Trials / NCT04966741
Setmelanotide in Pediatric Participants With Rare Genetic Diseases of Obesity
Completed Phase 3 Drug / neuroprotection
- Sponsor
- Rhythm Pharmaceuticals, Inc.
- Treatment
- Setmelanotide
- Gene
- Not stated
- Conditions
- Bardet-Biedl Syndrome, POMC Deficiency Obesity, PCSK1 Deficiency Obesity, LEPR Deficiency Obesity
- Ages
- 2 Years to 5 Years
- Enrollment
- 12 participants
- Started
- 2022-03-08
- Main results expected
- 2023-09-18
- Results posted
- Yes
- Record last updated
- 2024-11-27
What the study is testing
This is a phase 3 open-label, clinical study to evaluate the efficacy, safety and tolerability of setmelanotide over 1 year of treatment, in pediatric participants aged 2 to \<6 years with obesity due to either biallelic variants of the pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) genes or Bardet-Biedl Syndrome (BBS).
Where it runs
Australia (1)
- Sydney Children's Hospital, Randwick
Spain (1)
- Hospital Infantil Niño Jesus, Madrid
United Kingdom (1)
- Addenbrooke's Hospital, Wellcome Trust-MRC Institute of Metabolic Science, Cambridge
United States (3)
- Children's Hospital Colorado, Aurora, Colorado
- Columbia University Medical Center, Division of Pediatric Endocrinology, Diabetes and Metabolism, New York, New York
- Marshfield Clinic Research Foundation, Marshfield, Wisconsin