A Study of GLWL-01 in Patients With Prader-Willi Syndrome
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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. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT03274856 |
Recruitment Status :
Completed
First Posted : September 7, 2017
Results First Posted : March 27, 2020
Last Update Posted : March 27, 2020
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Prader-Willi Syndrome | Drug: GLWL-01 Drug: Placebo | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 19 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 |
Actual Primary Completion Date : | June 12, 2019 |
Actual Study Completion Date : | June 12, 2019 |

Arm | Intervention/treatment |
---|---|
Treatment Sequence 1
GLWL-01 (450mg) twice a day/ Placebo
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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 |
- Post-treatment Total Score on the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) [ Time Frame: Up to approximately 4 weeks of double-blind treatment ]GLWL-01 compared with placebo on the post-treatment HQ-CT score. Total range of score of zero to 36, with higher score indicating a worse outcome.
- 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
- Caregiver Global Impression of Change (CGIC) [ Time Frame: Up to approximately 4 weeks of double-blind treatment ]GLWL-01 compared with placebo in the CGIC. Score ranges from 1 to 7, with larger number indicating a worse outcome.
- Area Under the Concentration Versus Time Curve From Time Zero to 12 Hours (AUC0-12) [ Time Frame: Day 14 and Day 42, pre-dose, and 0.5, 1, 2, 4, 6, and between 8 and 12 hours postdose ]Pharmacokinetics (PK) after single and multiple oral dosing
- Maximum Observed Drug Concentration (Cmax) [ Time Frame: Day 14 and Day 42, pre-dose, and 0.5, 1, 2, 4, 6, and between 8 and 12 hours postdose ]Pharmacokinetics after single and multiple oral dosing

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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
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Unable to refrain from or anticipates the use of:
- Any drugs known to be significant inhibitors of Cytochrome P450, family 3, subfamily A (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
- Any drugs known to be significant inducers of Cytochrome P450, family 3, subfamily A (CYP3A) enzymes and/or P-gp, including St. John's Wort
- 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

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 ClinicalTrials.gov identifier (NCT number): NCT03274856
United States, California | |
Rady Children's Hospital San Diego | |
San Diego, California, United States, 92123 | |
United States, Florida | |
University of Florida | |
Gainesville, Florida, United States, 32601 | |
United States, Ohio | |
University Hospitals, Cleveland Medical Center | |
Cleveland, Ohio, United States, 44106 | |
United States, Tennessee | |
Vanderbilt University | |
Nashville, Tennessee, United States, 37232 | |
Canada, Alberta | |
Alberta Diabetes Institute, University of Alberta | |
Edmonton, Alberta, Canada, T6G 2B7 | |
Canada, Quebec | |
CRCHUM | |
Montreal, Quebec, Canada, H2W 1T8 | |
Centre Hospitalier Universitaire Ste-Justine | |
Montreal, Quebec, Canada, H3T 1C5 |
Study Director: | Study Director | GLWL Research Inc. |
Documents provided by GLWL Research Inc.:
Responsible Party: | GLWL Research Inc. |
ClinicalTrials.gov Identifier: | NCT03274856 |
Other Study ID Numbers: |
GLWL-PWS |
First Posted: | September 7, 2017 Key Record Dates |
Results First Posted: | March 27, 2020 |
Last Update Posted: | March 27, 2020 |
Last Verified: | March 2020 |
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 |
Prader-Willi Syndrome Syndrome Disease Pathologic Processes Intellectual Disability Neurobehavioral Manifestations Neurologic Manifestations Nervous System Diseases |
Abnormalities, Multiple Congenital Abnormalities Chromosome Disorders Genetic Diseases, Inborn Obesity Overnutrition Nutrition Disorders |