Trial Outcomes & Findings for Study to Investigate the Efficacy and Safety of QGE031 in Adolescent Patients With Chronic Spontaneous Urticaria (CSU) (NCT NCT03437278)

Last Updated: 2026-01-13

Results Overview

UAS7 is a self-reported scoring system to evaluate urticaria signs and symptoms. UAS7 is the sum of daily urticaria activity scores (UAS) over a seven-day period. The possible range of UAS7 score is 0 to 42 (0 to 6 for daily UAS x 7 days). A higher urticaria activity score indicates more severe symptoms. A negative change score from baseline indicates improvement. Baseline was calculated using data from the 7 days prior to the first treatment date. To handle the missing data, if a participant had at least 4 non-missing daily scores within the 7 days prior to a study visit, the weekly score was calculated as the sum of the available scores in that week, divided by the number of days with daily scores available, multiplied by 7. However, if there were less than 4 non-missing daily scores within the prior 7 days, then the weekly score was missing for the week.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

49 participants

Primary outcome timeframe

Baseline, week 24

Results posted on

2026-01-13

Participant Flow

Participants were recruited from 20 sites: Argentina (3), Belgium (1), Canada (2), Germany (2), Hungary (1), India (3), Russia (3), Spain (2), Taiwan (1) and Turkey (2).

Participants underwent a Screening period of up to 4 weeks.

Participant milestones

Participant milestones
Measure	Ligelizumab 24 mg Participants received a dose of ligelizumab 24 mg (low dose) which consisted of one injection of 0.2 ml of ligelizumab 120 mg/ 1 ml vial every 4 weeks from Day 1 to Week 20 (inclusive).	Ligelizumab 120 mg Participants received a dose of ligelizumab 120 mg (high dose) which consisted of one injection of 1 ml of ligelizumab 120 mg/ 1 ml vial every 4 weeks from Day 1 to Week 20 (inclusive).	Placebo + Ligelizumab 120 mg Participants received Placebo which consisted of one injection of 1 ml placebo every 4 weeks from Day 1 to Week 8 (inclusive). From week 12 to week 20 (inclusive), participants received a dose of ligelizumab 120 mg (high dose) which consisted of one injection of 1 ml of ligelizumab 120 mg/ 1 ml vial.
Overall Study STARTED	24	13	12
Overall Study COMPLETED	22	13	10
Overall Study NOT COMPLETED	2	0	2

Reasons for withdrawal

Reasons for withdrawal
Measure	Ligelizumab 24 mg Participants received a dose of ligelizumab 24 mg (low dose) which consisted of one injection of 0.2 ml of ligelizumab 120 mg/ 1 ml vial every 4 weeks from Day 1 to Week 20 (inclusive).	Placebo + Ligelizumab 120 mg Participants received Placebo which consisted of one injection of 1 ml placebo every 4 weeks from Day 1 to Week 8 (inclusive). From week 12 to week 20 (inclusive), participants received a dose of ligelizumab 120 mg (high dose) which consisted of one injection of 1 ml of ligelizumab 120 mg/ 1 ml vial.
Overall Study Adverse Event	0	1
Overall Study Progressive disease	1	0
Overall Study Physician Decision	1	1

