Trial Outcomes & Findings for Omalizumab in Chronic Spontaneous Urticaria Patients Non Responding to Initial Standard antihistaminE Treatment (NCT NCT02550106)

NCT ID: NCT02550106

Last Updated: 2020-02-25

Results Overview

Number of participants with an Urticaria Control Test score of greater than or equal to 12 at Week 12 The UCT is a simple 4-item tool. A score between 0 and 4 is assigned to every answer option. Subsequently, the scores for all 4 questions are summed up. Accordingly, the minimum and maximum UCT scores are 0 and 16, with 16 points indicating complete disease control.

• Recruitment status: COMPLETED
• Study phase: PHASE4
• Target enrollment: 136 participants
• Primary outcome timeframe: WEEK 12
• Results posted on: 2020-02-25

Participant Flow

Full Analysis Set

Participant milestones

Measure	OMALIZUMAB sub cutaneous injections of 300 mg every 4 weeks until Week 8
Overall Study STARTED	136
Overall Study COMPLETED	124
Overall Study NOT COMPLETED	12

Reasons for withdrawal

Measure	OMALIZUMAB sub cutaneous injections of 300 mg every 4 weeks until Week 8
Overall Study Adverse Event	1
Overall Study Lack of Efficacy	3
Overall Study Protocol Violation	6
Overall Study Withdrawal by Subject	2

Baseline Characteristics

Omalizumab in Chronic Spontaneous Urticaria Patients Non Responding to Initial Standard antihistaminE Treatment

Baseline characteristics by cohort

Measure	OMALIZUMAB n=136 Participants sub cutaneous injections of 300 mg every 4 weeks until Week 8
Age, Continuous	44.4 years STANDARD_DEVIATION 12.67 • n=5 Participants
Sex: Female, Male Female	106 Participants n=5 Participants
Sex: Female, Male Male	30 Participants n=5 Participants

PRIMARY outcome

Timeframe: WEEK 12

Population: (Full analysis set, LOCF)

Number of participants with an Urticaria Control Test score of greater than or equal to 12 at Week 12

The UCT is a simple 4-item tool. A score between 0 and 4 is assigned to every answer option. Subsequently, the scores for all 4 questions are summed up. Accordingly, the minimum and maximum UCT scores are 0 and 16, with 16 points indicating complete disease control.

Outcome measures

Measure	OMALIZUMAB With ANGIOEDEMA n=136 Participants sub cutaneous injections of 300 mg every 4 weeks until W8	OMALIZUMAB Without Angioedema sub cutaneaous injections of 300 mg every 4 weeks until Week 8
Percent of Participants With an Urticaria Control Test [UCT] Score of Greater Than or Equal to 12	75.0 percent of participants Interval 66.9 to 82.0	—

SECONDARY outcome

Timeframe: WEEK 12

Population: Full Analysis Set 2 patients with angioedema status missing at baseline were not included so that FAS = 83 + 51 + 2= 136

2 patients with angioedema status were missing at baseline and not included

Outcome measures

Measure	OMALIZUMAB With ANGIOEDEMA n=83 Participants sub cutaneous injections of 300 mg every 4 weeks until W8	OMALIZUMAB Without Angioedema n=51 Participants sub cutaneaous injections of 300 mg every 4 weeks until Week 8
Percent of Participants With UAS7≤6 (Patients Achieving Disease Control), in Adult Patients With CSU, With or Without the Presence of Angioedema	69.6 percent of participants Interval 58.2 to 79.5	63.8 percent of participants Interval 48.5 to 77.3

SECONDARY outcome

Timeframe: baseline and week 12

Population: Full Analysis Set (FAS)

A total score of UAS7 is calculated by adding for 7 days the daily UAS. The UAS7 is the sum of the daily UAS over 7 days before baseline and W12. The UAS7 score ranges from 0 to 42 with higher scores reflecting higher activity of the disease.

Scores were categorized into five diseases states : urticaria-free (score =0), well-controlled (scores 1-6), mild (scores 7-15), moderate (scores 16-27) and severe urticaria (scores 28-42).

