Setmelanotide in patients aged 2–5 years with rare MC4R pathway-associated obesity (VENTURE): a 1 year, open-label, multicenter, phase 3 trial
Authors' conclusion
To our knowledge this is the first trial of setmelanotide in patients younger than 6 years old. These results support the benefit of the drug as an early intervention to manage obesity in this population.
Study context
- Population
- Patients aged 2–5 years with hyperphagia and obesity due to biallelic proopiomelanocortin (POMC) (incl. PCSK1) or leptin receptor (LEPR) variants or genetically confirmed Bardet-Biedl syndrome (BBS); mean age 3.6 years (SD 0.9)11 completed
- Participants
- 12 patients
- Outcome
- Co-primary: % of patients reaching ≥0.2-point decrease in BMI Z score, and mean % change in BMI
- Follow-up
- Week 52
Methodological detailsmulticentre, phase 3, 6 sites in USA, UK, Spain and Australia
- Multicentre
- Phase 3
- 6 sites in USA, UK, Spain and Australia
Main finding
As reported in the abstract
≥0.2-point reduction in BMI Z score (WHO methodology), overall
Ten (83%) of 12; 95% CI 58.7-99.8
Mean % change in BMI from baseline, overall safety population
−18%
SD 13
Secondary outcomes
Mean % change in BMI by subgroup · week 52
- POMC or LEPR deficiency
- −26% (SD 11)
- BBS
- −10% (9)
BMI Z score and % of BMI 95th percentile · week 52
- Mean reduction in BMI Z score
- 3.4 [2.5]
- Mean reduction in % of BMI 95th percentile
- 32.5 [22.9]
Hunger (caregiver-reported)
- Caregivers reporting patients less hungry than at baseline
- 91%
Safety
- Serious adverse events, adverse events leading to discontinuation, or death
- 0
- All adverse events mild or moderate; most common skin hyperpigmentation, vomiting, nasopharyngitis, upper respiratory tract infection, injection site reactions
Original publication
Setmelanotide in patients aged 2–5 years with rare MC4R pathway-associated obesity (VENTURE): a 1 year, open-label, multicenter, phase 3 trial. Lancet Diabetes Endocrinol. Published 13 November 2024. doi:10.
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