Baseline Characteristics

Study to Investigate the Efficacy and Safety of QGE031 in Adolescent Patients With Chronic Spontaneous Urticaria (CSU)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Ligelizumab 24 mg n=24 Participants Participants received a dose of ligelizumab 24 mg (low dose) which consisted of one injection of 0.2 ml of ligelizumab 120 mg/ 1 ml vial every 4 weeks from Day 1 to Week 20 (inclusive).	Ligelizumab 120 mg n=13 Participants Participants received a dose of ligelizumab 120 mg (high dose) which consisted of one injection of 1 ml of ligelizumab 120 mg/ 1 ml vial every 4 weeks from Day 1 to Week 20 (inclusive).	Placebo + Ligelizumab 120 mg n=12 Participants Participants received Placebo which consisted of one injection of 1 ml placebo every 4 weeks from Day 1 to Week 8 (inclusive). From week 12 to week 20 (inclusive), participants received a dose of ligelizumab 120 mg (high dose) which consisted of one injection of 1 ml of ligelizumab 120 mg/ 1 ml vial.	Total n=49 Participants Total of all reporting groups
Age, Continuous	14.9 Years STANDARD_DEVIATION 1.94 • n=210 Participants	15.2 Years STANDARD_DEVIATION 1.41 • n=19 Participants	14.4 Years STANDARD_DEVIATION 1.51 • n=123 Participants	14.8 Years STANDARD_DEVIATION 1.70 • n=123 Participants
Sex: Female, Male Female	10 Participants n=210 Participants	9 Participants n=19 Participants	9 Participants n=123 Participants	28 Participants n=123 Participants
Sex: Female, Male Male	14 Participants n=210 Participants	4 Participants n=19 Participants	3 Participants n=123 Participants	21 Participants n=123 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=210 Participants	0 Participants n=19 Participants	0 Participants n=123 Participants	0 Participants n=123 Participants
Race (NIH/OMB) Asian	7 Participants n=210 Participants	1 Participants n=19 Participants	2 Participants n=123 Participants	10 Participants n=123 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=210 Participants	0 Participants n=19 Participants	0 Participants n=123 Participants	0 Participants n=123 Participants
Race (NIH/OMB) Black or African American	1 Participants n=210 Participants	0 Participants n=19 Participants	0 Participants n=123 Participants	1 Participants n=123 Participants
Race (NIH/OMB) White	16 Participants n=210 Participants	12 Participants n=19 Participants	10 Participants n=123 Participants	38 Participants n=123 Participants
Race (NIH/OMB) More than one race	0 Participants n=210 Participants	0 Participants n=19 Participants	0 Participants n=123 Participants	0 Participants n=123 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=210 Participants	0 Participants n=19 Participants	0 Participants n=123 Participants	0 Participants n=123 Participants

PRIMARY outcome

Timeframe: Baseline, week 24

Population: All participants to whom study treatment was assigned. Only participants with at least 4 non-missing daily scores within the 7 days prior to the study visit at week 24 were analyzed.

Outcome measures

Outcome measures
Measure	Ligelizumab 24 mg n=23 Participants Participants received a dose of ligelizumab 24 mg (low dose) which consisted of one injection of 0.2 ml of ligelizumab 120 mg/ 1 ml vial every 4 weeks from Day 1 to Week 20 (inclusive).	Ligelizumab 120 mg n=13 Participants Participants received a dose of ligelizumab 120 mg (high dose) which consisted of one injection of 1 ml of ligelizumab 120 mg/ 1 ml vial every 4 weeks from Day 1 to Week 20 (inclusive).	Placebo + Ligelizumab 120 mg n=11 Participants Participants received Placebo which consisted of one injection of 1 ml placebo every 4 weeks from Day 1 to Week 8 (inclusive). From week 12 to week 20 (inclusive), participants received a dose of ligelizumab 120 mg (high dose) which consisted of one injection of 1 ml of ligelizumab 120 mg/ 1 ml vial.
Change From Baseline of Weekly Urticaria Activity Score (UAS7) at Week 24	-20.36 Score on a scale Standard Deviation 12.963	-22.50 Score on a scale Standard Deviation 13.503	-21.26 Score on a scale Standard Deviation 14.480

SECONDARY outcome

Timeframe: Baseline, weeks 12 and 40

Population: All participants to whom study treatment was assigned. Only participants with at least 4 non-missing daily scores within the 7 days prior to the specified data points (weeks 12 and 40) were analyzed.

