Trial Outcomes & Findings for Pilot Efficacy and Safety Study of AQX-1125 in Atopic Dermatitis (NCT NCT02324972)

NCT ID: NCT02324972

Last Updated: 2018-10-05

Results Overview

The TLSS is an assessment of the severity of each of the following three signs: erythema, papulation/infiltration, excoriation and lichenification. Each of these items are rated using a 4-point scale severity where 0 is clear, 1 = mild, 2= moderate and 3 = severe. These ratings are then added to create a total score ranging from 0 to 12

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 54 participants
• Primary outcome timeframe: 12 weeks
• Results posted on: 2018-10-05

Participant Flow

Participant milestones

Measure	AQX-1125 1 x AQX-1125 Capsule daily AQX-1125: Synthetic SHIP1 activator	Placebo 1 x placebo capsule daily Placebo: Double blind placebo capsule
Overall Study STARTED	26	28
Overall Study COMPLETED	19	23
Overall Study NOT COMPLETED	7	5

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Pilot Efficacy and Safety Study of AQX-1125 in Atopic Dermatitis

Baseline characteristics by cohort

Measure	AQX-1125 n=26 Participants 1 x AQX-1125 Capsule daily AQX-1125: Synthetic SHIP1 activator	Placebo n=28 Participants 1 x placebo capsule daily Placebo	Total n=54 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	26 Participants n=5 Participants	28 Participants n=7 Participants	54 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Sex: Female, Male Female	18 Participants n=5 Participants	14 Participants n=7 Participants	32 Participants n=5 Participants
Sex: Female, Male Male	8 Participants n=5 Participants	14 Participants n=7 Participants	22 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	24 Participants n=5 Participants	28 Participants n=7 Participants	52 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	1 Participants n=5 Participants	1 Participants n=7 Participants	2 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	6 Participants n=5 Participants	1 Participants n=7 Participants	7 Participants n=5 Participants
Race (NIH/OMB) White	19 Participants n=5 Participants	25 Participants n=7 Participants	44 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	1 Participants n=7 Participants	1 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	26 participants n=5 Participants	28 participants n=7 Participants	54 participants n=5 Participants
Weight	72.96 kg STANDARD_DEVIATION 19.44 • n=5 Participants	76.30 kg STANDARD_DEVIATION 16.06 • n=7 Participants	74.69 kg STANDARD_DEVIATION 17.67 • n=5 Participants
BMI	26.4 kg/m2 STANDARD_DEVIATION 6.17 • n=5 Participants	27.1 kg/m2 STANDARD_DEVIATION 5.28 • n=7 Participants	26.8 kg/m2 STANDARD_DEVIATION 5.68 • n=5 Participants

PRIMARY outcome

Timeframe: 12 weeks

Population: The intent to treat analysis population (ITT) is the set of subjects who were randomized and received at least one dose of study medication.

The TLSS is an assessment of the severity of each of the following three signs: erythema, papulation/infiltration, excoriation and lichenification. Each of these items are rated using a 4-point scale severity where 0 is clear, 1 = mild, 2= moderate and 3 = severe. These ratings are then added to create a total score ranging from 0 to 12

Outcome measures

Measure	AQX-1125 n=26 Participants 1 x AQX-1125 Capsule daily AQX-1125: Synthetic SHIP1 activator	Placebo n=28 Participants 1 x placebo capsule daily Placebo: Double blind placebo capsule
Change From Baseline in Total Lesion Symptom Score (TLSS) Baseline	6.3 units on a scale Standard Deviation 1.16	6.6 units on a scale Standard Deviation 1.31
Change From Baseline in Total Lesion Symptom Score (TLSS) Week 12 (Change from baseline)	-1.9 units on a scale Standard Deviation 2.19	-1.9 units on a scale Standard Deviation 2.41

SECONDARY outcome

Timeframe: 12 weeks

Population: The intent to treat analysis population (ITT) is the set of subjects who were randomized and received at least one dose of study medication.

The IGA is a global assessment of the current state of the disease. It is a 5-point morphological assessment of overall disease severity and will be determined according to the following categories: 0 (clear), 1 (almost clear), 2 (mild), 3 (moderate) and 4 (severe).

