Trial Outcomes & Findings for Efficacy and Safety Study of QAW039 in the Treatment of Patients With Moderate to Severe Atopic Dermatitis. (NCT NCT01785602)

Last Updated: 2015-12-15

Results Overview

Investigators assessed presence and severity of erythema, induration/papulation, excoriation, and lichenification in four body areas: head/neck (H), upper limbs (UL), trunk (T), and lower limbs (LL). Investigators assigned a severity score from 0 - 3 for each area (none=0, mild=1, moderate=2, and severe=3). Investigators could assign half-points. Investigators also assigned an area score from 0 (no atopic dermatitis lesion in the area) to 6 (entire area is affected) for each area. The weighting factor was 0.1 for head/neck, 0.2 for upper limbs, 0.3 for trunk, and 0.4 for lower limbs. The total body score for each body region was obtained by multiplying the sum of the severity scores of the four key signs by the area score, then multiplying the result by the constant weighted value assigned to that body region. The sum of these scores gave the EASI total, ranging from 0 to 72. A higher score represented greater disease severity. A negative change from baseline indicates improvement.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

103 participants

Primary outcome timeframe

Baseline, 12 weeks

Results posted on

2015-12-15

Participant Flow

Participant milestones

Participant milestones
Measure	QAW039 Participants received QAW039 450 mg daily by mouth.	Placebo Participants received matching placebo to QAW039.
Overall Study STARTED	76	27
Overall Study COMPLETED	63	18
Overall Study NOT COMPLETED	13	9

Reasons for withdrawal

Reasons for withdrawal
Measure	QAW039 Participants received QAW039 450 mg daily by mouth.	Placebo Participants received matching placebo to QAW039.
Overall Study Withdrawal by Subject	2	0
Overall Study Lost to Follow-up	1	1
Overall Study Adverse Event	3	5
Overall Study Administrative problems	7	3

Baseline Characteristics

Efficacy and Safety Study of QAW039 in the Treatment of Patients With Moderate to Severe Atopic Dermatitis.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	QAW039 n=76 Participants Participants received QAW039 450 mg daily by mouth.	Placebo n=27 Participants Participants received matching placebo to QAW039.	Total n=103 Participants Total of all reporting groups
Age, Continuous	35.5 Years STANDARD_DEVIATION 13.08 • n=5 Participants	38.7 Years STANDARD_DEVIATION 9.95 • n=7 Participants	36.3 Years STANDARD_DEVIATION 12.37 • n=5 Participants
Sex: Female, Male Female	27 Participants n=5 Participants	12 Participants n=7 Participants	39 Participants n=5 Participants
Sex: Female, Male Male	49 Participants n=5 Participants	15 Participants n=7 Participants	64 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline, 12 weeks

Population: All randomized participants

Outcome measures

Outcome measures
Measure	QAW039 n=76 Participants Participants received QAW039 450 mg daily by mouth.	Placebo n=27 Participants Participants received matching placebo to QAW039.
Change From Baseline in Eczema Area and Severity Index (EASI)	-8.65 Score on a scale Standard Error 0.010	-6.95 Score on a scale Standard Error 0.017

SECONDARY outcome

Timeframe: Baseline, 4 weeks, 8 weeks

Population: All randomized participants

Outcome measures

Outcome measures
Measure	QAW039 n=76 Participants Participants received QAW039 450 mg daily by mouth.	Placebo n=27 Participants Participants received matching placebo to QAW039.
Change From Baseline in Eczema Area and Severity Index Week 4	-6.22 Score on a scale Standard Error 0.010	-5.89 Score on a scale Standard Error 0.017
Change From Baseline in Eczema Area and Severity Index Week 8	-7.49 Score on a scale Standard Error 0.010	-5.61 Score on a scale Standard Error 0.017

Adverse Events

QAW039

Serious events: 1 serious events

Other events: 44 other events

Deaths: 0 deaths

Placebo

Serious events: 4 serious events

Other events: 16 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	QAW039 n=76 participants at risk Participants received QAW039 450 mg daily by mouth.	Placebo n=27 participants at risk Participants received matching placebo to QAW039.
Cardiac disorders Atrial fibrillation	1.3% 1/76 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)	0.00% 0/27 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)
Infections and infestations Diverticulitis	0.00% 0/76 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)	3.7% 1/27 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)
Skin and subcutaneous tissue disorders Dermatitis atopic	0.00% 0/76 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)	11.1% 3/27 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)

Other adverse events

Additional Information

Study Director

Novartis

Phone: 862-778-8300

Results disclosure agreements

Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial or disclosure of trial results in their entirety.
Publication restrictions are in place

Restriction type: OTHER

Measure	QAW039 n=76 participants at risk Participants received QAW039 450 mg daily by mouth.	Placebo n=27 participants at risk Participants received matching placebo to QAW039.
Gastrointestinal disorders Abdominal pain	5.3% 4/76 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)	3.7% 1/27 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)
Gastrointestinal disorders Abdominal pain upper	3.9% 3/76 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)	7.4% 2/27 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)
Gastrointestinal disorders Diarrhoea	13.2% 10/76 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)	7.4% 2/27 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)
Gastrointestinal disorders Nausea	7.9% 6/76 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)	11.1% 3/27 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)
General disorders Fatigue	6.6% 5/76 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)	3.7% 1/27 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)
Infections and infestations Nasopharyngitis	14.5% 11/76 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)	3.7% 1/27 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)
Infections and infestations Oral herpes	5.3% 4/76 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)	3.7% 1/27 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)
Musculoskeletal and connective tissue disorders Back pain	1.3% 1/76 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)	7.4% 2/27 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)
Nervous system disorders Dizziness	3.9% 3/76 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)	7.4% 2/27 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)
Nervous system disorders Headache	9.2% 7/76 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)	14.8% 4/27 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)
Nervous system disorders Hypoaesthesia	0.00% 0/76 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)	7.4% 2/27 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)
Nervous system disorders Tension headache	1.3% 1/76 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)	7.4% 2/27 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)
Skin and subcutaneous tissue disorders Dermatitis atopic	31.6% 24/76 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)	33.3% 9/27 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)
Skin and subcutaneous tissue disorders Pruritus	3.9% 3/76 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)	7.4% 2/27 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)
Vascular disorders Hot flush	0.00% 0/76 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)	7.4% 2/27 • Serious adverse events were collected from day 1 until 4 weeks after end of study (week 20)