Outcome measures

Outcome measures
Measure	Ligelizumab 24 mg n=24 Participants Participants received a dose of ligelizumab 24 mg (low dose) which consisted of one injection of 0.2 ml of ligelizumab 120 mg/ 1 ml vial every 4 weeks from Day 1 to Week 20 (inclusive).	Ligelizumab 120 mg n=13 Participants Participants received a dose of ligelizumab 120 mg (high dose) which consisted of one injection of 1 ml of ligelizumab 120 mg/ 1 ml vial every 4 weeks from Day 1 to Week 20 (inclusive).	Placebo + Ligelizumab 120 mg n=12 Participants Participants received Placebo which consisted of one injection of 1 ml placebo every 4 weeks from Day 1 to Week 8 (inclusive). From week 12 to week 20 (inclusive), participants received a dose of ligelizumab 120 mg (high dose) which consisted of one injection of 1 ml of ligelizumab 120 mg/ 1 ml vial.
Change From Baseline of Weekly Urticaria Activity Score (UAS7) at Weeks 12 and 40 Week 12	-15.70 Score on a scale Standard Deviation 10.867	-18.38 Score on a scale Standard Deviation 12.268	-12.96 Score on a scale Standard Deviation 13.043
Change From Baseline of Weekly Urticaria Activity Score (UAS7) at Weeks 12 and 40 Week 40	-17.50 Score on a scale Standard Deviation 12.619	-15.65 Score on a scale Standard Deviation 11.096	-19.43 Score on a scale Standard Deviation 17.667

SECONDARY outcome

Timeframe: Weeks 12, 24 and 40

Population: All participants to whom study treatment was assigned. Participants with post-baseline missing data were considered as non-responders.

Outcome measures

Outcome measures
Measure	Ligelizumab 24 mg n=24 Participants Participants received a dose of ligelizumab 24 mg (low dose) which consisted of one injection of 0.2 ml of ligelizumab 120 mg/ 1 ml vial every 4 weeks from Day 1 to Week 20 (inclusive).	Ligelizumab 120 mg n=13 Participants Participants received a dose of ligelizumab 120 mg (high dose) which consisted of one injection of 1 ml of ligelizumab 120 mg/ 1 ml vial every 4 weeks from Day 1 to Week 20 (inclusive).	Placebo + Ligelizumab 120 mg n=12 Participants Participants received Placebo which consisted of one injection of 1 ml placebo every 4 weeks from Day 1 to Week 8 (inclusive). From week 12 to week 20 (inclusive), participants received a dose of ligelizumab 120 mg (high dose) which consisted of one injection of 1 ml of ligelizumab 120 mg/ 1 ml vial.
Percentage of Participants With Complete Response in Weekly Urticaria Activity Score (UAS7) Week 12	4 Participants	5 Participants	2 Participants
Percentage of Participants With Complete Response in Weekly Urticaria Activity Score (UAS7) Week 24	8 Participants	8 Participants	4 Participants
Percentage of Participants With Complete Response in Weekly Urticaria Activity Score (UAS7) Week 40	3 Participants	2 Participants	4 Participants

SECONDARY outcome

Timeframe: Baseline, weeks 12, 24 and 40

ISS7 is the sum of daily Itch Severity Score (ISS) over a seven-day period. The possible range of ISS7 score is 0-21 (0-3 for daily ISS x 7 days), where 0 is defined as complete ISS7 response (no itching) and 21 is the worst score. A negative change score from baseline indicates improvement. Baseline was calculated using data from the 7 days prior to the first treatment date. To handle the missing data, if a participant had at least 4 non-missing daily scores within the 7 days prior to a study visit, the weekly score was calculated as the sum of the available scores in that week, divided by the number of days with daily scores available, multiplied by 7. However, if there were less than 4 non-missing daily scores within the prior 7 days, then the weekly score was missing for the week.