Outcome measures

Measure	OMALIZUMAB With ANGIOEDEMA n=79 Participants sub cutaneous injections of 300 mg every 4 weeks until W8	OMALIZUMAB Without Angioedema n=47 Participants sub cutaneaous injections of 300 mg every 4 weeks until Week 8
CSU Disease Activity Using the Urticaria Activity Score (UAS7), With or Without the Presence of Angioedema baseline	29.7 scores on a scale Standard Deviation 7.32	29.2 scores on a scale Standard Deviation 6.72
CSU Disease Activity Using the Urticaria Activity Score (UAS7), With or Without the Presence of Angioedema at week 12	6.6 scores on a scale Standard Deviation 10.08	6.5 scores on a scale Standard Deviation 9.11

SECONDARY outcome

Timeframe: baseline and week 12

Population: FAS

The UCT is a questionnaire collecting retrospective information over the last 4 weeks before baseline and W12.

The UCT score ranges from 0 to 16 with higher scores reflecting lower control of the disease.

A score of ≥12 indicates well-controlled urticaria.

Outcome measures

Measure	OMALIZUMAB With ANGIOEDEMA n=82 Participants sub cutaneous injections of 300 mg every 4 weeks until W8	OMALIZUMAB Without Angioedema n=50 Participants sub cutaneaous injections of 300 mg every 4 weeks until Week 8
Urticaria Control Test (UCT) Score According to the Presence of Angioedema at Baseline and Week 12 baseline	3.0 scores on a scale Standard Deviation 2.06	3.6 scores on a scale Standard Deviation 2.55
Urticaria Control Test (UCT) Score According to the Presence of Angioedema at Baseline and Week 12 at week 12	13.1 scores on a scale Standard Deviation 3.95	12.9 scores on a scale Standard Deviation 3.97

SECONDARY outcome

Timeframe: week 16

Population: FAS

The UCT is a simple 4-item tool. A score between 0 and 4 is assigned to every answer option. Subsequently, the scores for all 4 questions are summed up. Accordingly, the minimum and maximum UCT scores are 0 and 16, with 16 points indicating complete disease control.

Outcome measures

Measure	OMALIZUMAB With ANGIOEDEMA n=70 Participants sub cutaneous injections of 300 mg every 4 weeks until W8	OMALIZUMAB Without Angioedema n=42 Participants sub cutaneaous injections of 300 mg every 4 weeks until Week 8
Control of the CSU Using the UCT Score for Patients in Extension Treatment Period Phase at Week 16, With or Without the Presence of Angioedema	14.2 scores on a scale Standard Deviation 2.96	13.4 scores on a scale Standard Deviation 3.40

SECONDARY outcome

Timeframe: week 20

Population: FAS

The UCT is a simple 4-item tool. A score between 0 and 4 is assigned to every answer option. Subsequently, the scores for all 4 questions are summed up. Accordingly, the minimum and maximum UCT scores are 0 and 16, with 16 points indicating complete disease control.

Outcome measures

Measure	OMALIZUMAB With ANGIOEDEMA n=48 Participants sub cutaneous injections of 300 mg every 4 weeks until W8	OMALIZUMAB Without Angioedema n=34 Participants sub cutaneaous injections of 300 mg every 4 weeks until Week 8
Control of the CSU Using the UCT Score for Patients in Extension Treatment Period Phase at Week 20, With or Without the Presence of Angioedema	14.4 scores on a scale Standard Deviation 2.40	13.3 scores on a scale Standard Deviation 4.11

SECONDARY outcome

Timeframe: week 24

Population: FAS

The UCT is a simple 4-item tool. A score between 0 and 4 is assigned to every answer option. Subsequently, the scores for all 4 questions are summed up. Accordingly, the minimum and maximum UCT scores are 0 and 16, with 16 points indicating complete disease control

Outcome measures

Measure	OMALIZUMAB With ANGIOEDEMA n=14 Participants sub cutaneous injections of 300 mg every 4 weeks until W8	OMALIZUMAB Without Angioedema n=12 Participants sub cutaneaous injections of 300 mg every 4 weeks until Week 8
Control of the CSU Using the UCT Score for Patients in Extension Treatment Period Phase at Week 24, With or Without the Presence of Angioedema	14.5 scores on a scale Standard Deviation 2.14	13.4 scores on a scale Standard Deviation 3.75

SECONDARY outcome

Timeframe: week 28

Population: FAS

The UCT is a simple 4-item tool. A score between 0 and 4 is assigned to every answer option. Subsequently, the scores for all 4 questions are summed up. Accordingly, the minimum and maximum UCT scores are 0 and 16, with 16 points indicating complete disease control.

