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โ† Trials
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Real-World Effects of MC4R Agonist Therapy in BBS and Severe Genetic Obesity

RecruitingPhase 4Ages any - anyAllTom Hรผhne
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In plain words

What the trial is, and whether it could suit you

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.

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
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
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What it tests

  • Setmelanotide
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Where

1 site
University Hospital Essen, Deparment of Pediatrics II
Essen, Germany
๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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