Prader-Willi SyndromePhase 2NCT03274856
A Study of GLWL-01 in Patients With Prader-Willi Syndrome
A Phase 2 study testing GLWL-01 in people with Prader-Willi Syndrome, run by GLWL Research Inc. at 7 sites in 2 countries.
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). Read more about this study
- Who is it for?
- Ages 16 to 65 years
- Conditions the study is for
- Prader-Willi Syndrome
- What is being tested?
- GLWL-01
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 19 participants
- Where
- 7 sitesin 2 countries
- Study dates
- 20 Feb 2018to 12 Jun 2019
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 12Show fewer
- 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
Trial locations and contacts
7 sites in 2 countries.
7 sites in 2 countries
United States4 sites
University Hospitals, Cleveland Medical Center
Cleveland, Ohio, 44106
University of Florida
Gainesville, Florida, 32601
Vanderbilt University
Nashville, Tennessee, 37232
Rady Children's Hospital San Diego
San Diego, California, 92123
Canada3 sites
Alberta Diabetes Institute, University of Alberta
Edmonton, Alberta, T6G 2B7
CRCHUM
Montreal, Quebec, H2W 1T8
Centre Hospitalier Universitaire Ste-Justine
Montreal, Quebec, H3T 1C5
About this study
Official title: A Phase 2 Study to Evaluate Efficacy, Safety, and Pharmacokinetics of GLWL-01 in the Treatment of Patients With Prader-Willi Syndrome
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).
Read the 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
- Conditions
- Prader-Willi Syndrome
- Being tested
- GLWL-01, Placebo
- Medical subject headings
- Prader-Willi Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GLWL-01Drug
Oral administration of 3 capsules, twice a day
PlaceboDrug
Oral administration of 3 capsules, twice a day
| Study group | Role | What they receive |
|---|---|---|
| Treatment Sequence 1 | Other | GLWL-01 (450mg) twice a day/ Placebo Receives: Drug: GLWL-01; Drug: Placebo |
| Treatment Sequence 2 | Other | Placebo / GLWL-01 (450mg), twice a day Receives: Drug: GLWL-01; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
Evaluate the safety and tolerability of GLWL-01
Measured: Baseline up to approximately 18 weeks
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.
Measured: Up to approximately 4 weeks of double-blind treatment
Area Under the Concentration Versus Time Curve From Time Zero to 12 Hours (AUC0-12)
Pharmacokinetics (PK) after single and multiple oral dosing
Measured: 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)
Pharmacokinetics after single and multiple oral dosing
Measured: Day 14 and Day 42, pre-dose, and 0.5, 1, 2, 4, 6, and between 8 and 12 hours postdose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Sep 2017
- Study start20 Feb 2018
- Primary completion12 Jun 2019
- Study completion12 Jun 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Prader-Willi SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- GLWL Research Inc.
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, GLWL Research Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03274856
- Sponsor's protocol ID
- GLWL-PWS
- Status last verified
- Mar 2020
- First submitted
- 5 Sep 2017
- Last updated
- 27 Mar 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 35213714
Study at a glance
From the registry record, last updated by the sponsor on 27 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Study to Evaluate Efficacy, Safety, and Pharmacokinetics of GLWL-01 in the Treatment of Patients With Prader-Willi Syndrome
- ClinicalTrials.gov ID
- NCT03274856
- Acronym / sponsor ID
- GLWL-PWS
- Conditions
- Prader-Willi Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 19 participants (actual)
- Lead sponsor
- GLWL Research Inc.University, hospital or other
- Study start
- 20 Feb 2018 (actual)
- Primary completion
- 12 Jun 2019 (actual)
- Study completion
- 12 Jun 2019 (actual)
- First posted
- 7 Sep 2017 (actual)
- Results
- Posted 27 Mar 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Treatment Sequence 1 | Other | Drug: GLWL-01, Drug: PlaceboGLWL-01 (450mg) twice a day/ Placebo |
| Treatment Sequence 2 | Other | Drug: GLWL-01, Drug: PlaceboPlacebo / GLWL-01 (450mg), twice a day |
Who may qualify
- Ages
- 16 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (12 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GLWL-01Oral administration of 3 capsules, twice a day | Drug | Treatment Sequence 1, Treatment Sequence 2 |
| PlaceboOral administration of 3 capsules, twice a day | Drug | Treatment Sequence 1, Treatment Sequence 2 |
Primary outcome
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.
4 secondary outcomes
Number of Participants With One or More Treatment Emergent Adverse Events (AEs) or Any Serious AEs
Time frame: Baseline up to approximately 18 weeks
Caregiver Global Impression of Change (CGIC)
Time frame: Up to approximately 4 weeks of double-blind treatment
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
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
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Mar 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 35213714Miller 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director study director
GLWL Research Inc.
7 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing GLWL-01 in people with Prader-Willi Syndrome, run by GLWL Research Inc. at 7 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 27 Mar 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 27 Mar 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Post-treatment Total Score on the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) (time frame: Up to approximately 4 weeks of double-blind treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03274856, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Mar 2020.