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 ]
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