Setmelanotide in patients with specific gene defects

• Research type

  Research Study

• Full title

  A 2-Stage (Open-Label Run-in followed by Randomized Withdrawal),Double-Blind, Placebo-Controlled, Phase 2 study of Setmelanotide in Patients with Specific Gene Defects in the Melanocortin-4 Receptor Pathway

• IRAS ID

  302661

• Contact name

  Sadaf Farooqi

• Contact email

  isf20@cam.ac.uk

• Sponsor organisation

  Rhythm Pharmaceuticals Inc.

• Eudract number

  2021-002855-12

• Clinicaltrials.gov Identifier

  NCT04963231

• Duration of Study in the UK

  3 years, 0 months, 1 days

• Research summary

  This is a Phase 2 study of Setmelanotide in Patients with Specific Gene Defects in the Melanocortin-4 Receptor Pathway.

  Severe obesity is strongly influenced by genetic factors. Many of the genes involved in causing obesity affect the function of the melanocortin-4 receptor (MC4R) pathway which is the principal regulator of body weight.

  The main purpose of this study is to look at whether Setmelanotide (RM-493), a MC4R agonist (referred to as study drug), can lead to weight loss in people with obesity due to specific gene defects in the MC4R pathway. The primary objective is to evaluate the proportion of obese patients with genetic defects in the MC4R pathway who achieve a clinically meaningful reduction in body weight in response to setmelanotide after an initial response to open-label treatment.

  This study will also look at:
  
  • The safety of the study drug
  • If the study drug works to control hunger
  • The effect of study drug on the quality of life of the participant’s parent/ caregiver

  Specifically, regarding children less than 12 years old, the United States (US) FDA has recently approved setmelanotide for chronic weight management in adult and pediatric patients aged ≥6 with obesity due to three genetic conditions that cause obesity (POMC, PCSK1, and LEPR deficiency). The product is now the first-ever FDA-approved therapy for these genetic diseases which cause obesity.

• REC name

  Wales REC 5

• REC reference

  21/WA/0294

• Date of REC Opinion

  1 Nov 2021

• REC opinion

  Further Information Favourable Opinion