Outcome measures

Outcome measures
Measure	Ligelizumab 24 mg n=24 Participants Participants received a dose of ligelizumab 24 mg (low dose) which consisted of one injection of 0.2 ml of ligelizumab 120 mg/ 1 ml vial every 4 weeks from Day 1 to Week 20 (inclusive).	Ligelizumab 120 mg n=13 Participants Participants received a dose of ligelizumab 120 mg (high dose) which consisted of one injection of 1 ml of ligelizumab 120 mg/ 1 ml vial every 4 weeks from Day 1 to Week 20 (inclusive).	Placebo + Ligelizumab 120 mg n=12 Participants Participants received Placebo which consisted of one injection of 1 ml placebo every 4 weeks from Day 1 to Week 8 (inclusive). From week 12 to week 20 (inclusive), participants received a dose of ligelizumab 120 mg (high dose) which consisted of one injection of 1 ml of ligelizumab 120 mg/ 1 ml vial.
Change From Baseline of Weekly Itch Severity Score (ISS7) Week 12	-7.66 Score on a scale Standard Deviation 5.824	-7.81 Score on a scale Standard Deviation 6.047	-6.34 Score on a scale Standard Deviation 6.936
Change From Baseline of Weekly Itch Severity Score (ISS7) Week 40	-8.73 Score on a scale Standard Deviation 6.656	-6.86 Score on a scale Standard Deviation 5.848	-9.88 Score on a scale Standard Deviation 8.687
Change From Baseline of Weekly Itch Severity Score (ISS7) Week 24	-9.71 Score on a scale Standard Deviation 7.049	-9.85 Score on a scale Standard Deviation 6.488	-10.28 Score on a scale Standard Deviation 7.811

SECONDARY outcome

Timeframe: Weeks 12, 24 and 40

Population: All participants to whom study treatment was assigned. Participants with post-baseline missing data were considered as non-responders.

Outcome measures

Outcome measures
Measure	Ligelizumab 24 mg n=24 Participants Participants received a dose of ligelizumab 24 mg (low dose) which consisted of one injection of 0.2 ml of ligelizumab 120 mg/ 1 ml vial every 4 weeks from Day 1 to Week 20 (inclusive).	Ligelizumab 120 mg n=13 Participants Participants received a dose of ligelizumab 120 mg (high dose) which consisted of one injection of 1 ml of ligelizumab 120 mg/ 1 ml vial every 4 weeks from Day 1 to Week 20 (inclusive).	Placebo + Ligelizumab 120 mg n=12 Participants Participants received Placebo which consisted of one injection of 1 ml placebo every 4 weeks from Day 1 to Week 8 (inclusive). From week 12 to week 20 (inclusive), participants received a dose of ligelizumab 120 mg (high dose) which consisted of one injection of 1 ml of ligelizumab 120 mg/ 1 ml vial.
Percentage of Participants With Complete Response in Weekly Itch Severity Score (ISS7) Week 12	4 Participants	5 Participants	2 Participants
Percentage of Participants With Complete Response in Weekly Itch Severity Score (ISS7) Week 24	9 Participants	8 Participants	4 Participants
Percentage of Participants With Complete Response in Weekly Itch Severity Score (ISS7) Week 40	4 Participants	2 Participants	4 Participants

SECONDARY outcome

Timeframe: Baseline, weeks 12, 24 and 40

HSS7 is the sum of daily Hives Severity Score (HSS) over a seven-day period. The possible range of HSS7 score is 0-21 (0-3 for daily HSS x 7 days), where 0 is defined as complete HSS7 response (no wheals) and 21 is the worst score. A negative change score from baseline indicates improvement. Baseline was calculated using data from the 7 days prior to the first treatment date. To handle the missing data, if a participant had at least 4 non-missing daily scores within the 7 days prior to a study visit, the weekly score was calculated as the sum of the available scores in that week, divided by the number of days with daily scores available, multiplied by 7. However, if there were less than 4 non-missing daily scores within the prior 7 days, then the weekly score was missing for the week.

