Clinical Trial: Setmelanotide for the Treatment of Early-Onset POMC Deficiency Obesity

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: An Open Label, 1-Year Trial, Including a Double-Blind Placebo-Controlled Withdrawal Period, of RM-493, a MC4R Agonist, in Early Onset POMC Deficiency Obesity Due to Bi-All

Brief Summary: The purpose of the study is to determine the effect of setmelanotide (RM-493) on weight and other factors in patients with pro-opiomelanocortin (POMC) deficiency obesity due to rare bi-allelic loss-of function POMC or PCSK1 genetic mutations.

Detailed Summary:
Sponsor: Rhythm Pharmaceuticals, Inc.

Current Primary Outcome: Effect on weight loss [ Time Frame: 1 year ]

Measurement of the effect of RM-493 on weight loss.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] [ Time Frame: 1 year ]
    Assessment of Adverse Events related to treatment
  • Effect on Body Fat Mass [ Time Frame: 1 year ]
    Assessment of body composition as measured by bioelectrical impedance (BIA) or Dual-energy x-ray absorptiometry (DXA).
  • Effect on Hunger [ Time Frame: 1 year ]
    Assessment of hunger using a Hunger Questionnaire.
  • Improvements in insulin resistance [ Time Frame: 1 year ]
    Assessment of fasting glucose, glycated hemoglobin (HbA1c), and oral glucose tolerance test (OGTT).
  • Effect on Waist Circumference [ Time Frame: 1 year ]
    Assessment of waist circumference.
  • Reversal of weight during the double-blind placebo controlled withdrawal phase [ Time Frame: 8 weeks ]
    Assessment of weight regain during the double blind withdrawal phase


Original Secondary Outcome:

  • Assess safety and tolerability of setmelanotide [ Time Frame: 1 year ]
    Assessment of Adverse Events related to treatment
  • Effect on Body Fat Mass [ Time Frame: 1 year ]
    Assessment of body composition as measured by bioelectrical impedance (BIA) or Dual-energy x-ray absorptiometry (DXA).
  • Effect on Hunger [ Time Frame: 1 year ]
    Assessment of hunger using a Hunger Questionnaire.
  • Improvements in insulin resistance [ Time Frame: 1 year ]
    Assessment of fasting glucose, glycated hemoglobin (HbA1c), and oral glucose tolerance test (OGTT).
  • Effect on Waist Circumference [ Time Frame: 1 year ]
    Assessment of waist circumference.
  • Reversal of weight during the double-blind placebo controlled withdrawal phase [ Time Frame: 8 weeks ]
    Assessment of weight regain during the double blind withdrawal phase


Information By: Rhythm Pharmaceuticals, Inc.

Dates:
Date Received: August 18, 2016
Date Started: February 14, 2017
Date Completion: October 2018
Last Updated: April 5, 2017
Last Verified: April 2017