Outcome measures

Measure	AQX-1125 n=26 Participants 1 x AQX-1125 Capsule daily AQX-1125: Synthetic SHIP1 activator	Placebo n=28 Participants 1 x placebo capsule daily Placebo: Double blind placebo capsule
Change From Baseline in Investigator's Global Assessment (IGA) Baseline	2.6 units on a scale Standard Deviation 0.50	2.8 units on a scale Standard Deviation 0.52
Change From Baseline in Investigator's Global Assessment (IGA) Week 12 (Change from baseline)	-0.7 units on a scale Standard Deviation 0.98	-0.8 units on a scale Standard Deviation 0.87

SECONDARY outcome

Timeframe: 12 weeks

Population: The intent to treat analysis population (ITT) is the set of subjects who were randomized and received at least one dose of study medication.

The Eczema Area and Severity Index (EASI) quantifies the severity of a subject's atopic dermatitis based on both lesion severity and the percentage of body surface area (BSA) affected. Lesions are assessed on four domains; 1) erythema, 2) induration/papulation, 3) excoriation and 4) lichenification. Each domain is scored separately over four body regions (head/neck, upper limbs, trunk and lower limbs) on a scale of 0-3 (0 = none, 1 = mild, 2 = moderate and 3 = severe) with adjustment for the percentage of BSA involved for each body region and for the proportion of the body region relative to the whole body. The sum of these scores provides the EASI total score, ranging from 0 to 72 with a higher score indicating greater severity of atopic dermatitis.

Outcome measures

Measure	AQX-1125 n=26 Participants 1 x AQX-1125 Capsule daily AQX-1125: Synthetic SHIP1 activator	Placebo n=28 Participants 1 x placebo capsule daily Placebo: Double blind placebo capsule
Change From Baseline in Eczema Area and Severity Index (EASI) Score Baseline	5.58 units on a scale Standard Deviation 3.34	5.35 units on a scale Standard Deviation 2.24
Change From Baseline in Eczema Area and Severity Index (EASI) Score Week 12 (Change from baseline)	-2.84 units on a scale Standard Deviation 1.55	-2.12 units on a scale Standard Deviation 3.19

SECONDARY outcome

Timeframe: 12 weeks

Population: The intent to treat analysis population (ITT) is the set of subjects who were randomized and received at least one dose of study medication.

The severity scoring of atopic dermatitis (SCORAD) index is a standard tool to assess the atopic dermatitis (AD) severity in clinical studies. Six items; erythema, edema/papulation, oozing/crusts, excoriation, lichenification and dryness are measured on a scale from 0-3 for a total of 18 points, with a higher score indicating greater severity. The percentage of BSA affected by AD is evaluated (percentage divided by 5) and added to the total SCORAD score. Loss of sleep and pruritus are also evaluated by subjects on a visual analog scale (scores ranging from 0-10) with a higher score indicating greater severity. The sum of these measures represents the SCORAD which ranges from 0 (absent disease) to 103 (severe disease).

Outcome measures

Measure	AQX-1125 n=26 Participants 1 x AQX-1125 Capsule daily AQX-1125: Synthetic SHIP1 activator	Placebo n=28 Participants 1 x placebo capsule daily Placebo: Double blind placebo capsule
Change From Baseline in Scoring Atopic Dermatitis (SCORAD) Score Baseline	36.19 units on a scale Standard Deviation 8.70	37.21 units on a scale Standard Deviation 6.38
Change From Baseline in Scoring Atopic Dermatitis (SCORAD) Score Week 12 (Change from baseline)	-12.06 units on a scale Standard Deviation 11.15	-11.54 units on a scale Standard Deviation 11.49

SECONDARY outcome

Timeframe: 12 weeks

Population: The intent to treat analysis population (ITT) is the set of subjects who were randomized and received at least one dose of study medication.

The POEM is a 7-item self-assessment questionnaire that assesses disease symptoms (dryness, itching, flaking, cracking, sleep loss, bleeding and weeping) on a scale ranging from 0-4 (0 = no days, 1 = 1-2 days, 2 = 3-4 days, 3 = 5-6 days, 4 = everyday). The sum of the 7 items gives the total POEM score of 0 (absent disease) to 28 (severe disease). High scores are indicative of more severe disease and poor quality of life.

