Trial Outcomes & Findings for A Safety Extension Study to Evaluate the Long-term Safety of QGE031 in Chronic Spontaneous Urticaria (CSU) Patients (NCT NCT02649218)
NCT ID: NCT02649218
Last Updated: 2021-10-11
Results Overview
The primary objective of this study was to assess the long-term safety of one-year treatment of QGE031 in adult Chronic Spontaneous Urticaria (CSU) patients who completed the core study CQGE031C2201 using the following evaluations: number of participants with treatment emergent AEs of non-serious and serious nature including any events of special interest.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
226 participants
Primary outcome timeframe
Within 16 weeks after Week 48
Results posted on
2021-10-11
Participant Flow
Of the subjects who completed core study (NCT02477332) that were eligible for extension study, 237 were screened; 226 were enrolled to open-label treatment epoch; 201 (88.9%) completed treatment epoch; 209 (92.5%) entered the post-treatment follow-up epoch and 152 (67.3%) completed the post-treatment follow-up epoch
Participant milestones
| Measure |
Ligelizumab
QGE031 240 mg s.c. q4w x 13 treatments
|
|---|---|
|
Overall Study
STARTED
|
226
|
|
Overall Study
COMPLETED
|
201
|
|
Overall Study
NOT COMPLETED
|
25
|
Reasons for withdrawal
| Measure |
Ligelizumab
QGE031 240 mg s.c. q4w x 13 treatments
|
|---|---|
|
Overall Study
Adverse Event
|
8
|
|
Overall Study
Lack of Efficacy
|
8
|
|
Overall Study
Pregnancy
|
3
|
|
Overall Study
Protocol Violation
|
3
|
|
Overall Study
Physician Decision
|
1
|
|
Overall Study
Withdrawal by Subject
|
2
|
Baseline Characteristics
The Safety set (SS) included all subjects who received at least one dose of study drug during this open-label study.
Baseline characteristics by cohort
| Measure |
Ligelizumab
n=226 Participants
QGE031 240 mg s.c. q4w x 13 treatments
|
|---|---|
|
Age, Continuous
|
44.5 years
STANDARD_DEVIATION 12.69 • n=226 Participants
|
|
Age, Customized
<65 years
|
211 participants
n=226 Participants
|
|
Age, Customized
>= 65 years
|
15 participants
n=226 Participants
|
|
Sex/Gender, Customized
Female
|
75.2 Percentage of Participants
n=170 Participants • The Safety set (SS) included all subjects who received at least one dose of study drug during this open-label study.
|
|
Sex/Gender, Customized
Male
|
24.8 Percentage of Participants
n=56 Participants • The Safety set (SS) included all subjects who received at least one dose of study drug during this open-label study.
|
|
Race/Ethnicity, Customized
Asian
|
22.6 Percentage of participants
n=51 Participants • The Safety set (SS) included all subjects who received at least one dose of study drug during this open-label study.
|
|
Race/Ethnicity, Customized
Black or African American
|
1.3 Percentage of participants
n=3 Participants • The Safety set (SS) included all subjects who received at least one dose of study drug during this open-label study.
|
|
Race/Ethnicity, Customized
White
|
72.1 Percentage of participants
n=163 Participants • The Safety set (SS) included all subjects who received at least one dose of study drug during this open-label study.
|
|
Race/Ethnicity, Customized
American Indian or Alaska Native
|
0.4 Percentage of participants
n=1 Participants • The Safety set (SS) included all subjects who received at least one dose of study drug during this open-label study.
|
|
Race/Ethnicity, Customized
Unknown
|
0.9 Percentage of participants
n=2 Participants • The Safety set (SS) included all subjects who received at least one dose of study drug during this open-label study.
|
|
Race/Ethnicity, Customized
Other
|
2.7 Percentage of participants
n=6 Participants • The Safety set (SS) included all subjects who received at least one dose of study drug during this open-label study.
|
PRIMARY outcome
Timeframe: Within 16 weeks after Week 48Population: Safety Set: All 226 subjects who received at least one dose of study drug during this open-label study were included.
The primary objective of this study was to assess the long-term safety of one-year treatment of QGE031 in adult Chronic Spontaneous Urticaria (CSU) patients who completed the core study CQGE031C2201 using the following evaluations: number of participants with treatment emergent AEs of non-serious and serious nature including any events of special interest.
Outcome measures
| Measure |
Ligelizumab
n=226 Participants
QGE031 240 mg s.c. q4w x 13 treatments
|
|---|---|
|
Number of Participants With at Least One Treatment Emergent Adverse Event (AE)
|
190 Participants
|
SECONDARY outcome
Timeframe: Baseline, Week 52 and Week 100Population: Safety Set
The secondary objective of this study was to assess the long-term efficacy of QGE031 in adult CSU patients who completed the CQGE031C2201 study using the following evaluations: Sustained remission defined as maintaining (Urticaria Activity Score) UAS7 ≤ 6 over 48 weeks post-treatment follow up epoch among the participants achieving remission at the end of treatment epoch.
