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| ID | Type | Description | Link |
|---|---|---|---|
| 2017-004207-52 | EudraCT Number |
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This clinical study was designed to evaluate the pharmacokinetics, safety and efficacy of ligelizumab in children from 12 to < 18 years of age, with chronic spontaneous urticaria (CSU). The participants were treated with ligelizumab as an add-on therapy to approved doses of H1 antihistamines (H1AH) following the guideline on treatment of CSU.
This was a Phase IIb dose-finding, randomized, double-blind, parallel-group, placebo-controlled, multicenter study in adolescent patients. The study consisted of 3 distinct study periods: Screening, Treatment and Follow-up period.
After the screening period (up to 4 weeks), at Day 1 participants were randomized into one of the three treatment arms in 1:2:1 fashion to ligelizumab high dose (120 mg every four weeks (q4w)) versus ligelizumab low dose (24 mg q4w) versus placebo. During the 24 weeks of treatment period, doses were administered on Day 1 then on weeks 4, 8, 12, 16, and 20 weeks after randomization. Participants randomized to placebo received placebo on Day 1, Weeks 4 and 8; thereafter they received 120 mg ligelizumab (high dose) on Weeks 12, 16 and 20 such that by the end of the study, the same number of participants received ligelizumab high dose as low dose. The treatment period was followed by a treatment-free follow-up period of 16 weeks to a maximum of Week 40.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Ligelizumab 120 mg | Experimental | 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). |
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| Ligelizumab 24 mg | Experimental | 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). |
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| Placebo + Ligelizumab 120 mg | Placebo Comparator | 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. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Ligelizumab | Drug | Ligelizumab comes in 120 mg per 1 ml liquid vials. Participants received one injection every 4 weeks at a dose of 120 mg or 24 mg, high and low doses respectively. |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline of Weekly Urticaria Activity Score (UAS7) at Week 24 | 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. | Baseline, week 24 |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline of Weekly Urticaria Activity Score (UAS7) at Weeks 12 and 40 | 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. |
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Inclusion Criteria:
Parent or legal guardian's written informed consent and child's assent, if appropriate, must be obtained before any study related activity or assessment is performed. Of note, if the subject reaches age of consent (age as per local law) during the study, they will also need to sign the corresponding study ICF (Informed Consent Form) at the next study visit.
Male and female adolescent patients aged ≥ 12 to <18 years at the time of screening.
Diagnosis of CSU refractory to approved doses of H1-antihistamines at the time of randomization, as defined by all of the following:
Willing and able to complete a daily symptom e-Diary for the duration of the study and adhere to the study visit schedules.
Demonstration of compliance with the e-Diary: patients should not have had any missing e-Diary entries in the 7 days prior to randomization. Re-screening may be considered.
Exclusion Criteria:
Clearly defined underlying etiology for chronic urticarias other than CSU. This includes the following:
Any other skin disease associated with chronic itching that might confound the study evaluations and results (e.g. atopic dermatitis, bullous pemphigoid, dermatitis herpetiformis etc.)
Previous exposure to omalizumab
History of anaphylaxis
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis Investigative Site | Rosario | Santa Fe Province | S2000BRH | Argentina | ||
| Novartis Investigative Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37492920 | Derived | Staubach P, Alvaro-Lozano M, Sekerel BE, Maurer M, Ben-Shoshan M, Porter M, Hua E, Ji Y, Burciu A, Savelieva M, Severin T, Drollmann A, Bienczak A. Ligelizumab in adolescents with chronic spontaneous urticaria: Results of a dedicated phase 2b randomized clinical trial supported with pharmacometric analysis. Pediatr Allergy Immunol. 2023 Jul;34(7):e13982. doi: 10.1111/pai.13982. |
| Label | URL |
|---|---|
| A Plain Language Trial Summary is available on novctrd.com | View source |
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Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.
This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
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Participants underwent a Screening period of up to 4 weeks.
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).
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| ID | Title | Description |
|---|---|---|
| FG000 | 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). |
| FG001 | Ligelizumab 120 mg |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jan 18, 2018 | Jul 28, 2021 |
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This was a Phase 2b dose-finding, randomized, double-blind, parallel group, placebo controlled multicenter study in adolescent patients
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Participants, investigator staff and personnel performing the study assessments remained blinded to the identity of the treatment from the time of randomization until final database lock. Data managers, programmers, statisticians, pharmacometricians and clinical experts of the Novartis trial team also remained blinded until final database lock. The study drug was prepared by an independent unblinded pharmacist (or authorized delegate) and administered by an independent unblinded administrator.