Outcome measures

Outcome measures
Measure	Ligelizumab 24 mg n=24 Participants Participants received a dose of ligelizumab 24 mg (low dose) which consisted of one injection of 0.2 ml of ligelizumab 120 mg/ 1 ml vial every 4 weeks from Day 1 to Week 20 (inclusive).	Ligelizumab 120 mg n=13 Participants Participants received a dose of ligelizumab 120 mg (high dose) which consisted of one injection of 1 ml of ligelizumab 120 mg/ 1 ml vial every 4 weeks from Day 1 to Week 20 (inclusive).	Placebo + Ligelizumab 120 mg n=12 Participants Participants received Placebo which consisted of one injection of 1 ml placebo every 4 weeks from Day 1 to Week 8 (inclusive). From week 12 to week 20 (inclusive), participants received a dose of ligelizumab 120 mg (high dose) which consisted of one injection of 1 ml of ligelizumab 120 mg/ 1 ml vial.
Change From Baseline of Weekly Hives Severity Score (HSS7) Week 12	-8.03 Score on a scale Standard Deviation 6.307	-10.58 Score on a scale Standard Deviation 7.225	-6.62 Score on a scale Standard Deviation 6.385
Change From Baseline of Weekly Hives Severity Score (HSS7) Week 40	-8.77 Score on a scale Standard Deviation 6.770	-8.78 Score on a scale Standard Deviation 6.084	-9.55 Score on a scale Standard Deviation 9.197
Change From Baseline of Weekly Hives Severity Score (HSS7) Week 24	-10.65 Score on a scale Standard Deviation 6.673	-12.65 Score on a scale Standard Deviation 7.785	-10.98 Score on a scale Standard Deviation 6.842

SECONDARY outcome

Timeframe: Weeks 12, 24 and 40

Population: All participants to whom study treatment was assigned. Participants with post-baseline missing data were considered as non-responders.

Outcome measures

Outcome measures
Measure	Ligelizumab 24 mg n=24 Participants Participants received a dose of ligelizumab 24 mg (low dose) which consisted of one injection of 0.2 ml of ligelizumab 120 mg/ 1 ml vial every 4 weeks from Day 1 to Week 20 (inclusive).	Ligelizumab 120 mg n=13 Participants Participants received a dose of ligelizumab 120 mg (high dose) which consisted of one injection of 1 ml of ligelizumab 120 mg/ 1 ml vial every 4 weeks from Day 1 to Week 20 (inclusive).	Placebo + Ligelizumab 120 mg n=12 Participants Participants received Placebo which consisted of one injection of 1 ml placebo every 4 weeks from Day 1 to Week 8 (inclusive). From week 12 to week 20 (inclusive), participants received a dose of ligelizumab 120 mg (high dose) which consisted of one injection of 1 ml of ligelizumab 120 mg/ 1 ml vial.
Percentage of Participants With Complete Response in Weekly Hives Severity Score (HSS7) Week 12	6 Participants	6 Participants	2 Participants
Percentage of Participants With Complete Response in Weekly Hives Severity Score (HSS7) Week 24	10 Participants	9 Participants	4 Participants
Percentage of Participants With Complete Response in Weekly Hives Severity Score (HSS7) Week 40	5 Participants	2 Participants	5 Participants

SECONDARY outcome

Timeframe: Baseline, weeks 12, 24 and 40

Population: All participants to whom study treatment was assigned. Participants with 2 or more item missing scores at specified data points (weeks 12, 24 and 40) were not analyzed (total score for that data point was considered as missing).

The children dermatology life quality index questionnaire is a 10-item dermatology- specific health-related quality of life measure designed for use in children. Participants rated their dermatology symptoms as well as the impact of their skin condition on various aspects of their lives. The CDLQI total score is a sum of all 10 item responses, each individual response ranging from 0 (not at all) to 3 (very much). Total score ranges from 0 to 30 with higher scores indicating greater health-related quality of life impairment. A negative change score from baseline indicates improvement. Baseline was defined as the last non-missing value prior to or on the first treatment date. To handle the missing data, if a participant had only one item missing score per visit, then it was imputed to 0 and total score was calculated accordingly. If there were 2 or more item missing scores per visit, then the total score for the visit was considered as missing.

