What the trial was testing
The trial enrolled 15 patients with genetic obesity. The study was sponsored by Rhythm Pharmaceuticals and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was a large trial designed to confirm whether the treatment works well enough for wider use. Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
80% of POMC-deficiency patients lost 10% or more body weight at one year.
The Lancet Diabetes & Endocrinology · 2020 · NCT02896192
These findings — that of POMC- and LEPR-deficiency patients lost at least 10% body weight on setmelanotide — were published in the The Lancet Diabetes & Endocrinology and represent the headline result of the study.
Researchers tracked outcomes across 15 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with genetic obesity, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
Setmelanotide (Imcivree) is FDA-approved and available now for genetic obesity caused by POMC, PCSK1, LEPR, or Bardet-Biedl syndrome variants. Genetic testing is required to confirm eligibility. Most common side effects are skin darkening and injection-site reactions. Ask a pediatric or obesity specialist about access.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open genetic obesity trials
Genetics of the Acute Response to Oral Semaglutide
The study aims to investigate the genetic basis of the response to short-term (3 months) orally administered semaglutide treatment, in terms of improving metabolic parameters, including the hormonal response to a standardized meal, and changes in body composition and liver steatosis. In the study, parameters such as fasting and 2-hour glucose during OGTT, HbA1c, body fat mass, body weight, total cholesterol, HDL and LDL, triglycerides, HOMA-IR, Matsuda Index and liver steatosis will be assessed. All the patients will undergo genome-wide genotyping. Moreover, in a subset of participants, muscle and fat biopsies will be performed, before and after the treatment, and liver, muscle and pancreas fat content will be assessed using MRI.
Periodontal Assessment of a Bariatric Care Population
Our primary aim is to investigate the prevalence and severity of Periodonotal Disease (PD) in a population of obese patients. Our secondary objectives are to: Investigate inflammatory biomarkers that have been associated with PD in the saliva of obese patients. Investigate the association of FTO gene (Obesity) polymorphisms with the prevalence of PD in this population. Investigate and describe the subgingival microbial flora in obese patients with PD from subgingival dental plaque samples as well as the salivary samples.