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

Primary Purpose

Prader-Willi Syndrome

Status

Completed

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

GLWL-01

Placebo

About this trial

This is an interventional treatment trial for Prader-Willi Syndrome

Eligibility Criteria

16 Years - 65 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Rady Children's Hospital San Diego
University of Florida
University Hospitals, Cleveland Medical Center
Vanderbilt University
Alberta Diabetes Institute, University of Alberta
CRCHUM
Centre Hospitalier Universitaire Ste-Justine

Arms of the Study

Arm 1

Arm 2

Arm Type

Other

Arm Label

Treatment Sequence 1

Treatment Sequence 2

Arm Description

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

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

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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

Evaluate the safety and tolerability of GLWL-01

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.

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

Pharmacokinetics (PK) after single and multiple oral dosing

Maximum Observed Drug Concentration (Cmax)

Pharmacokinetics after single and multiple oral dosing

Full Information

NCT ID

NCT03274856

First Posted

September 5, 2017

Last Updated

March 9, 2020

Sponsor

GLWL Research Inc.

1. Study Identification

Unique Protocol Identification Number

NCT03274856

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

March 2020

Overall Recruitment Status

Completed

Study Start Date

February 20, 2018 (Actual)

Primary Completion Date

June 12, 2019 (Actual)

Study Completion Date

June 12, 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

GLWL Research Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Product Manufactured in and Exported from the U.S.

Yes

Data Monitoring Committee

5. Study Description

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).

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Prader-Willi Syndrome

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Crossover Assignment

Masking

ParticipantCare ProviderInvestigator

Allocation

Randomized

Enrollment

19 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Treatment Sequence 1

Arm Type

Other

Arm Description

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

Arm Title

Treatment Sequence 2

Arm Type

Other

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

GLWL-01

Intervention Description

Oral administration of 3 capsules, twice a day

Intervention Type

Drug

Intervention Name(s)

Placebo

Intervention Description

Oral administration of 3 capsules, twice a day

Primary Outcome Measure Information:

Title

Post-treatment Total Score on the Hyperphagia Questionnaire for Clinical Trials (HQ-CT)

Description

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.

Time Frame

Up to approximately 4 weeks of double-blind treatment

Secondary Outcome Measure Information:

Title

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

Description

Evaluate the safety and tolerability of GLWL-01

Time Frame

Baseline up to approximately 18 weeks

Title

Caregiver Global Impression of Change (CGIC)

Description

GLWL-01 compared with placebo in the CGIC. Score ranges from 1 to 7, with larger number indicating a worse outcome.

Time Frame

Up to approximately 4 weeks of double-blind treatment

Title

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

Description

Pharmacokinetics (PK) after single and multiple oral dosing

Time Frame

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

Title

Maximum Observed Drug Concentration (Cmax)

Description

Pharmacokinetics after single and multiple oral dosing

Time Frame

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

10. Eligibility

Sex

All

Minimum Age & Unit of Time

16 Years

Maximum Age & Unit of Time

65 Years

Accepts Healthy Volunteers

Eligibility Criteria

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: 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Study Director

Organizational Affiliation

GLWL Research Inc.

Official's Role

Study Director

Facility Information:

Facility Name

Rady Children's Hospital San Diego

City

San Diego

State/Province

California

ZIP/Postal Code

92123

Country

United States

Facility Name

University of Florida

City

Gainesville

State/Province

Florida

ZIP/Postal Code

32601

Country

United States

Facility Name

University Hospitals, Cleveland Medical Center

City

Cleveland

State/Province

Ohio

ZIP/Postal Code

44106

Country

United States

Facility Name

Vanderbilt University

City

Nashville

State/Province

Tennessee

ZIP/Postal Code

37232

Country

United States

Facility Name

Alberta Diabetes Institute, University of Alberta

City

Edmonton

State/Province

Alberta

ZIP/Postal Code

T6G 2B7

Country

Canada

Facility Name

CRCHUM

City

Montreal

State/Province

Quebec

ZIP/Postal Code

H2W 1T8

Country

Canada

Facility Name

Centre Hospitalier Universitaire Ste-Justine

City

Montreal

State/Province

Quebec

ZIP/Postal Code

H3T 1C5

Country

Canada

12. IPD Sharing Statement

Plan to Share IPD

Citations:

PubMed Identifier

35213714

Citation

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.

Results Reference

derived

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

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