Real-World Effects of MC4R Agonist Therapy in BBS and Severe Genetic Obesity
Recruiting
Phase 4
Interventional Study
Bardet Biedl Syndrome (BBS)
Bardet Biedl Syndrome
Bardet-Biedl Syndrome (BBS)
Alstrom Syndrome
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Sex
- Any
- Trial phase
- Phase 4
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 200 (estimated)
- Sponsor
- Tom Hühne · Other
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About This Trial
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 effectivene…
Trial Locations
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Eligibility 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
Contacts