PWS Clinical Trial: GLWL 01 As Potential Hyperphagia Treatment [VIDEO]

This blog contains excerpts from the PWS Clinical Trial GLWL 01 Overview presented Caroline Fortier, CEO of GLWL Research, Inc., at the FPWR 2017 conference. You can watch the full presentation by clicking on the embedded video below.

In case you don't have time to watch the full video, we've captured some of the key points in the notes below. 

 

Clinical Trial Details

The trial is being lead by GLWL Research Inc., a Canadian company, in collaboration with a division of Indianapolis-based Eli Lilly and Company.

Why PWS and Why This Molecule

  • It’s been reported in the literature that people with PWS have elevated acylated ghrelin levels (AG)
  • There are two types of ghrelin in the body: acylated and unacylated
  • The molecule, GLWL 01, decreases the levels of AG circulating in plasma
  • Elevated AG is associated with hyperphagia
  • It’s believed that the elevated levels could also be contributing to morbid obesity in PWS
  • GLWL 01 has been tested in a Type 2 diabetes mellitus population
  • Tested at single dose and multiple doses over an extended period of time
  • When GLWL 01 is administered at doses of 150 mg or higher twice a day, it causes a reduction in AG levels from baseline and compared to placebo
  • Researchers have seen AG level decreases of up to 170 percent in one obese patient
  • The molecule has been proven safe and well tolerated for doses up to 600 mg twice a day

Note: The clinical trial protocol has been approved, and regulatory actions are under way in both the United States and Canada.

Objectives

The primary objective of the clinical trial is to evaluate the efficacy of the GLWL 01 molecule:

  • At a dose of 450 mg twice a day
  • Compared to placebo
  • In reducing hyperphagia-related behaviors
  • After multiple dosing
  • In patients with PWS
  • As measured by the Hyperphagia Questionnaire for Clinical Trials (HQ-CT)

The secondary objectives are to:

  • Evaluate the safety and tolerability of GLWL 01 after multiple oral dosings with PWS
  • Assess efficacy compared with placebo in reducing hyperphagia-related behaviors
  • As measured by the Caregiver Global Impression of Change (CGIC) to validate the primary objective tool
  • Evaluate the pharmacokinetics after single and multiple oral dosing in patients with PWS

The exploratory objectives are to evaluate the effect of GLWL 01 on:

  • AG, UAG, AG/UAG ratio
  • Body weight, percentage fat mass, BMI, waist circumference
  • Low-density lipoproteins and total cholesterol 

Study Design

  • Randomized, double-blind
  • Conducted at multiple centers
  • Placebo-controlled
  • 34 patients with PWS
  • Duration: 18 weeks

Key Inclusion and Exclusion Criteria 

  • Males and females with a confirmed diagnosis PWS
  • Age: 16 years and older
  • BMI: 27-60 kg/m2
  • Stable body weight for previous 3 months (no more than 10 percent variation, gain or loss)
  • Caregiver must have been caring for the patient for at least six months and remain with the patient throughout the study
  • The caregiver must also spend at least 4 waking hours per day with the patient 

Exclusion Criteria

  • Can’t live in a group home for more than 50 percent of the time
  • Can’t have used any weight loss agent, including herbal medication, within the last three months
  • Can’t have Type 1 diabetes or use insulin
  • Primary caregiver must be able to understand the questionnaire and communicate with the investigator or site leader

Side Effects?

Audience question: Have patients taking the molecule experienced any side effects?

  • Yes, the side effects have been mild to moderate
  • There have been no severe adverse events
  • They’re mostly GI tract related, i.e., nausea

Learn more about PWS clinical trails.

Understanding PWS - Slide Deck

Topics: Research

Susan Hedstrom

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Susan Hedstrom is the Executive Director for the Foundation for Prader-Willi Research. Passionate about finding treatments for PWS, Susan joined FPWR in 2009 shortly after her son, Jayden, was diagnosed with Prader-Willi Syndrome. Rather than accepting PWS as it has been defined, Susan has chosen to work with a team of pro-active and tireless individuals to accelerate PWS research in order to change the natural history of PWS. Inspired by her first FPWR conference and the team of researchers that were working to find answers for the syndrome, she hosted her first One SMALL Step walk in 2010 and began the development of the One SMALL Step walk program which now raises over $1.5 million a year for PWS research.

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