Clinical Trial: Ph 2 Trial to Evaluate Safety & Efficacy of RM-493 in Obese Patients With Prader-Willi Syndrome

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Ph 2, Randomized, Double-Blind, Placebo-controlled Pilot Study to Assess the Effects of RM-493, a Melanocortin 4 Receptor (MC4R) Agonist, in Obese Subjects With Prader-Willi Sy

Brief Summary:

The purpose of this study is to evaluate the effects of a once daily subcutaneous (SC) injectable formulation of RM-493 in obese subjects with Prader-Willi syndrome on tolerability, weight loss and hyperphagia-related behavior. The study drug (RM-493 and placebo) will be administered in a blinded fashion.

Funding Source - FDA OOPD

Detailed Summary:
Sponsor: Rhythm Pharmaceuticals, Inc.

Current Primary Outcome:

• Assess safety and tolerability of RM-493 (adverse events and clinical laboratory evaluations) [ Time Frame: Baseline to Day 70 or until any adverse events are resolved ]
  Assessment of adverse events and clinical laboratory evaluations.
• Effect on weight loss [ Time Frame: Baseline to Day 56 ]
  Measurement of the effect of RM-493 on weight loss.
• Effect on hyperphagia-related behavior (using the PWS Hyperphagia Questionnaire) [ Time Frame: Baseline to Day 56 ]
  Measurement of the effect of RM-493 on hyperphagia-related behavior using the PWS Hyperphagia Questionnaire.

Original Primary Outcome: Same as current

Current Secondary Outcome:

• Effect on quality of life. [ Time Frame: Baseline to Day 56 ]
  Measurement of the effect of RM-493 on quality of life as measured by thee Food Related Problem Questionnaire and/or the Hospital Anxiety and Depression Questionnaire
• Effect on food-related behavior. (daily journal and/or Rate of Eating Questionnaire) [ Time Frame: Baseline to Day 56 ]
  Measurement of the effect of RM-493 on food-related behavior. A daily journal will be used to quantify aberrant behavior and/or a Rate of Eating Questionnaire will be used
• Effect on psychiatric status. (C-SSRS and PHQ-9 Questionnaires) [ Time Frame: Baseline to Day 70 ]
  Measurement of the effect of RM-493 on psychiatric status as assessed using the C-SSRS and PHQ-9 Questionnaires.
• Effect of on Pharmacokinetics (PK) (Frequent PK (trough) sampling) [ Time Frame: Baseline to Day 70 ]
  Frequent PK (trough) sampling throughout each stage of the study, including a 12-hour PK profile.
• Effect of dose adjustment of RM-493 [ Time Frame: Baseline to Day 56 ]
  Measurement of the effect of dose adjustment of RM-493 as assessed by measuring weight fluctuations from Baseline to Day 56.

Original Secondary Outcome:

• Effect on quality of life. [ Time Frame: Baseline to Day 56 ]
  Measurement of the effect of RM-493 on quality of life as measured by thee Food Related Problem Questionnaire and/or the Hospital Anxiety and Depression Questionnaire
• Effect on food-related behavior. (daily journal and/or Rate of Eating Questionnaire) [ Time Frame: Baseline to Day 56 ]
  Measurement of the effect of RM-493 on food-related behavior. A daily journal will be used to quantify aberrant behavior and/or a Rate of Eating Questionnaire will be used
• Effect on psychiatric status. (C-SSRS and PHQ-9 Questionnaires) [ Time Frame: Baseline to Day 70 ]
  Measurement of the effect of RM-493 on psychiatric status as assessed using the C-SSRS and PHQ-9 Questionnaires.
• Effect of on Pharmacokinetics (PK) (Frequent PK (trough) sampling) [ Time Frame: Baseline to Day 42 ]
  Frequent PK (trough) sampling throughout each stage of the study, including a 12-hour PK profile.
• Effect of dose adjustment of RM-493 [ Time Frame: Baseline to Day 56 ]
  Measurement of the effect of dose adjustment of RM-493 as assessed by measuring weight fluctuations from Baseline to Day 56.

Information By: Rhythm Pharmaceuticals, Inc.

Dates:
Date Received: November 25, 2014
Date Started: February 2015
Date Completion:
Last Updated: April 19, 2017
Last Verified: April 2017