Outcome measures

Measure	OMALIZUMAB With ANGIOEDEMA n=2 Participants sub cutaneous injections of 300 mg every 4 weeks until W8	OMALIZUMAB Without Angioedema n=2 Participants sub cutaneaous injections of 300 mg every 4 weeks until Week 8
Control of the CSU Using the UCT Score for Patients in Extension Treatment Period Phase at Week 28, With or Without the Presence of Angioedema	13.5 scores on a scale Standard Deviation 3.54	12.0 scores on a scale Standard Deviation 5.66

SECONDARY outcome

Timeframe: baseline and week 12

Population: FAS

The Cu-QoL is a questionnaire collecting retrospective information over the last 2 weeks before baseline and W12.

The CU-QoL total score is transformed to range from 0 to 100, with higher scores indicating worse Health Related Quality of life.

Outcome measures

Measure	OMALIZUMAB With ANGIOEDEMA n=80 Participants sub cutaneous injections of 300 mg every 4 weeks until W8	OMALIZUMAB Without Angioedema n=49 Participants sub cutaneaous injections of 300 mg every 4 weeks until Week 8
The Quality of Life Using the Chronic Urticaria Quality of Life (CU-QoL) Questionnaire baseline	68.9 scores on a scale Standard Deviation 15.64	62.4 scores on a scale Standard Deviation 17.76
The Quality of Life Using the Chronic Urticaria Quality of Life (CU-QoL) Questionnaire at 12 weeks	32.5 scores on a scale Standard Deviation 13.33	33.3 scores on a scale Standard Deviation 13.32

SECONDARY outcome

Timeframe: baseline and week 12

Population: FAS

The angioedema Quality of Life Questionnaire is a valuable tool to assess changes of QoL impairment in angioedema patients.

The results of all the answered questions are summed up and transferred to a scale ranging from 0 to 100, with higher scores indicative of a higher QoL impairment

Outcome measures

Measure	OMALIZUMAB With ANGIOEDEMA n=78 Participants sub cutaneous injections of 300 mg every 4 weeks until W8	OMALIZUMAB Without Angioedema sub cutaneaous injections of 300 mg every 4 weeks until Week 8
The Angioedema Quality of Life (AE-QoL) baseline	57.88 scores on a scale Standard Deviation 22.474	—
The Angioedema Quality of Life (AE-QoL) at week 12	16.40 scores on a scale Standard Deviation 20.074	—

SECONDARY outcome

Timeframe: baseline and week 12

Population: FAS

The Dermatology life Quality Index (DLQI) is a ten-question questionnaire used to measure the impact of skin disease on the quality of life of an affected person. It is designed for people aged 16 years and above. Each question is scored from 0 to 3, giving a possible score range form 0 (meaning no impact of skin disease on quality of life) to 30 (meaning maximum impact on quality of life).

Outcome measures

Measure	OMALIZUMAB With ANGIOEDEMA n=79 Participants sub cutaneous injections of 300 mg every 4 weeks until W8	OMALIZUMAB Without Angioedema n=49 Participants sub cutaneaous injections of 300 mg every 4 weeks until Week 8
The Dermatology Life Quality Index (DLQI) baseline	14.2 scores on a scale Standard Deviation 5.39	13.2 scores on a scale Standard Deviation 6.67
The Dermatology Life Quality Index (DLQI) at week 12	2.4 scores on a scale Standard Deviation 3.95	2.7 scores on a scale Standard Deviation 5.12

SECONDARY outcome

Timeframe: baseline and week 12

Population: FAS

The Angioedema Activity Score (AAS) was completed by patients on a daily basis. The AAS is a validated questionnaire to determine the severity and impact of the angioedema episode. Te daily AAS values are added over 7 days before baseline and W12. Weekly AAS (AAS7) scores range from 0 to 105, with higher scores indicative of a higher disease activity.