Outcome measures

Outcome measures
Measure	Ligelizumab 24 mg n=24 Participants Participants received a dose of ligelizumab 24 mg (low dose) which consisted of one injection of 0.2 ml of ligelizumab 120 mg/ 1 ml vial every 4 weeks from Day 1 to Week 20 (inclusive).	Ligelizumab 120 mg n=13 Participants Participants received a dose of ligelizumab 120 mg (high dose) which consisted of one injection of 1 ml of ligelizumab 120 mg/ 1 ml vial every 4 weeks from Day 1 to Week 20 (inclusive).	Placebo + Ligelizumab 120 mg n=12 Participants Participants received Placebo which consisted of one injection of 1 ml placebo every 4 weeks from Day 1 to Week 8 (inclusive). From week 12 to week 20 (inclusive), participants received a dose of ligelizumab 120 mg (high dose) which consisted of one injection of 1 ml of ligelizumab 120 mg/ 1 ml vial.
Change From Baseline of the Children Dermatology Life Quality Index (CDLQI) Week 24	-11.5 Score on a scale Standard Deviation 6.85	-8.8 Score on a scale Standard Deviation 10.43	-10.1 Score on a scale Standard Deviation 6.74
Change From Baseline of the Children Dermatology Life Quality Index (CDLQI) Week 12	-10.1 Score on a scale Standard Deviation 4.88	-6.6 Score on a scale Standard Deviation 8.05	-5.0 Score on a scale Standard Deviation 6.23
Change From Baseline of the Children Dermatology Life Quality Index (CDLQI) Week 40	-10.3 Score on a scale Standard Deviation 6.86	-5.5 Score on a scale Standard Deviation 9.04	-8.6 Score on a scale Standard Deviation 8.54

SECONDARY outcome

Timeframe: Baseline, weeks 12, 24 and 40

Population: All participants who received at least one dose of study treatment. Only those participants with data available for this endpoint at specified data points (weeks 12, 24 and 40) were analyzed.

Change from baseline in IgE (free IgE plus IgE bound to ligelizumab) at weeks 12, 24 and 40 as a pharmacodynamic measurement.

Outcome measures

Outcome measures
Measure	Ligelizumab 24 mg n=24 Participants Participants received a dose of ligelizumab 24 mg (low dose) which consisted of one injection of 0.2 ml of ligelizumab 120 mg/ 1 ml vial every 4 weeks from Day 1 to Week 20 (inclusive).	Ligelizumab 120 mg n=13 Participants Participants received a dose of ligelizumab 120 mg (high dose) which consisted of one injection of 1 ml of ligelizumab 120 mg/ 1 ml vial every 4 weeks from Day 1 to Week 20 (inclusive).	Placebo + Ligelizumab 120 mg n=12 Participants Participants received Placebo which consisted of one injection of 1 ml placebo every 4 weeks from Day 1 to Week 8 (inclusive). From week 12 to week 20 (inclusive), participants received a dose of ligelizumab 120 mg (high dose) which consisted of one injection of 1 ml of ligelizumab 120 mg/ 1 ml vial.
Change From Baseline in Total Human Immunoglobulin E (IgE) Week 12	186 International units / millilitre Standard Deviation 160	245 International units / millilitre Standard Deviation 291	-43.1 International units / millilitre Standard Deviation 56.2
Change From Baseline in Total Human Immunoglobulin E (IgE) Week 24	169 International units / millilitre Standard Deviation 126	328 International units / millilitre Standard Deviation 504	325 International units / millilitre Standard Deviation 411
Change From Baseline in Total Human Immunoglobulin E (IgE) Week 40	30.4 International units / millilitre Standard Deviation 105	-15.6 International units / millilitre Standard Deviation 118	-22.0 International units / millilitre Standard Deviation 93.0

SECONDARY outcome

Timeframe: Weeks 0 (baseline), 4, 8, 12, 16, 20 (all pre-dose) and weeks 24, 32 and 40

Population: All participants who received at least one dose of study treatment and provided an evaluable PK profile (at least 7 samples per patient collected). All ligelizumab concentration-time data were combined and included in the population PK analysis.

Model-based estimate of apparent clearance (CL/F) was derived using compartmental pharmacokinetic population (PopPK) modelling using non-linear mixed effects model for ligelizumab. Apparent clearance population estimate was derived through fitting individual drug administration history and collected ligelizumab concentrations at the specified data points (listed in Time Frame).

