Trial Outcomes & Findings for An Open-label Extension Study to Evaluate the Long-term Safety and Tolerability of LOU064 in Subjects With CSU (NCT NCT04109313)

Results Overview

An AE refers to any undesirable medical occurrence, such as an unintended sign (including abnormal laboratory findings), symptom, or disease, experienced by a participant. Serious AEs (SAEs) is defined as any AE that results in death, is life-threatening, requires hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a congenital anomaly/birth defect, or any other medically significant condition. Treatment-emergent AEs were defined as AEs that either begin on the same day or after the first dose of study medication during the treatment period in the extension study or worsen on the same day or after the first dose of study medication in the extension study and within the minimum of either 28 days post last dose or the end of the study visit. The number of participants with treatment-emergent AEs was summarized.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

229 participants

Primary outcome timeframe

From first dose of treatment up to 28 days after last dose, assessed up to 56 weeks

Results posted on

2024-06-20

Participant Flow

229 subjects were enrolled in the observational period or the treatment period across 72 sites in 15 countries. One subject was a screening failure and, as a result, was not enrolled in either the observational or treatment period.

Participants with UAS7\<16 at Week 16 of CLOU064A2201 (NCT03926611) entered a 12-week observational period. Participants with UAS7≥16 at Week 12 or Week 16 of CLOU064A2201, as well as those who relapsed during the observational period, entered the treatment period.

Participant milestones

Participant milestones
Measure	All Participants Participants with UAS7\<16 at Week 16 of CLOU064A2201 were followed up to 12 weeks without receiving treatment (observational period). If participants relapsed (UAS7≥16 at least once), they were transitioned to the treatment period. Participants with a UAS7≥16 at Week 12 or Week 16 in the CLOU064A2201, as well as participants who experienced a relapse during the 12-week observational period, were administered 100 mg of LOU064 b.i.d. open-label for up to 52 weeks.
Overall Study STARTED	229
Overall Study Treatment-free Cohort	68
Overall Study Treatment Cohort	194
Overall Study COMPLETED	188
Overall Study NOT COMPLETED	41

Reasons for withdrawal

Reasons for withdrawal
Measure	All Participants Participants with UAS7\<16 at Week 16 of CLOU064A2201 were followed up to 12 weeks without receiving treatment (observational period). If participants relapsed (UAS7≥16 at least once), they were transitioned to the treatment period. Participants with a UAS7≥16 at Week 12 or Week 16 in the CLOU064A2201, as well as participants who experienced a relapse during the 12-week observational period, were administered 100 mg of LOU064 b.i.d. open-label for up to 52 weeks.
Overall Study Pregnancy	1
Overall Study Adverse Event	11
Overall Study Lack of Efficacy	11
Overall Study Lost to Follow-up	1
Overall Study Physician Decision	2
Overall Study Subject Decision	13
Overall Study Covid-19 situation	2

Baseline Characteristics

An Open-label Extension Study to Evaluate the Long-term Safety and Tolerability of LOU064 in Subjects With CSU

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	All Participants n=229 Participants Participants with UAS7\<16 at Week 16 of CLOU064A2201 were followed up to 12 weeks without receiving treatment (observational period). If participants relapsed (UAS7≥16 at least once), they were transitioned to the treatment period. Participants with a UAS7≥16 at Week 12 or Week 16 in the CLOU064A2201, as well as participants who experienced a relapse during the 12-week observational period, were administered 100 mg of LOU064 b.i.d. open-label for up to 52 weeks.
Age, Continuous	45 Years n=5 Participants
Sex: Female, Male Female	165 Participants n=5 Participants
Sex: Female, Male Male	64 Participants n=5 Participants
Race/Ethnicity, Customized White	181 Participants n=5 Participants
Race/Ethnicity, Customized Black	2 Participants n=5 Participants
Race/Ethnicity, Customized Asian	44 Participants n=5 Participants
Race/Ethnicity, Customized Multiple	1 Participants n=5 Participants
Race/Ethnicity, Customized American Indian or Alaska Native	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: From first dose of treatment up to 28 days after last dose, assessed up to 56 weeks

Population: All subjects who received at least one dose of study treatment during the treatment period of this extension study.

