Setmelanotide for the treatment of acquired hypothalamic obesity: a phase 2, open-label, multicentre trial
Authors' conclusion
These findings support setmelanotide as a novel effective treatment of hypothalamic obesity.
Study context
- Population
- Patients aged 6–40 years with obesity and hypothalamic injury or non-malignant tumour affecting the hypothalamus treated with surgery, chemotherapy or radiation; 5 US centres; mean age 15.0 years, mean BMI 38.0 kg/m²16 completed
- Participants
- 18 patients
- Comparison
- Setmelanotide vs historic control rate
- Setmelanotiden = 18
- Outcome
- Proportion with BMI reduction ≥5% from baseline, vs historic control rate <5%
- Follow-up
- 16 weeks
Methodological detailsopen-label, multicentre, historic control rate of <5%
- Open-label
- Multicentre
- Historic control rate of <5%
Main finding
As reported in the abstract
BMI reduction ≥5% from baseline, vs historic control rate <5%
Patients who completed the study and met the primary endpoint (BMI reduction ≥5% from baseline)
16 (89%) of 18
p<0.0001
Further findings
Mean BMI reduction, all patients
- Mean reduction in BMI across all patients
- 15% (SD 10)
Composite clinically meaningful change
- Proportion with a clinically meaningful change (composite)
- 89%, 90% CI 69-98%; p<0.0001
- BMI Z score reduction ≥0.2 points, patients <18 years
- 92%, 68-100%; p<0.0001
- Bodyweight reduction ≥5%, patients ≥18 years
- 80%, 34-99%; p<0.0001
Hunger score, patients aged ≥12 years
- Mean reduction in hunger score
- 45%
BMI change from index trial baseline in long-term extension · ≥12 months of treatment
- Mean change in BMI (12 of 14 patients continuing in extension)
- −26% (SD 12)
Safety
Frequent adverse events
- Nausea
- 61%
- Vomiting
- 33%
- Skin hyperpigmentation
- 33%
- Diarrhoea
- 22%
Original publication
Setmelanotide for the treatment of acquired hypothalamic obesity: a phase 2, open-label, multicentre trial. Lancet Diabetes Endocrinol. Published 30 April 2024. doi:10.
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