ultrasound Attenuation imaging across different time points

Study locations

1 locations were listed when this page was built. The first 40 are shown.

University Hospital Essen, Deparment of Pediatrics II🇩🇪 Essen, Germany

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Metin Cetiner, PD Dr. med.Contact

Email · Call +49 201 723 84722