Study of Setmelanotide in paediatric patients 2 to <6
Research type
Research Study
Full title
A Phase 3 Multi-Center, One-Year, Open-Label study of Setmelanotide in Pediatric Patients Aged 2 to <6 years of age with Rare Genetic Causes of Obesity
IRAS ID
1004402
Contact name
Amy Herbert
Contact email
Sponsor organisation
Rhythm Pharmaceuticals Limited
Eudract number
2021-004167-27
Clinicaltrials.gov Identifier
Research summary
This study is to primarily assess the effectiveness of Setmelanotide, given as a subcutaneous injection, on the weight of patients between the ages of 2 and 6. These patients present with genetic obesity as a result of variants of the POMC PCSK1 and LEPR genes, or Bardet-Biedl Syndrome which progress over time and can end up as life threatening. Setmelanotide has been approved in the US for chronic weight management in patients over the age of 6 with obesity due to POMC, PCSK1 and LEPR. The youngest child treated with Setmelanotide during previous research was 6 years old. As these conditions present from a very early age, and can result in severe complications later in life, there is an unmet need to assess the effectiveness of Setmelanotide in patients as young as 2, and hopefully to improve the long term outcomes by starting treatment as early as possible. The study will be open label and last for 1 year. The screening period will last between 1 and 8 weeks, where potential participants will be assessed for their suitability; caregivers will also be trained during this period on how to administer the injection. Once the participant is enrolled, they will have a dose of Setmelanotide every day, with the dose increasing by 0.5mg every 2 weeks. The dose amounts will be determined by the study team based on the participants weight during the enrollment visit. Study assessments will be carried out every 4 weeks throughout the study. The End of Treatment visit will occur on Day 365 and will be conducted via telephone. After this point, participants may be eligible to enroll in a long term extension study.
REC name
East Midlands - Nottingham 2 Research Ethics Committee
REC reference
22/EM/0052
Date of REC Opinion
28 Mar 2022
REC opinion
Further Information Favourable Opinion