Outcome measures

Measure	OMALIZUMAB With ANGIOEDEMA n=78 Participants sub cutaneous injections of 300 mg every 4 weeks until W8	OMALIZUMAB Without Angioedema sub cutaneaous injections of 300 mg every 4 weeks until Week 8
Angioedema Activity Using the Angioedema Activity Score (AAS) baseline	32.7 scores on a scale Standard Deviation 27.21	—
Angioedema Activity Using the Angioedema Activity Score (AAS) at week 12	3.7 scores on a scale Standard Deviation 10.40	—

Adverse Events

Omalizumab 300 mg

Serious events: 9 serious events

Other events: 44 other events

Deaths: 0 deaths

Serious adverse events

Measure	Omalizumab 300 mg n=136 participants at risk Omalizumab 300 mg
Infections and infestations PNEUMONIA	0.74% 1/136 • Adverse events were collected from first dose of study treatment until end of study treatment at week 12 An adverse event (AE) is any sign or symptom that occurs during the study treatment plus the # days post treatment
Injury, poisoning and procedural complications CERVICAL VERTEBRAL FRACTURE	0.74% 1/136 • Adverse events were collected from first dose of study treatment until end of study treatment at week 12 An adverse event (AE) is any sign or symptom that occurs during the study treatment plus the # days post treatment
Injury, poisoning and procedural complications FRACTURED SACRUM	0.74% 1/136 • Adverse events were collected from first dose of study treatment until end of study treatment at week 12 An adverse event (AE) is any sign or symptom that occurs during the study treatment plus the # days post treatment
Injury, poisoning and procedural complications LIGAMENT SPRAIN	0.74% 1/136 • Adverse events were collected from first dose of study treatment until end of study treatment at week 12 An adverse event (AE) is any sign or symptom that occurs during the study treatment plus the # days post treatment
Metabolism and nutrition disorders OBESITY	0.74% 1/136 • Adverse events were collected from first dose of study treatment until end of study treatment at week 12 An adverse event (AE) is any sign or symptom that occurs during the study treatment plus the # days post treatment
Musculoskeletal and connective tissue disorders FOOT DEFORMITY	0.74% 1/136 • Adverse events were collected from first dose of study treatment until end of study treatment at week 12 An adverse event (AE) is any sign or symptom that occurs during the study treatment plus the # days post treatment
Musculoskeletal and connective tissue disorders INTERVERTEBRAL DISC PROTRUSION	0.74% 1/136 • Adverse events were collected from first dose of study treatment until end of study treatment at week 12 An adverse event (AE) is any sign or symptom that occurs during the study treatment plus the # days post treatment
Musculoskeletal and connective tissue disorders SACROILIITIS	0.74% 1/136 • Adverse events were collected from first dose of study treatment until end of study treatment at week 12 An adverse event (AE) is any sign or symptom that occurs during the study treatment plus the # days post treatment
Renal and urinary disorders BLADDER DILATATION	0.74% 1/136 • Adverse events were collected from first dose of study treatment until end of study treatment at week 12 An adverse event (AE) is any sign or symptom that occurs during the study treatment plus the # days post treatment
Renal and urinary disorders URINARY INCONTINENCE	0.74% 1/136 • Adverse events were collected from first dose of study treatment until end of study treatment at week 12 An adverse event (AE) is any sign or symptom that occurs during the study treatment plus the # days post treatment
Respiratory, thoracic and mediastinal disorders PHARYNGEAL OEDEMA	0.74% 1/136 • Adverse events were collected from first dose of study treatment until end of study treatment at week 12 An adverse event (AE) is any sign or symptom that occurs during the study treatment plus the # days post treatment

Other adverse events

Measure	Omalizumab 300 mg n=136 participants at risk Omalizumab 300 mg
General disorders ASTHENIA	16.9% 23/136 • Adverse events were collected from first dose of study treatment until end of study treatment at week 12 An adverse event (AE) is any sign or symptom that occurs during the study treatment plus the # days post treatment
Investigations WEIGHT INCREASED	5.1% 7/136 • Adverse events were collected from first dose of study treatment until end of study treatment at week 12 An adverse event (AE) is any sign or symptom that occurs during the study treatment plus the # days post treatment
Musculoskeletal and connective tissue disorders ARTHRALGIA	8.8% 12/136 • Adverse events were collected from first dose of study treatment until end of study treatment at week 12 An adverse event (AE) is any sign or symptom that occurs during the study treatment plus the # days post treatment
Nervous system disorders HEADACHE	16.2% 22/136 • Adverse events were collected from first dose of study treatment until end of study treatment at week 12 An adverse event (AE) is any sign or symptom that occurs during the study treatment plus the # days post treatment

Additional Information

Study Director

Novartis Pharmaceuticals

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 (ie, data from all sites) in the clinical trial
• Publication restrictions are in place

Restriction type: OTHER