A Study of GLWL-01 in Patients With Prader-Willi Syndrome

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03274856
Recruitment Status : Recruiting
First Posted : September 7, 2017
Last Update Posted : January 21, 2019
Information provided by (Responsible Party):
GLWL Research Inc.

Brief Summary:
The aim of this study is to evaluate efficacy, safety, and pharmacokinetics of GLWL-01 in the treatment of patients with Prader-Willi Syndrome (PWS).

Condition or disease Intervention/treatment Phase
Prader-Willi Syndrome Drug: GLWL-01 Drug: Placebo Phase 2

Detailed Description:
Participants will be assigned to one of two treatment sequences (GLWL-01/Placebo or Placebo/GLWL-01), with each sequence consisting of two treatment periods separated by a washout period

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 34 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: A Phase 2 Study to Evaluate Efficacy, Safety, and Pharmacokinetics of GLWL-01 in the Treatment of Patients With Prader-Willi Syndrome
Actual Study Start Date : February 20, 2018
Estimated Primary Completion Date : July 2019
Estimated Study Completion Date : July 2019

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Treatment Sequence 1
GLWL-01 (450mg) twice a day/ Placebo
Drug: GLWL-01
Oral administration of 3 capsules, twice a day

Drug: Placebo
Oral administration of 3 capsules, twice a day

Treatment Sequence 2
Placebo / GLWL-01 (450mg), twice a day
Drug: GLWL-01
Oral administration of 3 capsules, twice a day

Drug: Placebo
Oral administration of 3 capsules, twice a day

Primary Outcome Measures :
  1. Post-treatment total score on the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) [ Time Frame: Up to approximately 6 weeks ]
    GLWL-01 compared with placebo on the post-treatment HQ-CT score

Secondary Outcome Measures :
  1. Number of Participants With One or More Treatment Emergent Adverse Events (AEs) or Any Serious AEs [ Time Frame: Baseline up to approximately 18 weeks ]
    Evaluate the safety and tolerability of GLWL-01

  2. Caregiver Global Impression of Change (CGIC) [ Time Frame: Baseline up to approximately 6 weeks ]
    GLWL-01 compared with placebo in the CGIC

  3. Area under the concentration versus time curve from time zero to 12 hours (AUC0-12) [ Time Frame: Baseline up to approximately 6 weeks ]
    Pharmacokinetics after single and multiple oral dosing

  4. Maximum observed drug concentration (Cmax) [ Time Frame: Baseline up to approximately 6 weeks ]
    Pharmacokinetics after single and multiple oral dosing

Information from the National Library of Medicine

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Ages Eligible for Study:   16 Years to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Confirmed diagnosis of PWS based on genetic confirmation using DNA method
  • Body mass index (BMI) of 27 to 60 kg/m2
  • No evidence of weight excursion beyond 10% of baseline weight
  • Patients must provide assent and have a reliable caregiver (must have been caring for the patient for at least 6 months) who provides a separate written informed consent to participate. The caregiver is expected to be the primary caregiver throughout the study and must be in frequent contact with the patient (defined as at least 4 awake hours per day). The caregiver must be able to communicate with site personnel and in the investigator's opinion must have adequate literacy to complete questionnaires. If a caregiver cannot continue, 1 caregiver replacement is allowed
  • Are on a stable diet and exercise regimen for >2 months prior

Exclusion Criteria:

  • Current enrollment in or discontinuation within the last 30 days from a clinical trial involving any investigational drug or device
  • Are currently living in a group home for more than 50% of the time
  • A history or presence of other medical illness that indicates a medical problem that would preclude study participation
  • Have an estimated glomerular filtration rate <60 mL/minute/1.73 m2. Have macroalbuminuria (defined as spot urine albumin to creatinine ratio of >300 μg/mg) or hematuria
  • Are hypertensive (defined as sitting systolic blood pressure (BP) greater than or equal to (≥)140 millimeters of mercury (mmHg) and diastolic BP ≥90 mmHg)
  • Patients on weight loss medications within 30 days of dosing, or with a history of bariatric surgery
  • Unable to refrain from or anticipates the use of:

    1. Any drugs known to be significant inhibitors of cytochrome P450 (CYP)3A enzymes and/or P-glycoprotein (P-gp) including regular consumption of grapefruit or grapefruit juice for 14 days prior to the first dose. Acetaminophen (up to 2 grams per 24-hour period) may be permitted
    2. Any drugs known to be significant inducers of CYP3A enzymes and/or P-gp, including St. John's Wort
    3. Any medications that prolong the QT/QTc interval, unless the participant has been stable on the medication for at least 3 months and has a corrected QT interval (QTc) <450 msec
  • Currently taking simvastatin >10 mg per day, atorvastatin >20 mg per day, or lovastatin >20 mg per day, or have a history of statin-induced myopathy/rhabdomyolysis
  • Unsuitable for inclusion in the study in the opinion of the investigator

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03274856

Contact: Study Director

United States, California
Rady Children's Hospital San Diego Recruiting
San Diego, California, United States, 92123
Contact: Rachel Winograd    858-966-8453   
Principal Investigator: Lynne Bird         
United States, Florida
University of Florida Active, not recruiting
Gainesville, Florida, United States, 32601
United States, Ohio
University Hospitals, Cleveland Medical Center Recruiting
Cleveland, Ohio, United States, 44106
Contact: Audrey Lynn    216-844-7124   
Principal Investigator: Naveen Uli         
United States, Tennessee
Vanderbilt University Recruiting
Nashville, Tennessee, United States, 37232
Contact: Margo Black    615-343-5846   
Principal Investigator: Ashley Shoemaker         
Canada, Alberta
Alberta Diabetes Institute, University of Alberta Recruiting
Edmonton, Alberta, Canada, T6G 2B7
Contact: Kristie Dehaan    780-492-1098   
Principal Investigator: Andrea Haqq         
Canada, Quebec
CRCHUM Recruiting
Montreal, Quebec, Canada, H2W 1T8
Contact: Suzanne Dallaire    514-890-8000 ext 12914   
Principal Investigator: Andre Lacroix         
Centre Hospitalier Universitaire Ste-Justine Recruiting
Montreal, Quebec, Canada, H3T 1C5
Contact: Christine Massicotte    514-345-4931 ext 3209   
Principal Investigator: Cheri Deal         
Sponsors and Collaborators
GLWL Research Inc.
Study Director: Study Director GLWL Research Inc.

Responsible Party: GLWL Research Inc. Identifier: NCT03274856     History of Changes
Other Study ID Numbers: GLWL-PWS
First Posted: September 7, 2017    Key Record Dates
Last Update Posted: January 21, 2019
Last Verified: January 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes

Additional relevant MeSH terms:
Prader-Willi Syndrome
Pathologic Processes
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Nutrition Disorders