An AE refers to any undesirable medical occurrence, such as an unintended sign (including abnormal laboratory findings), symptom, or disease, experienced by a participant. Serious AEs (SAEs) is defined as any AE that results in death, is life-threatening, requires hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a congenital anomaly/birth defect, or any other medically significant condition. Treatment-emergent AEs were defined as AEs that either begin on the same day or after the first dose of study medication during the treatment period in the extension study or worsen on the same day or after the first dose of study medication in the extension study and within the minimum of either 28 days post last dose or the end of the study visit. The number of participants with treatment-emergent AEs was summarized.

Outcome measures

Outcome measures
Measure	Treated Cohort n=194 Participants Participants with a UAS7≥16 at Week 12 or Week 16 in the CLOU064A2201, as well as participants who experienced a relapse during the 12-week observational period, were administered 100 mg of LOU064 b.i.d. open-label for up to 52 weeks.
Number of Participants With Treatment-emergent Adverse Events (AEs) Discontinued treatment due to any SAE(s)	2 Participants
Number of Participants With Treatment-emergent Adverse Events (AEs) Treatment interruption due to SAE(s)	1 Participants
Number of Participants With Treatment-emergent Adverse Events (AEs) AEs	139 Participants
Number of Participants With Treatment-emergent Adverse Events (AEs) Deaths	0 Participants
Number of Participants With Treatment-emergent Adverse Events (AEs) Non-fatal Serious AEs (SAEs)	6 Participants
Number of Participants With Treatment-emergent Adverse Events (AEs) SAE(s)	6 Participants
Number of Participants With Treatment-emergent Adverse Events (AEs) Discontinued treatment due to any AE(s)	11 Participants
Number of Participants With Treatment-emergent Adverse Events (AEs) Treatment interruption due to AE(s)	13 Participants

SECONDARY outcome

Timeframe: Baseline, Week 4 of treatment period

Population: All subjects who received at least one dose of study treatment during the treatment period of this extension study with a value at both baseline and Week 4 of the treatment period.

The Urticaria Activity Score (UAS) is a composite, diary-recorded score with numeric severity intensity ratings (0=none to 3=intense/severe) for the number of wheals (hives) and the intensity of the pruritus (itch) over the past 12 hours (twice daily). The daily UAS is calculated as the average of the morning and evening scores. The UAS7 is the weekly sum of the daily UAS, which is the composite score of the intensity of pruritus and the number of wheals. UAS7 scores ranged from 0 to 42. A higher UAS7 indicated greater urticaria disease activity. A minimum of 4 out of 7 daily scores were needed to calculate the UAS7 values. Otherwise, the weekly score was missing for that week. The change from baseline in UAS7 at Week 4 of the treatment period was calculated. A negative change score from baseline indicates improvement. The UAS7 at baseline was considered as the UAS7 derived over the last 7 days before day 1 of the treatment period.

Outcome measures

Outcome measures
Measure	Treated Cohort n=187 Participants Participants with a UAS7≥16 at Week 12 or Week 16 in the CLOU064A2201, as well as participants who experienced a relapse during the 12-week observational period, were administered 100 mg of LOU064 b.i.d. open-label for up to 52 weeks.
Change From Baseline in Weekly Urticaria Activity Score (UAS7) at Week 4 of the Treatment Period	-17.58 Score on a Scale Standard Deviation 13.400

SECONDARY outcome

Timeframe: Week 4 of the treatment period

Population: All subjects who received at least one dose of study treatment during the treatment period of this extension study.

The Urticaria Activity Score (UAS) is a composite, diary-recorded score with numeric severity intensity ratings (0=none to 3=intense/severe) for the number of wheals (hives) and the intensity of the pruritus (itch) over the past 12 hours (twice daily). The daily UAS is calculated as the average of the morning and evening scores. The UAS7 is the weekly sum of the daily UAS, which is the composite score of the intensity of pruritus and the number of wheals. UAS7 scores ranged from 0 to 42. A higher UAS7 indicated greater urticaria disease activity. A minimum of 4 out of 7 daily scores were needed to calculate the UAS7 values. Otherwise, the weekly score was missing for that week. Missing values were imputed by non-responder imputation method regardless of the reason for missingness. The percentage of subjects with UAS7≤ 6 at Week 4 of the treatment period was calculated. The 90% confidence interval was derived based on the score method with continuity correction.

