Setmelanotide trial in BBS and AS patients with obesity

• Research type

  Research Study

• Full title

  A Phase 3 trial of Setmelanotide (RM-493), a Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström syndrome (AS) Patients with Moderate to Severe Obesity

• IRAS ID

  258584

• Contact name

  Philip Beales

• Contact email

  p.beales@ucl.ac.uk

• Sponsor organisation

  Rhythm Pharmaceuticals, Inc.

• Eudract number

  2018-004058-11

• Duration of Study in the UK

  1 years, 9 months, 0 days

• Research summary

  This is designed for patients with Bardet-Biedl Syndrome (BBS) or Alström syndrome (AS) which have Moderate to Severe Obesity and hyperphagia (increased appetite and hunger with impaired satiety).
  Setmelanotide is a novel investigational drug, which works by activating neurons responsible for decreased appetite and weight. So far research results have demonstrated that the use of setmelanotide has been associated with significant weight and appetite reduction and that it has been well-tolerated.
  It is anticipated that approximately 30 male and female patients 6 years of age and above (including at least 20 with BBS and at least 6 with AS) will be included in the study across approximately 10 centers worldwide.

  The study will consist of 3 treatment periods. Eligible patients will enter a 14-week, randomized, double-blind, placebo-controlled treatment period (Period 1) that will be followed by a 38-week open-label treatment period (Period 2) in which all patients will receive setmelanotide. The primary analysis will be performed after Period 2. Following Period 2, patients will continue to receive open-label setmelanotide for 14 weeks (Period 3) after which they may be enrolled into a separate treatment extension study.
  The patients will daily receive sub-cutaneous injections of setmelanotide.
  Study procedures primarily include frequent obesity measurements including hunger assessment, body composition assessments (including total body weight, fat mass, and non-bone lean mass) and waist circumference. Other study procedures include vital signs, collection of blood and urine samples, physical examination and ECG.
  There is also optional sub-study to evaluate PK.

• REC name

  Wales REC 3

• REC reference

  19/WA/0080

• Date of REC Opinion

  13 Sep 2019

• REC opinion

  Further Information Unfavourable Opinion