Trial Outcomes & Findings for A Study of GLWL-01 in Patients With Prader-Willi Syndrome (NCT NCT03274856)

Last Updated: 2020-03-27

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

19 participants

Primary outcome timeframe

Up to approximately 4 weeks of double-blind treatment

Results posted on

2020-03-27

Participant Flow

Five additional participants screened but not randomized

Participant milestones

Participant milestones
Measure	Treatment Sequence 1 GLWL-01 (450mg) twice a day/ Placebo. Participants randomized to 1 of 2 treatment sequences; GLWL-01/placebo or placebo/GLWL-01 (Treatment Period 1 double-blind treatment phase/Treatment Period 2 double-blind treatment phase). During single-blind placebo lead-in phases, participants receive 3 capsules of 150-mg placebo twice daily (BID) for 14 days. During double-blind treatment phases, participants receive 3 capsules of 150-mg GLWL-01 (450 mg total dose) BID or identical placebo BID for 28 days	Treatment Sequence 2 Placebo / GLWL-01 (450mg), twice a day. Participants randomized to 1 of 2 treatment sequences; GLWL-01/placebo or placebo/GLWL-01 (Treatment Period 1 double-blind treatment phase/Treatment Period 2 double-blind treatment phase). During single-blind placebo lead-in phases, participants receive 3 capsules of 150-mg placebo twice daily (BID) for 14 days. During double-blind treatment phases, participants receive 3 capsules of 150-mg GLWL-01 (450 mg total dose) BID or identical placebo BID for 28 days
Overall Study STARTED	10	9
Overall Study COMPLETED	10	9
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment Sequence 1 n=10 Participants GLWL-01 (450mg) twice a day/ Placebo. Participants randomized to 1 of 2 treatment sequences; GLWL-01/placebo or placebo/GLWL-01 (Treatment Period 1 double-blind treatment phase/Treatment Period 2 double-blind treatment phase). During single-blind placebo lead-in phases, participants receive 3 capsules of 150-mg placebo twice daily (BID) for 14 days. During double-blind treatment phases, participants receive 3 capsules of 150-mg GLWL-01 (450 mg total dose) BID or identical placebo BID for 28 days	Treatment Sequence 2 n=9 Participants Placebo / GLWL-01 (450mg), twice a day. Participants randomized to 1 of 2 treatment sequences; GLWL-01/placebo or placebo/GLWL-01 (Treatment Period 1 double-blind treatment phase/Treatment Period 2 double-blind treatment phase). During single-blind placebo lead-in phases, participants receive 3 capsules of 150-mg placebo twice daily (BID) for 14 days. During double-blind treatment phases, participants receive 3 capsules of 150-mg GLWL-01 (450 mg total dose) BID or identical placebo BID for 28 days	Total n=19 Participants Total of all reporting groups
Age, Continuous	22.0 years STANDARD_DEVIATION 5.54 • n=5 Participants	22.1 years STANDARD_DEVIATION 4.83 • n=7 Participants	22.1 years STANDARD_DEVIATION 5.07 • n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants	5 Participants n=7 Participants	9 Participants n=5 Participants
Sex: Female, Male Male	6 Participants n=5 Participants	4 Participants n=7 Participants	10 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	9 Participants n=5 Participants	8 Participants n=7 Participants	17 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	10 Participants n=5 Participants	9 Participants n=7 Participants	19 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment Canada	2 participants n=5 Participants	3 participants n=7 Participants	5 participants n=5 Participants
Region of Enrollment United States	8 participants n=5 Participants	6 participants n=7 Participants	14 participants n=5 Participants

PRIMARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	GLWL-01 n=19 Participants Participants who received GLWL in either double blind treatment phase	Placebo n=19 Participants Participants who received placebo in either double blind treatment phase
Post-treatment Total Score on the Hyperphagia Questionnaire for Clinical Trials (HQ-CT)	15.9 score on a scale Standard Error 1.21	14.7 score on a scale Standard Error 1.23

SECONDARY outcome

Timeframe: Baseline up to approximately 18 weeks

Evaluate the safety and tolerability of GLWL-01

Outcome measures

Outcome measures
Measure	GLWL-01 n=19 Participants Participants who received GLWL in either double blind treatment phase	Placebo n=19 Participants Participants who received placebo in either double blind treatment phase
Number of Participants With One or More Treatment Emergent Adverse Events (AEs) or Any Serious AEs	8 Participants	7 Participants

SECONDARY outcome

Timeframe: Up to approximately 4 weeks of double-blind treatment

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

Outcome measures

Outcome measures
Measure	GLWL-01 n=19 Participants Participants who received GLWL in either double blind treatment phase	Placebo n=19 Participants Participants who received placebo in either double blind treatment phase
Caregiver Global Impression of Change (CGIC)	4.0 score on a scale Standard Error 0.18	3.8 score on a scale Standard Error 0.19

SECONDARY outcome

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

Population: Because PK data were collected only during Treatment 1, and the patients were randomized 1:1 to GLWL-01 or placebo, PK data were available from 9 patients. Evaluable data only available to compute AUC for some participants