Outcome measures

Outcome measures
Measure	Treated Cohort n=194 Participants Participants with a UAS7≥16 at Week 12 or Week 16 in the CLOU064A2201, as well as participants who experienced a relapse during the 12-week observational period, were administered 100 mg of LOU064 b.i.d. open-label for up to 52 weeks.
Percentage of Participants With Well-controlled Disease (UAS7≤6) at Week 4 of the Treatment Period	51.0 Percentage of participants Interval 44.9 to 57.1

SECONDARY outcome

Timeframe: Week 4 of the treatment period

Population: All subjects who received at least one dose of study treatment during the treatment period of this extension study.

The Urticaria Activity Score (UAS) is a composite, diary-recorded score with numeric severity intensity ratings (0=none to 3=intense/severe) for the number of wheals (hives) and the intensity of the pruritus (itch) over the past 12 hours (twice daily). The daily UAS is calculated as the average of the morning and evening scores. The UAS7 is the weekly sum of the daily UAS, which is the composite score of the intensity of pruritus and the number of wheals. UAS7 scores ranged from 0 to 42. A higher UAS7 indicated greater urticaria disease activity. A minimum of 4 out of 7 daily scores were needed to calculate the UAS7 values. Otherwise, the weekly score was missing for that week. Missing values were imputed by non-responder imputation method regardless of the reason for missingness. The percentage of subjects with UAS7= 0 at Week 4 of the treatment period was calculated. The 90% confidence interval was derived based on the score method with continuity correction.

Outcome measures

Outcome measures
Measure	Treated Cohort n=194 Participants Participants with a UAS7≥16 at Week 12 or Week 16 in the CLOU064A2201, as well as participants who experienced a relapse during the 12-week observational period, were administered 100 mg of LOU064 b.i.d. open-label for up to 52 weeks.
Percentage of Participants With Complete Response (UAS7=0) at Week 4 of the Treatment Period	27.3 Percentage of participants Interval 22.2 to 33.1

SECONDARY outcome

Timeframe: From baseline until Week 52 of the treatment period

Population: All subjects who received at least one dose of study treatment during the treatment period of this extension study. Number analyzed refers to the number of participants with an evaluable value at the specified time points.

The Urticaria Activity Score (UAS) is a composite, diary-recorded score with numeric severity intensity ratings (0=none to 3=intense/severe) for the number of wheals (hives) and the intensity of the pruritus (itch) over the past 12 hours (twice daily). The daily UAS is calculated as the average of the morning and evening scores. The UAS7 is the weekly sum of the daily UAS, which is the composite score of the intensity of pruritus and the number of wheals. UAS7 scores ranged from 0 to 42. A higher UAS7 indicated greater urticaria disease activity. A minimum of 4 out of 7 daily scores were needed to calculate the UAS7 values. Otherwise, the weekly score was missing for that week. The percentage of subjects with UAS7≤ 6 during the treatment period was calculated. The 90% confidence interval was derived based on the score method with continuity correction. The UAS7 at baseline was considered as the UAS7 derived over the last 7 days before day 1 of the treatment period.

