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

NCT03274856 | Completed Phase 2 Results FDA Drug

• 1 other identifier: GLWL-PWS
• Study Type: interventional
• Target: 19
• Locations: 2 countries
• Sites: 7

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).

Conditions

Prader-Willi Syndrome

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (4)

• Number of Participants With One or More Treatment Emergent Adverse Events (AEs) or Any Serious AEs

  Baseline up to approximately 18 weeks

• Caregiver Global Impression of Change (CGIC)

  Up to approximately 4 weeks of double-blind treatment

• Area Under the Concentration Versus Time Curve From Time Zero to 12 Hours (AUC0-12)

  Day 14 and Day 42, pre-dose, and 0.5, 1, 2, 4, 6, and between 8 and 12 hours postdose

• Maximum Observed Drug Concentration (Cmax)

  Day 14 and Day 42, pre-dose, and 0.5, 1, 2, 4, 6, and between 8 and 12 hours postdose

Study Arms (2)

Treatment Sequence 1

OTHER

GLWL-01 (450mg) twice a day/ Placebo

Drug: GLWL-01; Drug: Placebo

Treatment Sequence 2

OTHER

Placebo / GLWL-01 (450mg), twice a day

Drug: GLWL-01; Drug: Placebo

Interventions

GLWL-01 DRUG

Oral administration of 3 capsules, twice a day

Treatment Sequence 1; Treatment Sequence 2

Placebo DRUG

Oral administration of 3 capsules, twice a day

Treatment Sequence 1; Treatment Sequence 2

Eligibility Criteria

• Age: 16 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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:
• 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

Sponsors & Collaborators

• GLWL Research Inc.: lead

Study Sites (7)

Rady Children's Hospital San Diego

San Diego, California, 92123, United States

Location

University of Florida

Gainesville, Florida, 32601, United States

Location

University Hospitals, Cleveland Medical Center

Cleveland, Ohio, 44106, United States

Location

Vanderbilt University

Nashville, Tennessee, 37232, United States

Location

Alberta Diabetes Institute, University of Alberta

Edmonton, Alberta, T6G 2B7, Canada

Location

CRCHUM

Montreal, Quebec, H2W 1T8, Canada

Location

Centre Hospitalier Universitaire Ste-Justine

Montreal, Quebec, H3T 1C5, Canada

Location

Related Publications (1)

• Miller JL, Lacroix A, Bird LM, Shoemaker AH, Haqq A, Deal CL, Clark KA, Ames MH, Suico JG, de la Pena A, Fortier C. The Efficacy, Safety, and Pharmacology of a Ghrelin O-Acyltransferase Inhibitor for the Treatment of Prader-Willi Syndrome. J Clin Endocrinol Metab. 2022 May 17;107(6):e2373-e2380. doi: 10.1210/clinem/dgac105.

  PMID: 35213714 DERIVED

MeSH Terms

Conditions

Prader-Willi Syndrome

Condition Hierarchy (Ancestors)

Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Imprinting Disorders; Obesity; Overweight; Overnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Caroline Fortier
• Organization: GLWL Research Inc

caroline.fortier@glwlresearch.com

1.514.708.4417

Study Officials

• Study Director

  GLWL Research Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 5, 2017
• First Posted: September 7, 2017
• Study Start: February 20, 2018
• Primary Completion: June 12, 2019
• Study Completion: June 12, 2019
• Last Updated: March 27, 2020
• Results First Posted: March 27, 2020

Record last verified: 2020-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (4); CA (3)