Outcome measures

Measure	AQX-1125 n=26 Participants 1 x AQX-1125 Capsule daily AQX-1125: Synthetic SHIP1 activator	Placebo n=28 Participants 1 x placebo capsule daily Placebo: Double blind placebo capsule
Change From Baseline in Patient Oriented Eczema Measure (POEM) Score Baseline	16.7 units on a scale Standard Deviation 6.98	15.9 units on a scale Standard Deviation 5.48
Change From Baseline in Patient Oriented Eczema Measure (POEM) Score Week 12 (Change from baseline)	-5.6 units on a scale Standard Deviation 9.70	-6.5 units on a scale Standard Deviation 5.82

Adverse Events

AQX-1125

Serious events: 0 serious events

Other events: 17 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 14 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	AQX-1125 n=26 participants at risk 1 x AQX-1125 Capsule daily AQX-1125: Synthetic SHIP1 activator	Placebo n=28 participants at risk 1 x placebo capsule daily Placebo: Double blind placebo capsule
Gastrointestinal disorders Nausea	15.4% 4/26 • Adverse events were collected from Day 0 after 1st dose of study drug until discharge from study at Week 16 Adverse events were documented by the investigator as reported by the subject, and through the asking of nonleading questions by the investigator. In addition clinically significant abnormal findings from safety assessments including; vital signs, physical exam, ECG, clinical labs and ophthalmology exam were also documented as adverse events.	0.00% 0/28 • Adverse events were collected from Day 0 after 1st dose of study drug until discharge from study at Week 16 Adverse events were documented by the investigator as reported by the subject, and through the asking of nonleading questions by the investigator. In addition clinically significant abnormal findings from safety assessments including; vital signs, physical exam, ECG, clinical labs and ophthalmology exam were also documented as adverse events.
Gastrointestinal disorders Diarrhoea	7.7% 2/26 • Adverse events were collected from Day 0 after 1st dose of study drug until discharge from study at Week 16 Adverse events were documented by the investigator as reported by the subject, and through the asking of nonleading questions by the investigator. In addition clinically significant abnormal findings from safety assessments including; vital signs, physical exam, ECG, clinical labs and ophthalmology exam were also documented as adverse events.	3.6% 1/28 • Adverse events were collected from Day 0 after 1st dose of study drug until discharge from study at Week 16 Adverse events were documented by the investigator as reported by the subject, and through the asking of nonleading questions by the investigator. In addition clinically significant abnormal findings from safety assessments including; vital signs, physical exam, ECG, clinical labs and ophthalmology exam were also documented as adverse events.
Gastrointestinal disorders Abdominal Pain	7.7% 2/26 • Adverse events were collected from Day 0 after 1st dose of study drug until discharge from study at Week 16 Adverse events were documented by the investigator as reported by the subject, and through the asking of nonleading questions by the investigator. In addition clinically significant abnormal findings from safety assessments including; vital signs, physical exam, ECG, clinical labs and ophthalmology exam were also documented as adverse events.	0.00% 0/28 • Adverse events were collected from Day 0 after 1st dose of study drug until discharge from study at Week 16 Adverse events were documented by the investigator as reported by the subject, and through the asking of nonleading questions by the investigator. In addition clinically significant abnormal findings from safety assessments including; vital signs, physical exam, ECG, clinical labs and ophthalmology exam were also documented as adverse events.
Gastrointestinal disorders Dyspepsia	7.7% 2/26 • Adverse events were collected from Day 0 after 1st dose of study drug until discharge from study at Week 16 Adverse events were documented by the investigator as reported by the subject, and through the asking of nonleading questions by the investigator. In addition clinically significant abnormal findings from safety assessments including; vital signs, physical exam, ECG, clinical labs and ophthalmology exam were also documented as adverse events.	0.00% 0/28 • Adverse events were collected from Day 0 after 1st dose of study drug until discharge from study at Week 16 Adverse events were documented by the investigator as reported by the subject, and through the asking of nonleading questions by the investigator. In addition clinically significant abnormal findings from safety assessments including; vital signs, physical exam, ECG, clinical labs and ophthalmology exam were also documented as adverse events.