Outcome measures

Outcome measures
Measure	Treated Cohort n=194 Participants Participants with a UAS7≥16 at Week 12 or Week 16 in the CLOU064A2201, as well as participants who experienced a relapse during the 12-week observational period, were administered 100 mg of LOU064 b.i.d. open-label for up to 52 weeks.
Percentage of Participants With Well-controlled Disease (UAS7≤ 6) Overtime Baseline	1.0 Percentage of participants Interval 0.2 to 3.5
Percentage of Participants With Well-controlled Disease (UAS7≤ 6) Overtime Week 1	37.4 Percentage of participants Interval 31.4 to 43.8
Percentage of Participants With Well-controlled Disease (UAS7≤ 6) Overtime Week 4	52.7 Percentage of participants Interval 46.4 to 58.8
Percentage of Participants With Well-controlled Disease (UAS7≤ 6) Overtime Week 12	56.6 Percentage of participants Interval 50.1 to 63.0
Percentage of Participants With Well-controlled Disease (UAS7≤ 6) Overtime Week 20	62.7 Percentage of participants Interval 56.1 to 68.9
Percentage of Participants With Well-controlled Disease (UAS7≤ 6) Overtime Week 28	68.5 Percentage of participants Interval 61.9 to 74.5
Percentage of Participants With Well-controlled Disease (UAS7≤ 6) Overtime Week 40	66.5 Percentage of participants Interval 59.6 to 72.7
Percentage of Participants With Well-controlled Disease (UAS7≤ 6) Overtime Week 52	68.0 Percentage of participants Interval 61.1 to 74.3

SECONDARY outcome

Timeframe: From baseline until Week 52 of the treatment period

Population: Subjects who received at least one dose of study treatment during the treatment period of this extension study and did not have missing values at the specified time points. Number analyzed refers to the number of participants with an evaluable value at the specified time points.

The Urticaria Activity Score (UAS) is a composite, diary-recorded score with numeric severity intensity ratings (0=none to 3=intense/severe) for the number of wheals (hives) and the intensity of the pruritus (itch) over the past 12 hours (twice daily). The daily UAS is calculated as the average of the morning and evening scores. The UAS7 is the weekly sum of the daily UAS, which is the composite score of the intensity of pruritus and the number of wheals. UAS7 scores ranged from 0 to 42. A higher UAS7 indicated greater urticaria disease activity. A minimum of 4 out of 7 daily scores were needed to calculate the UAS7 values. Otherwise, the weekly score was missing for that week. The change from baseline in UAS7 during the treatment period was calculated. A negative change score from baseline indicates improvement. The UAS7 at baseline was considered as the UAS7 derived over the last 7 days before day 1 of the treatment period.

Outcome measures

Outcome measures
Measure	Treated Cohort n=193 Participants Participants with a UAS7≥16 at Week 12 or Week 16 in the CLOU064A2201, as well as participants who experienced a relapse during the 12-week observational period, were administered 100 mg of LOU064 b.i.d. open-label for up to 52 weeks.
Change From Baseline in UAS7 Overtime Week 1	-14.76 Score on a Scale Standard Deviation 11.502
Change From Baseline in UAS7 Overtime Week 4	-17.58 Score on a Scale Standard Deviation 13.400
Change From Baseline in UAS7 Overtime Week 12	-19.37 Score on a Scale Standard Deviation 12.502
Change From Baseline in UAS7 Overtime Week 20	-20.61 Score on a Scale Standard Deviation 11.634
Change From Baseline in UAS7 Overtime Week 28	-21.54 Score on a Scale Standard Deviation 11.525
Change From Baseline in UAS7 Overtime Week 40	-21.25 Score on a Scale Standard Deviation 11.400
Change From Baseline in UAS7 Overtime Week 52	-21.82 Score on a Scale Standard Deviation 10.699

Adverse Events

Treatment-free Cohort (Observational Period)

Serious events: 1 serious events

Other events: 0 other events

Deaths: 0 deaths

Treated Cohort (Treatment+Follow-up Period)

