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

NCT ID: NCT03274856

Last Updated: 2020-03-27

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

19 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-02-20

Study Completion Date

2019-06-12

Brief Summary

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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).

Detailed Description

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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

Conditions

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Prader-Willi Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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Treatment Sequence 1

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

Group Type OTHER

GLWL-01

Intervention Type DRUG

Oral administration of 3 capsules, twice a day

Placebo

Intervention Type DRUG

Oral administration of 3 capsules, twice a day

Treatment Sequence 2

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

Group Type OTHER

GLWL-01

Intervention Type DRUG

Oral administration of 3 capsules, twice a day

Placebo

Intervention Type DRUG

Oral administration of 3 capsules, twice a day

Interventions

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GLWL-01

Oral administration of 3 capsules, twice a day

Intervention Type DRUG

Placebo

Oral administration of 3 capsules, twice a day

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

16 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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GLWL Research Inc.

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Study Director

Role: STUDY_DIRECTOR

GLWL Research Inc.

Locations

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Rady Children's Hospital San Diego

San Diego, California, United States

Site Status

University of Florida

Gainesville, Florida, United States

Site Status

University Hospitals, Cleveland Medical Center

Cleveland, Ohio, United States

Site Status

Vanderbilt University

Nashville, Tennessee, United States

Site Status

Alberta Diabetes Institute, University of Alberta

Edmonton, Alberta, Canada

Site Status

CRCHUM

Montreal, Quebec, Canada

Site Status

Centre Hospitalier Universitaire Ste-Justine

Montreal, Quebec, Canada

Site Status

Countries

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United States Canada

References

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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.

Reference Type DERIVED
PMID: 35213714 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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GLWL-PWS

Identifier Type: -

Identifier Source: org_study_id

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