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

Prader-Willi Syndrome Clinical Trial

Official title:

A Phase 2 Study to Evaluate Efficacy, Safety, and Pharmacokinetics of GLWL-01 in the Treatment of Patients With Prader-Willi Syndrome

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03274856
• Other study ID #: GLWL-PWS
• Status: Completed
• Phase: Phase 2
• Start date: February 20, 2018
• Est. completion date: June 12, 2019

Study information

• Verified date: March 2020
• Source: GLWL Research Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 19
• Est. completion date: June 12, 2019
• Est. primary completion date: June 12, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 16 Years to 65 Years

Eligibility

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, 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

2. 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

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

Related Conditions & MeSH terms

• Prader-Willi Syndrome
• Syndrome

Intervention

Drug:
• GLWL-01
Oral administration of 3 capsules, twice a day
• Placebo
Oral administration of 3 capsules, twice a day

Locations

Country	Name	City	State
Canada	Alberta Diabetes Institute, University of Alberta	Edmonton	Alberta
Canada	Centre Hospitalier Universitaire Ste-Justine	Montreal	Quebec
Canada	CRCHUM	Montreal	Quebec
United States	University Hospitals, Cleveland Medical Center	Cleveland	Ohio
United States	University of Florida	Gainesville	Florida
United States	Vanderbilt University	Nashville	Tennessee
United States	Rady Children's Hospital San Diego	San Diego	California

Sponsors (1)

Lead Sponsor	Collaborator
GLWL Research Inc.

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Post-treatment Total Score on the Hyperphagia Questionnaire for Clinical Trials (HQ-CT)	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.	Up to approximately 4 weeks of double-blind treatment
Secondary	Number of Participants With One or More Treatment Emergent Adverse Events (AEs) or Any Serious AEs	Evaluate the safety and tolerability of GLWL-01	Baseline up to approximately 18 weeks
Secondary	Caregiver Global Impression of Change (CGIC)	GLWL-01 compared with placebo in the CGIC. Score ranges from 1 to 7, with larger number indicating a worse outcome.	Up to approximately 4 weeks of double-blind treatment
Secondary	Area Under the Concentration Versus Time Curve From Time Zero to 12 Hours (AUC0-12)	Pharmacokinetics (PK) after single and multiple oral dosing	Day 14 and Day 42, pre-dose, and 0.5, 1, 2, 4, 6, and between 8 and 12 hours postdose
Secondary	Maximum Observed Drug Concentration (Cmax)	Pharmacokinetics after single and multiple oral dosing	Day 14 and Day 42, pre-dose, and 0.5, 1, 2, 4, 6, and between 8 and 12 hours postdose