A phase-2 randomized placebo-controlled pilot study in obese subjects with Prader-Willi syndrome

HS 2015-2108
Prader-Willi syndrome, Pediatric
Virginia Kimonis, MD

A phase 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 Syndrome (PWS) on safety, weight reduction and food-related behaviors.

Subjects who:

  • Have a confirmed diagnosis of PWS
  • Are age 16-65 years
  • Are of all race/ethnic backgrounds
  • Are male or female
  • Have a body-mass index greater than 27 kg/m2
  • Are receiving or not receiving growth hormone
  • Are generally healthy based on initial screening exam results

This study includes five (5) to eight (8) visits and will last a period of 70 to 163 days. There will be one (1) visit every two (2) weeks for the duration of the study. Each visit will be about an hour. The first two (2) visits will involve a screening to determine health status. The subsequent three (3) to six (6) visits will include the drug treatment, a physical exam, a blood draw, a bone scan and questionnaires.

There may be therapeutic effects for Prader-Willi syndrome by decreasing symptoms. There are potential side effects associated with this drug that will be discussed in detail with you before beginning the study.

$50 per completed visit for the patient and $50 per visit for one parent or guardian, plus travel expenses.

Drug therapy
Claudia Shambaugh, study coordinator