Outcome measures
| Measure |
Ligelizumab
n=226 Participants
QGE031 240 mg s.c. q4w x 13 treatments
|
|---|---|
|
Percentage of Subjects Having Achieved UAS7 ≤ 6
Baseline
|
0.44 percentage of participants
Interval 0.0 to 2.4
|
|
Percentage of Subjects Having Achieved UAS7 ≤ 6
Week 52
|
61.06 percentage of participants
Interval 54.4 to 67.5
|
|
Percentage of Subjects Having Achieved UAS7 ≤ 6
Week 100
|
28.32 percentage of participants
Interval 22.5 to 34.7
|
SECONDARY outcome
Timeframe: Baseline, Week 52, Week 100Population: Safety Set
Summary of subjects with UAS7 ≤ 6. The long term efficacy of one-year treatment of ligelizumab 240 mg s.c. q4w is assessed by number and proportion of participants who achieved well controlled disease (UAS7≤ 6) at end of the treatment period (Week 52) and end of follow up period (Week 100).
Outcome measures
| Measure |
Ligelizumab
n=226 Participants
QGE031 240 mg s.c. q4w x 13 treatments
|
|---|---|
|
Number and Proportion of Participants Who Achieved UAS7≤ 6
Week 52
|
138 Participants
|
|
Number and Proportion of Participants Who Achieved UAS7≤ 6
Week 100
|
64 Participants
|
|
Number and Proportion of Participants Who Achieved UAS7≤ 6
Baseline
|
1 Participants
|
Adverse Events
QGE031 240 mg q4w (TEAE)
Serious events: 15 serious events
Other events: 133 other events
Deaths: 1 deaths
QGE031 240 mg q4w (Non-TEAE)
Serious events: 6 serious events
Other events: 38 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
QGE031 240 mg q4w (TEAE)
n=226 participants at risk
QGE031 240 mg every four weeks (TEAE)
|
QGE031 240 mg q4w (Non-TEAE)
n=226 participants at risk
QGE031 240 mg every four weeks (non-TEAE)
|
|---|---|---|
|
Cardiac disorders
Angina pectoris
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Cardiac disorders
Supraventricular tachycardia
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Ear and labyrinth disorders
Vertigo
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Gastrointestinal disorders
Gastritis
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Gastrointestinal disorders
Haemorrhoids
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Gastrointestinal disorders
Mouth cyst
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
General disorders
Non-cardiac chest pain
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Hepatobiliary disorders
Cholecystitis
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Hepatobiliary disorders
Cholecystitis chronic
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Immune system disorders
Hypersensitivity
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Infections and infestations
Complicated appendicitis
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Infections and infestations
Localised infection
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Injury, poisoning and procedural complications
Tendon rupture
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Investigations
Blood pressure increased
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Metabolism and nutrition disorders
Dehydration
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Metabolism and nutrition disorders
Hypocalcaemia
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Cervix carcinoma stage 0
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Pancreatic neoplasm
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Nervous system disorders
Headache
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Nervous system disorders
Presyncope
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Pregnancy, puerperium and perinatal conditions
Abortion spontaneous
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Renal and urinary disorders
Acute kidney injury
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Renal and urinary disorders
Nephrolithiasis
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Vascular disorders
Hypertension
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Vascular disorders
Hypotension
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Cardiac disorders
Myocardial infarction
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Gastrointestinal disorders
Colitis
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Infections and infestations
Pneumonia
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Infections and infestations
Viral infection
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Injury, poisoning and procedural complications
Ligament rupture
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Musculoskeletal and connective tissue disorders
Foot deformity
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Basal cell carcinoma
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Nervous system disorders
Metabolic encephalopathy
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Nervous system disorders
Syncope
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Nervous system disorders
Toxic encephalopathy
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Psychiatric disorders
Mental status changes
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
Other adverse events
| Measure |
QGE031 240 mg q4w (TEAE)
n=226 participants at risk
QGE031 240 mg every four weeks (TEAE)
|
QGE031 240 mg q4w (Non-TEAE)
n=226 participants at risk
QGE031 240 mg every four weeks (non-TEAE)
|
|---|---|---|
|
General disorders
Injection site erythema
|
5.8%
13/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.00%
0/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Infections and infestations
Nasopharyngitis
|
25.2%
57/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
4.4%
10/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Infections and infestations
Sinusitis
|
5.8%
13/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.88%
2/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Infections and infestations
Upper respiratory tract infection
|
10.2%
23/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
1.3%
3/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Infections and infestations
Urinary tract infection
|
5.3%
12/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
1.3%
3/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Investigations
Blood creatinine increased
|
5.3%
12/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
5.3%
12/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.44%
1/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
7.1%
16/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.88%
2/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Nervous system disorders
Headache
|
12.8%
29/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
0.88%
2/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
10.2%
23/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
8.0%
18/226 • Within 16 weeks after Week 48
Adverse Events (AEs) are any untoward sign or symptom that occurs during the study treatment
|
Additional Information
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