Outcome measures

Outcome measures
Measure	Ligelizumab 24 mg n=47 Participants Participants received a dose of ligelizumab 24 mg (low dose) which consisted of one injection of 0.2 ml of ligelizumab 120 mg/ 1 ml vial every 4 weeks from Day 1 to Week 20 (inclusive).	Ligelizumab 120 mg Participants received a dose of ligelizumab 120 mg (high dose) which consisted of one injection of 1 ml of ligelizumab 120 mg/ 1 ml vial every 4 weeks from Day 1 to Week 20 (inclusive).	Placebo + Ligelizumab 120 mg Participants received Placebo which consisted of one injection of 1 ml placebo every 4 weeks from Day 1 to Week 8 (inclusive). From week 12 to week 20 (inclusive), participants received a dose of ligelizumab 120 mg (high dose) which consisted of one injection of 1 ml of ligelizumab 120 mg/ 1 ml vial.
Apparent Clearance (CL/F) of Ligelizumab Estimated With a PopPK Model	0.66 Liters / day Interval 0.44 to 1.03	—	—

SECONDARY outcome

Timeframe: Weeks 0 (baseline), 4, 8, 12, 16, 20 (all pre-dose) and weeks 24, 32 and 40

Population: All participants who received at least one dose of study treatment and provided an evaluable PK profile (at least 7 samples per patient are required). All ligelizumab concentration-time data were combined and included in the population PK analysis.

Model-based estimate of apparent volume of distribution was derived using compartmental pharmacokinetic population (PopPK) modelling using non-linear mixed effects model for ligelizumab. Apparent volume of distribution population estimate was derived through fitting individual drug administration history and collected ligelizumab concentrations at the specified data points (listed in Time Frame).

Outcome measures

Outcome measures
Measure	Ligelizumab 24 mg n=47 Participants Participants received a dose of ligelizumab 24 mg (low dose) which consisted of one injection of 0.2 ml of ligelizumab 120 mg/ 1 ml vial every 4 weeks from Day 1 to Week 20 (inclusive).	Ligelizumab 120 mg Participants received a dose of ligelizumab 120 mg (high dose) which consisted of one injection of 1 ml of ligelizumab 120 mg/ 1 ml vial every 4 weeks from Day 1 to Week 20 (inclusive).	Placebo + Ligelizumab 120 mg Participants received Placebo which consisted of one injection of 1 ml placebo every 4 weeks from Day 1 to Week 8 (inclusive). From week 12 to week 20 (inclusive), participants received a dose of ligelizumab 120 mg (high dose) which consisted of one injection of 1 ml of ligelizumab 120 mg/ 1 ml vial.
Apparent Volume of Distribution of Ligelizumab Estimated With a PopPK Model	14.5 Liters Interval 11.02 to 16.65	—	—

SECONDARY outcome

Timeframe: From the start of treatment to 20 weeks after end of treatment, assessed up to maximum duration of 40 weeks

Population: All participants who received at least one dose of study treatment.

Number of participants with AEs and SAEs, including significant changes from baseline in vital signs (blood pressure, pulse rate), electrocardiograms and laboratory values qualifying and reported as AEs. The number of participants in each category is reported in the table.

Outcome measures

Outcome measures
Measure	Ligelizumab 24 mg n=24 Participants Participants received a dose of ligelizumab 24 mg (low dose) which consisted of one injection of 0.2 ml of ligelizumab 120 mg/ 1 ml vial every 4 weeks from Day 1 to Week 20 (inclusive).	Ligelizumab 120 mg n=13 Participants Participants received a dose of ligelizumab 120 mg (high dose) which consisted of one injection of 1 ml of ligelizumab 120 mg/ 1 ml vial every 4 weeks from Day 1 to Week 20 (inclusive).	Placebo + Ligelizumab 120 mg n=12 Participants Participants received Placebo which consisted of one injection of 1 ml placebo every 4 weeks from Day 1 to Week 8 (inclusive). From week 12 to week 20 (inclusive), participants received a dose of ligelizumab 120 mg (high dose) which consisted of one injection of 1 ml of ligelizumab 120 mg/ 1 ml vial.
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) SAEs	1 Participants	0 Participants	1 Participants
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) Treatment related AEs	6 Participants	5 Participants	2 Participants
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) AEs	18 Participants	11 Participants	9 Participants
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) SAEs leading to treatment discontinuation	0 Participants	0 Participants	1 Participants