Serious events: 6 serious events

Other events: 56 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Treatment-free Cohort (Observational Period) n=68 participants at risk Participants with UAS7\<16 at Week 16 of CLOU064A2201 were followed up to 12 weeks without receiving treatment (observational period). If participants relapsed (UAS7≥16 at least once), they were transitioned to the treatment period (treated cohort).	Treated Cohort (Treatment+Follow-up Period) n=194 participants at risk Participants with a UAS7≥16 at Week 12 or Week 16 in the CLOU064A2201, as well as participants who experienced a relapse during the 12-week observational period, were administered 100 mg of LOU064 b.i.d. open-label for up to 52 weeks.
Gastrointestinal disorders Melaena	0.00% 0/68 • In the Treated cohort, treatment-emergent AEs were collected from first dose of treatment up to 28 days post last dose, up to 56 weeks; and deaths were collected from first dose of treatment until end of study, assessed up to 68 weeks. In the Treatment-free cohort, AEs and deaths were collected from start of observation period until relapse (UAS7≥16) or end of observation period, up to 12 weeks. An AE refers to any undesirable medical occurrence, such as an unintended sign (including abnormal laboratory findings), symptom, or disease, experienced by a participant.	0.52% 1/194 • In the Treated cohort, treatment-emergent AEs were collected from first dose of treatment up to 28 days post last dose, up to 56 weeks; and deaths were collected from first dose of treatment until end of study, assessed up to 68 weeks. In the Treatment-free cohort, AEs and deaths were collected from start of observation period until relapse (UAS7≥16) or end of observation period, up to 12 weeks. An AE refers to any undesirable medical occurrence, such as an unintended sign (including abnormal laboratory findings), symptom, or disease, experienced by a participant.
General disorders Chest pain	0.00% 0/68 • In the Treated cohort, treatment-emergent AEs were collected from first dose of treatment up to 28 days post last dose, up to 56 weeks; and deaths were collected from first dose of treatment until end of study, assessed up to 68 weeks. In the Treatment-free cohort, AEs and deaths were collected from start of observation period until relapse (UAS7≥16) or end of observation period, up to 12 weeks. An AE refers to any undesirable medical occurrence, such as an unintended sign (including abnormal laboratory findings), symptom, or disease, experienced by a participant.	0.52% 1/194 • In the Treated cohort, treatment-emergent AEs were collected from first dose of treatment up to 28 days post last dose, up to 56 weeks; and deaths were collected from first dose of treatment until end of study, assessed up to 68 weeks. In the Treatment-free cohort, AEs and deaths were collected from start of observation period until relapse (UAS7≥16) or end of observation period, up to 12 weeks. An AE refers to any undesirable medical occurrence, such as an unintended sign (including abnormal laboratory findings), symptom, or disease, experienced by a participant.
Infections and infestations Appendicitis	0.00% 0/68 • In the Treated cohort, treatment-emergent AEs were collected from first dose of treatment up to 28 days post last dose, up to 56 weeks; and deaths were collected from first dose of treatment until end of study, assessed up to 68 weeks. In the Treatment-free cohort, AEs and deaths were collected from start of observation period until relapse (UAS7≥16) or end of observation period, up to 12 weeks. An AE refers to any undesirable medical occurrence, such as an unintended sign (including abnormal laboratory findings), symptom, or disease, experienced by a participant.	0.52% 1/194 • In the Treated cohort, treatment-emergent AEs were collected from first dose of treatment up to 28 days post last dose, up to 56 weeks; and deaths were collected from first dose of treatment until end of study, assessed up to 68 weeks. In the Treatment-free cohort, AEs and deaths were collected from start of observation period until relapse (UAS7≥16) or end of observation period, up to 12 weeks. An AE refers to any undesirable medical occurrence, such as an unintended sign (including abnormal laboratory findings), symptom, or disease, experienced by a participant.