Pharmacokinetics (PK) after single and multiple oral dosing

Outcome measures

Outcome measures
Measure	GLWL-01 n=7 Participants Participants who received GLWL in either double blind treatment phase	Placebo n=3 Participants Participants who received placebo in either double blind treatment phase
Area Under the Concentration Versus Time Curve From Time Zero to 12 Hours (AUC0-12)	55608 ng*h/mL Geometric Coefficient of Variation 23.6	121620 ng*h/mL Geometric Coefficient of Variation 23.7

SECONDARY outcome

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

Pharmacokinetics after single and multiple oral dosing

Outcome measures

Outcome measures
Measure	GLWL-01 n=9 Participants Participants who received GLWL in either double blind treatment phase	Placebo n=9 Participants Participants who received placebo in either double blind treatment phase
Maximum Observed Drug Concentration (Cmax)	7885 ng/mL Geometric Coefficient of Variation 34.4	13695 ng/mL Geometric Coefficient of Variation 29.2

Adverse Events

GLWL

Serious events: 0 serious events

Other events: 8 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 7 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Additional Information

Caroline Fortier

GLWL Research Inc

Phone: 1.514.708.4417

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: OTHER

Measure	GLWL n=19 participants at risk Participants who received GLWL in either double blind treatment phase	Placebo n=19 participants at risk Participants who received placebo in either double blind treatment phase
Gastrointestinal disorders Constipation	5.3% 1/19 • Number of events 1 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.	0.00% 0/19 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.
Gastrointestinal disorders Diarrhoea	15.8% 3/19 • Number of events 3 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.	0.00% 0/19 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.
Gastrointestinal disorders Dyspepsia	5.3% 1/19 • Number of events 1 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.	0.00% 0/19 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.
Gastrointestinal disorders Faeces discoloured	5.3% 1/19 • Number of events 1 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.	0.00% 0/19 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.
Gastrointestinal disorders Faeces soft	5.3% 1/19 • Number of events 1 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.	0.00% 0/19 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.
General disorders Facial pain	0.00% 0/19 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.	5.3% 1/19 • Number of events 1 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.
Infections and infestations Folliculitis	0.00% 0/19 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.	5.3% 1/19 • Number of events 1 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.
Infections and infestations Nail infection	5.3% 1/19 • Number of events 1 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.	0.00% 0/19 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.
Infections and infestations Sinusitis	0.00% 0/19 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.	5.3% 1/19 • Number of events 1 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.
Infections and infestations Upper respiratory tract infection	5.3% 1/19 • Number of events 1 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.	5.3% 1/19 • Number of events 1 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.
Infections and infestations Viral upper respiratory tract infection	10.5% 2/19 • Number of events 2 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.	5.3% 1/19 • Number of events 1 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.
Musculoskeletal and connective tissue disorders Muscle spasms	0.00% 0/19 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.	5.3% 1/19 • Number of events 1 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.
Musculoskeletal and connective tissue disorders Pain in extremity	0.00% 0/19 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.	10.5% 2/19 • Number of events 2 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.
Nervous system disorders Headache	15.8% 3/19 • Number of events 3 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.	21.1% 4/19 • Number of events 5 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.
Nervous system disorders Somnolence	0.00% 0/19 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.	5.3% 1/19 • Number of events 2 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.
Nervous system disorders Agitation	5.3% 1/19 • Number of events 1 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.	0.00% 0/19 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.
Nervous system disorders Anxiety	0.00% 0/19 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.	10.5% 2/19 • Number of events 2 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.
Reproductive system and breast disorders Amenorrhoea	5.3% 1/19 • Number of events 1 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.	0.00% 0/19 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.
Reproductive system and breast disorders Menstruation delayed	5.3% 1/19 • Number of events 1 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.	0.00% 0/19 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.
Reproductive system and breast disorders Menstruation irregular	5.3% 1/19 • Number of events 1 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.	0.00% 0/19 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.
Respiratory, thoracic and mediastinal disorders Cough	5.3% 1/19 • Number of events 1 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.	0.00% 0/19 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.
Respiratory, thoracic and mediastinal disorders Nasal Congestion	0.00% 0/19 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.	5.3% 1/19 • Number of events 1 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	5.3% 1/19 • Number of events 1 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.	0.00% 0/19 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.
Respiratory, thoracic and mediastinal disorders Throat irritation	5.3% 1/19 • Number of events 1 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.	0.00% 0/19 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.
Skin and subcutaneous tissue disorders Ecchymosis	0.00% 0/19 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.	5.3% 1/19 • Number of events 1 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.
Skin and subcutaneous tissue disorders Pruritus	0.00% 0/19 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.	5.3% 1/19 • Number of events 3 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.
Skin and subcutaneous tissue disorders Skin fissures	0.00% 0/19 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.	5.3% 1/19 • Number of events 1 • Up to 18 weeks Treatment-emergent adverse event: An untoward medical occurrence that emerges during a defined treatment period, having been absent pretreatment, or worsens relative to the pretreatment state, and does not necessarily have to have a causal relationship with this treatment.