Setmelanotide in BBS and related genetic obesity: real-world effects

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a real-world, post-approval registry evaluating Setmelanotide for patients with Bardet-Biedl syndrome and other MC4R pathway–related obesity disorders. It collects longitudinal clinical data from routine visits to describe changes in weight, BMI, hunger, metabolic parameters, quality of life, adherence, and adverse events. The study aims to understand effectiveness, safety, and patient-reported outcomes in everyday practice and to serve as a platform for future MC4R agonists. It is conducted primarily at University Hospital Essen and will include both pediatric and adult participants treated per approved indications. Data will be gathered from standard clinical assessments and patient questionnaires to reflect real-world experiences.

Why it matters

Who can join: the full rules

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's running

• University Hospital Essen, Deparment of Pediatrics II
  Essen, Germany Recruiting

Study contacts

• Principal investigator: Metin Cetiner, PD Dr. med. — Universitätsmedizin Essen
• Study coordinator: Tom Hühne, Dr. med.
• Email: tom.huehne@uk-essen.de
• Phone: +49 201 723 86211

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Observing the health management of patients with rare genetic syndromes

  This study looks at how patients with rare genetic syndromes like Prader-Willi and Klinefelter manage their health as they move from children's care to adult care to see what support they need.

  Recruiting Erasmus Medical Center · Rotterdam, South Holland · NCT04463316

• Cohort for Bardet-Biedl and Alström Syndromes

  This study is trying to learn more about Bardet-Biedl and Alström Syndromes by collecting health information from both kids and adults to see how these rare conditions affect their lives over time.

  Recruiting University Hospital, Strasbourg, France · Strasbourg · NCT04461444

• Registry for inherited retinal degenerative diseases

  This study is creating a registry for people with rare inherited eye diseases to help track their health and experiences over time, making it easier to understand these conditions and support research.

  Recruiting Foundation Fighting Blindness · Columbia, Maryland · NCT02435940

• Registry for individuals with Bardet-Biedl Syndrome

  This study is creating a registry to gather health information from people with Bardet-Biedl Syndrome to better understand the condition and help develop new treatments.

  Recruiting Marshfield Clinic Research Foundation · Marshfield, Wisconsin · NCT02329210

• Registry for patients with rare diseases

  This study is creating a central registry for people with rare diseases to help connect them with researchers and improve treatments.

  Recruiting Sanford Health · Sioux Falls, South Dakota · NCT01793168

• Resource for Hepato/Renal Fibrocystic Diseases

  This study is trying to collect detailed health and genetic information from people with hepato/renal fibrocystic diseases to improve understanding and treatment options for these conditions.

  Recruiting at 4 sites Children's Hospital of Philadelphia · Washington D.C., District of Columbia and 5 other locations · NCT01401998

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.