Infections and infestations COVID-19 pneumonia	0.00% 0/68 • In the Treated cohort, treatment-emergent AEs were collected from first dose of treatment up to 28 days post last dose, up to 56 weeks; and deaths were collected from first dose of treatment until end of study, assessed up to 68 weeks. In the Treatment-free cohort, AEs and deaths were collected from start of observation period until relapse (UAS7≥16) or end of observation period, up to 12 weeks. An AE refers to any undesirable medical occurrence, such as an unintended sign (including abnormal laboratory findings), symptom, or disease, experienced by a participant.	1.0% 2/194 • In the Treated cohort, treatment-emergent AEs were collected from first dose of treatment up to 28 days post last dose, up to 56 weeks; and deaths were collected from first dose of treatment until end of study, assessed up to 68 weeks. In the Treatment-free cohort, AEs and deaths were collected from start of observation period until relapse (UAS7≥16) or end of observation period, up to 12 weeks. An AE refers to any undesirable medical occurrence, such as an unintended sign (including abnormal laboratory findings), symptom, or disease, experienced by a participant.
Injury, poisoning and procedural complications Tibia fracture	0.00% 0/68 • In the Treated cohort, treatment-emergent AEs were collected from first dose of treatment up to 28 days post last dose, up to 56 weeks; and deaths were collected from first dose of treatment until end of study, assessed up to 68 weeks. In the Treatment-free cohort, AEs and deaths were collected from start of observation period until relapse (UAS7≥16) or end of observation period, up to 12 weeks. An AE refers to any undesirable medical occurrence, such as an unintended sign (including abnormal laboratory findings), symptom, or disease, experienced by a participant.	0.52% 1/194 • In the Treated cohort, treatment-emergent AEs were collected from first dose of treatment up to 28 days post last dose, up to 56 weeks; and deaths were collected from first dose of treatment until end of study, assessed up to 68 weeks. In the Treatment-free cohort, AEs and deaths were collected from start of observation period until relapse (UAS7≥16) or end of observation period, up to 12 weeks. An AE refers to any undesirable medical occurrence, such as an unintended sign (including abnormal laboratory findings), symptom, or disease, experienced by a participant.
Reproductive system and breast disorders Ovarian cyst	0.00% 0/68 • In the Treated cohort, treatment-emergent AEs were collected from first dose of treatment up to 28 days post last dose, up to 56 weeks; and deaths were collected from first dose of treatment until end of study, assessed up to 68 weeks. In the Treatment-free cohort, AEs and deaths were collected from start of observation period until relapse (UAS7≥16) or end of observation period, up to 12 weeks. An AE refers to any undesirable medical occurrence, such as an unintended sign (including abnormal laboratory findings), symptom, or disease, experienced by a participant.	0.52% 1/194 • In the Treated cohort, treatment-emergent AEs were collected from first dose of treatment up to 28 days post last dose, up to 56 weeks; and deaths were collected from first dose of treatment until end of study, assessed up to 68 weeks. In the Treatment-free cohort, AEs and deaths were collected from start of observation period until relapse (UAS7≥16) or end of observation period, up to 12 weeks. An AE refers to any undesirable medical occurrence, such as an unintended sign (including abnormal laboratory findings), symptom, or disease, experienced by a participant.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Superficial spreading melanoma stage unspecified	1.5% 1/68 • In the Treated cohort, treatment-emergent AEs were collected from first dose of treatment up to 28 days post last dose, up to 56 weeks; and deaths were collected from first dose of treatment until end of study, assessed up to 68 weeks. In the Treatment-free cohort, AEs and deaths were collected from start of observation period until relapse (UAS7≥16) or end of observation period, up to 12 weeks. An AE refers to any undesirable medical occurrence, such as an unintended sign (including abnormal laboratory findings), symptom, or disease, experienced by a participant.	0.00% 0/194 • In the Treated cohort, treatment-emergent AEs were collected from first dose of treatment up to 28 days post last dose, up to 56 weeks; and deaths were collected from first dose of treatment until end of study, assessed up to 68 weeks. In the Treatment-free cohort, AEs and deaths were collected from start of observation period until relapse (UAS7≥16) or end of observation period, up to 12 weeks. An AE refers to any undesirable medical occurrence, such as an unintended sign (including abnormal laboratory findings), symptom, or disease, experienced by a participant.

