A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study: 5 Independent Sub-studies of Setmelanotide in Patients with POMC, PCSK1, LEPR, SRC1, SH2B1, and PCSK1 N221D Gene Defects in the Melanocortin-4 Receptor Pathway
Appetite (feeling of hunger and feeling of being full after eating) and weight can be controlled by certain genes located in the brain. These genes are part of the Melanocortin-4-Receptor pathway (MC4R). Sometimes there is a genetic change (variation) in one of these genes. You have been identified as potentially having such a variation. This study will include 5 sub-studies. Each sub-study will look at one of the following gene variations in the melanocortin-4 receptor (MC4R) pathway:Sub-study 035a: pro-opiomelanocortin (POMC) or proprotein convertase subtilisin/kexin type 1 (PCSK1); Sub-study 035b: leptin receptor (LEPR); sub-study 035c: steroid receptor coactivator-1 (SRC1); Sub-study 035d: src homology 2B adaptor protein 1 (SH2B1); Sub-study 035e: a certain variation in the PCSK1 gene called PCSK1 N221D. In each sub-study, the study Sponsor wants to find out if an investigational drug called setmelanotide (RM-493) can help control body weight and hunger in people with your condition.
This study is currently enrolling.