Infections and infestations Nasopharyngitis	23.1% 6/26 • Adverse events were collected from Day 0 after 1st dose of study drug until discharge from study at Week 16 Adverse events were documented by the investigator as reported by the subject, and through the asking of nonleading questions by the investigator. In addition clinically significant abnormal findings from safety assessments including; vital signs, physical exam, ECG, clinical labs and ophthalmology exam were also documented as adverse events.	21.4% 6/28 • Adverse events were collected from Day 0 after 1st dose of study drug until discharge from study at Week 16 Adverse events were documented by the investigator as reported by the subject, and through the asking of nonleading questions by the investigator. In addition clinically significant abnormal findings from safety assessments including; vital signs, physical exam, ECG, clinical labs and ophthalmology exam were also documented as adverse events.
Infections and infestations Folliculitis	0.00% 0/26 • Adverse events were collected from Day 0 after 1st dose of study drug until discharge from study at Week 16 Adverse events were documented by the investigator as reported by the subject, and through the asking of nonleading questions by the investigator. In addition clinically significant abnormal findings from safety assessments including; vital signs, physical exam, ECG, clinical labs and ophthalmology exam were also documented as adverse events.	10.7% 3/28 • Adverse events were collected from Day 0 after 1st dose of study drug until discharge from study at Week 16 Adverse events were documented by the investigator as reported by the subject, and through the asking of nonleading questions by the investigator. In addition clinically significant abnormal findings from safety assessments including; vital signs, physical exam, ECG, clinical labs and ophthalmology exam were also documented as adverse events.
Nervous system disorders Headache	26.9% 7/26 • Adverse events were collected from Day 0 after 1st dose of study drug until discharge from study at Week 16 Adverse events were documented by the investigator as reported by the subject, and through the asking of nonleading questions by the investigator. In addition clinically significant abnormal findings from safety assessments including; vital signs, physical exam, ECG, clinical labs and ophthalmology exam were also documented as adverse events.	14.3% 4/28 • Adverse events were collected from Day 0 after 1st dose of study drug until discharge from study at Week 16 Adverse events were documented by the investigator as reported by the subject, and through the asking of nonleading questions by the investigator. In addition clinically significant abnormal findings from safety assessments including; vital signs, physical exam, ECG, clinical labs and ophthalmology exam were also documented as adverse events.
Skin and subcutaneous tissue disorders Dermatitis Atopic	15.4% 4/26 • Adverse events were collected from Day 0 after 1st dose of study drug until discharge from study at Week 16 Adverse events were documented by the investigator as reported by the subject, and through the asking of nonleading questions by the investigator. In addition clinically significant abnormal findings from safety assessments including; vital signs, physical exam, ECG, clinical labs and ophthalmology exam were also documented as adverse events.	7.1% 2/28 • Adverse events were collected from Day 0 after 1st dose of study drug until discharge from study at Week 16 Adverse events were documented by the investigator as reported by the subject, and through the asking of nonleading questions by the investigator. In addition clinically significant abnormal findings from safety assessments including; vital signs, physical exam, ECG, clinical labs and ophthalmology exam were also documented as adverse events.
Skin and subcutaneous tissue disorders Pruritus	11.5% 3/26 • Adverse events were collected from Day 0 after 1st dose of study drug until discharge from study at Week 16 Adverse events were documented by the investigator as reported by the subject, and through the asking of nonleading questions by the investigator. In addition clinically significant abnormal findings from safety assessments including; vital signs, physical exam, ECG, clinical labs and ophthalmology exam were also documented as adverse events.	3.6% 1/28 • Adverse events were collected from Day 0 after 1st dose of study drug until discharge from study at Week 16 Adverse events were documented by the investigator as reported by the subject, and through the asking of nonleading questions by the investigator. In addition clinically significant abnormal findings from safety assessments including; vital signs, physical exam, ECG, clinical labs and ophthalmology exam were also documented as adverse events.

Additional Information

Clinical

Aquinox Pharmaceuticals (Canada) Inc.

Phone: +1 604-629-9223

Email: clinical@aqxpharma.com

Results disclosure agreements

• Principal investigator is a sponsor employee Investigators shall not publish any articles or make any presentations or communications (including any written, oral, or electronic manuscript, abstract, presentation, or other publication) relating to the Services, Sponsor Information, Study Drug or other Material, Deliverables or other Developments, in whole or in part, without the prior written consent of Sponsor.
• Publication restrictions are in place

Restriction type: OTHER