Other adverse events

Other adverse events
Measure	Treatment-free Cohort (Observational Period) n=68 participants at risk Participants with UAS7\<16 at Week 16 of CLOU064A2201 were followed up to 12 weeks without receiving treatment (observational period). If participants relapsed (UAS7≥16 at least once), they were transitioned to the treatment period (treated cohort).	Treated Cohort (Treatment+Follow-up Period) n=194 participants at risk Participants with a UAS7≥16 at Week 12 or Week 16 in the CLOU064A2201, as well as participants who experienced a relapse during the 12-week observational period, were administered 100 mg of LOU064 b.i.d. open-label for up to 52 weeks.
Infections and infestations COVID-19	0.00% 0/68 • In the Treated cohort, treatment-emergent AEs were collected from first dose of treatment up to 28 days post last dose, up to 56 weeks; and deaths were collected from first dose of treatment until end of study, assessed up to 68 weeks. In the Treatment-free cohort, AEs and deaths were collected from start of observation period until relapse (UAS7≥16) or end of observation period, up to 12 weeks. An AE refers to any undesirable medical occurrence, such as an unintended sign (including abnormal laboratory findings), symptom, or disease, experienced by a participant.	8.2% 16/194 • In the Treated cohort, treatment-emergent AEs were collected from first dose of treatment up to 28 days post last dose, up to 56 weeks; and deaths were collected from first dose of treatment until end of study, assessed up to 68 weeks. In the Treatment-free cohort, AEs and deaths were collected from start of observation period until relapse (UAS7≥16) or end of observation period, up to 12 weeks. An AE refers to any undesirable medical occurrence, such as an unintended sign (including abnormal laboratory findings), symptom, or disease, experienced by a participant.
Nervous system disorders Headache	0.00% 0/68 • In the Treated cohort, treatment-emergent AEs were collected from first dose of treatment up to 28 days post last dose, up to 56 weeks; and deaths were collected from first dose of treatment until end of study, assessed up to 68 weeks. In the Treatment-free cohort, AEs and deaths were collected from start of observation period until relapse (UAS7≥16) or end of observation period, up to 12 weeks. An AE refers to any undesirable medical occurrence, such as an unintended sign (including abnormal laboratory findings), symptom, or disease, experienced by a participant.	6.7% 13/194 • In the Treated cohort, treatment-emergent AEs were collected from first dose of treatment up to 28 days post last dose, up to 56 weeks; and deaths were collected from first dose of treatment until end of study, assessed up to 68 weeks. In the Treatment-free cohort, AEs and deaths were collected from start of observation period until relapse (UAS7≥16) or end of observation period, up to 12 weeks. An AE refers to any undesirable medical occurrence, such as an unintended sign (including abnormal laboratory findings), symptom, or disease, experienced by a participant.
Skin and subcutaneous tissue disorders Chronic spontaneous urticaria	0.00% 0/68 • In the Treated cohort, treatment-emergent AEs were collected from first dose of treatment up to 28 days post last dose, up to 56 weeks; and deaths were collected from first dose of treatment until end of study, assessed up to 68 weeks. In the Treatment-free cohort, AEs and deaths were collected from start of observation period until relapse (UAS7≥16) or end of observation period, up to 12 weeks. An AE refers to any undesirable medical occurrence, such as an unintended sign (including abnormal laboratory findings), symptom, or disease, experienced by a participant.	11.3% 22/194 • In the Treated cohort, treatment-emergent AEs were collected from first dose of treatment up to 28 days post last dose, up to 56 weeks; and deaths were collected from first dose of treatment until end of study, assessed up to 68 weeks. In the Treatment-free cohort, AEs and deaths were collected from start of observation period until relapse (UAS7≥16) or end of observation period, up to 12 weeks. An AE refers to any undesirable medical occurrence, such as an unintended sign (including abnormal laboratory findings), symptom, or disease, experienced by a participant.
Skin and subcutaneous tissue disorders Eczema	0.00% 0/68 • In the Treated cohort, treatment-emergent AEs were collected from first dose of treatment up to 28 days post last dose, up to 56 weeks; and deaths were collected from first dose of treatment until end of study, assessed up to 68 weeks. In the Treatment-free cohort, AEs and deaths were collected from start of observation period until relapse (UAS7≥16) or end of observation period, up to 12 weeks. An AE refers to any undesirable medical occurrence, such as an unintended sign (including abnormal laboratory findings), symptom, or disease, experienced by a participant.	5.2% 10/194 • In the Treated cohort, treatment-emergent AEs were collected from first dose of treatment up to 28 days post last dose, up to 56 weeks; and deaths were collected from first dose of treatment until end of study, assessed up to 68 weeks. In the Treatment-free cohort, AEs and deaths were collected from start of observation period until relapse (UAS7≥16) or end of observation period, up to 12 weeks. An AE refers to any undesirable medical occurrence, such as an unintended sign (including abnormal laboratory findings), symptom, or disease, experienced by a participant.

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