Adverse Events

Ligelizumab 24 mg

Serious events: 1 serious events

Other events: 18 other events

Deaths: 0 deaths

Ligelizumab 120 mg

Serious events: 0 serious events

Other events: 11 other events

Deaths: 0 deaths

Placebo + Ligelizumab 120 mg

Serious events: 1 serious events

Other events: 9 other events

Deaths: 0 deaths

Total

Serious events: 2 serious events

Other events: 38 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Ligelizumab 24 mg n=24 participants at risk Participants received a dose of ligelizumab 24 mg (low dose) which consisted of one injection of 0.2 ml of ligelizumab 120 mg/ 1 ml vial every 4 weeks from Day 1 to Week 20 (inclusive).	Ligelizumab 120 mg n=13 participants at risk Participants received a dose of ligelizumab 120 mg (high dose) which consisted of one injection of 1 ml of ligelizumab 120 mg/ 1 ml vial every 4 weeks from Day 1 to Week 20 (inclusive).	Placebo + Ligelizumab 120 mg n=12 participants at risk Participants received Placebo which consisted of one injection of 1 ml placebo every 4 weeks from Day 1 to Week 8 (inclusive). From week 12 to week 20 (inclusive), participants received a dose of ligelizumab 120 mg (high dose) which consisted of one injection of 1 ml of ligelizumab 120 mg/ 1 ml vial.	Total n=49 participants at risk Total
Cardiac disorders Pulmonary valve incompetence	0.00% 0/24 • Adverse events were reported from the start treatment to 20 weeks after end of treatment, assessed up to maximum duration of 40 weeks.	0.00% 0/13 • Adverse events were reported from the start treatment to 20 weeks after end of treatment, assessed up to maximum duration of 40 weeks.	8.3% 1/12 • Adverse events were reported from the start treatment to 20 weeks after end of treatment, assessed up to maximum duration of 40 weeks.	2.0% 1/49 • Adverse events were reported from the start treatment to 20 weeks after end of treatment, assessed up to maximum duration of 40 weeks.
Cardiac disorders Tricuspid valve incompetence	0.00% 0/24 • Adverse events were reported from the start treatment to 20 weeks after end of treatment, assessed up to maximum duration of 40 weeks.	0.00% 0/13 • Adverse events were reported from the start treatment to 20 weeks after end of treatment, assessed up to maximum duration of 40 weeks.	8.3% 1/12 • Adverse events were reported from the start treatment to 20 weeks after end of treatment, assessed up to maximum duration of 40 weeks.	2.0% 1/49 • Adverse events were reported from the start treatment to 20 weeks after end of treatment, assessed up to maximum duration of 40 weeks.
Psychiatric disorders Suicide attempt	4.2% 1/24 • Adverse events were reported from the start treatment to 20 weeks after end of treatment, assessed up to maximum duration of 40 weeks.	0.00% 0/13 • Adverse events were reported from the start treatment to 20 weeks after end of treatment, assessed up to maximum duration of 40 weeks.	0.00% 0/12 • Adverse events were reported from the start treatment to 20 weeks after end of treatment, assessed up to maximum duration of 40 weeks.	2.0% 1/49 • Adverse events were reported from the start treatment to 20 weeks after end of treatment, assessed up to maximum duration of 40 weeks.

Other adverse events

Additional Information

Study Director

Novartis Pharmaceuticals

Phone: + 1 862 778 8300

Email: [email protected]

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.
Publication restrictions are in place

Restriction type: OTHER