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| Placebo | Drug | Placebo 0 mg per 1 ml liquid injection once every 4 weeks. |
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| Baseline, weeks 12 and 40 |
| Percentage of Participants With Complete Response in Weekly Urticaria Activity Score (UAS7) | 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 complete UAS7 response is defined as UAS7=0, no wheals neither pruritus. Participants with post-baseline missing data were considered as non-responders. | Weeks 12, 24 and 40 |
| Change From Baseline of Weekly Itch Severity Score (ISS7) | 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. | Baseline, weeks 12, 24 and 40 |
| Percentage of Participants With Complete Response in Weekly Itch Severity Score (ISS7) | 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. Participants with post-baseline missing data were considered as non-responders. | Weeks 12, 24 and 40 |
| Change From Baseline of Weekly Hives Severity Score (HSS7) | 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. | Baseline, weeks 12, 24 and 40 |
| Percentage of Participants With Complete Response in Weekly Hives Severity Score (HSS7) | 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. Participants with post-baseline missing data were considered as non-responders. | Weeks 12, 24 and 40 |
| Change From Baseline of the Children Dermatology Life Quality Index (CDLQI) | 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. | Baseline, weeks 12, 24 and 40 |
| Change From Baseline in Total Human Immunoglobulin E (IgE) | Change from baseline in IgE (free IgE plus IgE bound to ligelizumab) at weeks 12, 24 and 40 as a pharmacodynamic measurement. | Baseline, weeks 12, 24 and 40 |
| Apparent Clearance (CL/F) of Ligelizumab Estimated With a PopPK Model | 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). | Weeks 0 (baseline), 4, 8, 12, 16, 20 (all pre-dose) and weeks 24, 32 and 40 |
| Apparent Volume of Distribution of Ligelizumab Estimated With a PopPK Model | 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). | Weeks 0 (baseline), 4, 8, 12, 16, 20 (all pre-dose) and weeks 24, 32 and 40 |
| Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | 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. | From the start of treatment to 20 weeks after end of treatment, assessed up to maximum duration of 40 weeks |
| Bahía Blanca |
| B8000JRB |
| Argentina |
| Novartis Investigative Site | Buenos Aires | C1125ABE | Argentina |
| Novartis Investigative Site | Brussels | 1200 | Belgium |
| Novartis Investigative Site | Montreal | Quebec | H3H 1P3 | Canada |
| Novartis Investigative Site | Québec | Quebec | G1V 4W2 | Canada |
| Novartis Investigative Site | Berlin | 13353 | Germany |
| Novartis Investigative Site | Mainz | 55131 | Germany |
| Novartis Investigative Site | Budapest | HUN | 1037 | Hungary |
| Novartis Investigative Site | Nashik | Maharashtra | 422 101 | India |
| Novartis Investigative Site | New Delhi | National Capital Territory of Delhi | 110 060 | India |
| Novartis Investigative Site | Bikaner | Rajasthan | 334001 | India |
| Novartis Investigative Site | Saint Petersburg | 191015 | Russia |
| Novartis Investigative Site | Saint Petersburg | 191123 | Russia |
| Novartis Investigative Site | Smolensk | 214019 | Russia |
| Novartis Investigative Site | Barcelona | Barcelona | 08006 | Spain |
| Novartis Investigative Site | Esplugues de Llobregat | Barcelona | 08950 | Spain |
| Novartis Investigative Site | Taipei | Taiwan | 10002 | Taiwan |
| Novartis Investigative Site | Aydin | Turkey | 09100 | Turkey (Türkiye) |
| Novartis Investigative Site | Ankara | 06100 | Turkey (Türkiye) |
| A Pediatric Plain Language Trial Summary is available on novctrd.com | View source |
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).
| FG002 | 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. |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | 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). |
| BG001 | 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). |
| BG002 | 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. |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
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| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change From Baseline of Weekly Urticaria Activity Score (UAS7) at Week 24 | 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. | 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. | Posted | Mean | Standard Deviation | Score on a scale | Baseline, week 24 |
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| Secondary | Change From Baseline of Weekly Urticaria Activity Score (UAS7) at Weeks 12 and 40 | 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. | 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. | Posted | Mean | Standard Deviation | Score on a scale | Baseline, weeks 12 and 40 |
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| Secondary | Percentage of Participants With Complete Response in Weekly Urticaria Activity Score (UAS7) | 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 complete UAS7 response is defined as UAS7=0, no wheals neither pruritus. Participants with post-baseline missing data were considered as non-responders. | All participants to whom study treatment was assigned. Participants with post-baseline missing data were considered as non-responders. | Posted | Count of Participants | Participants | Weeks 12, 24 and 40 |
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| Secondary | Change From Baseline of Weekly Itch Severity Score (ISS7) | 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. | 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, 24 and 40) were analyzed. | Posted | Mean | Standard Deviation | Score on a scale | Baseline, weeks 12, 24 and 40 |
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| Secondary | Percentage of Participants With Complete Response in Weekly Itch Severity Score (ISS7) | 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. Participants with post-baseline missing data were considered as non-responders. | All participants to whom study treatment was assigned. Participants with post-baseline missing data were considered as non-responders. | Posted | Count of Participants | Participants | Weeks 12, 24 and 40 |
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| Secondary | Change From Baseline of Weekly Hives Severity Score (HSS7) | 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. | 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, 24 and 40) were analyzed. | Posted | Mean | Standard Deviation | Score on a scale | Baseline, weeks 12, 24 and 40 |
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| Secondary | Percentage of Participants With Complete Response in Weekly Hives Severity Score (HSS7) | 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. Participants with post-baseline missing data were considered as non-responders. | All participants to whom study treatment was assigned. Participants with post-baseline missing data were considered as non-responders. | Posted | Count of Participants | Participants | Weeks 12, 24 and 40 |
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| Secondary | Change From Baseline of the Children Dermatology Life Quality Index (CDLQI) | 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. | 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). | Posted | Mean | Standard Deviation | Score on a scale | Baseline, weeks 12, 24 and 40 |
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| Secondary | Change From Baseline in Total Human Immunoglobulin E (IgE) | Change from baseline in IgE (free IgE plus IgE bound to ligelizumab) at weeks 12, 24 and 40 as a pharmacodynamic measurement. | 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. | Posted | Mean | Standard Deviation | International units / millilitre | Baseline, weeks 12, 24 and 40 |
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| Secondary | Apparent Clearance (CL/F) of Ligelizumab Estimated With a PopPK Model | 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). | 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. | Posted | Median | Inter-Quartile Range | Liters / day | Weeks 0 (baseline), 4, 8, 12, 16, 20 (all pre-dose) and weeks 24, 32 and 40 |
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| Secondary | Apparent Volume of Distribution of Ligelizumab Estimated With a PopPK Model | 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). | 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. | Posted | Median | Inter-Quartile Range | Liters | Weeks 0 (baseline), 4, 8, 12, 16, 20 (all pre-dose) and weeks 24, 32 and 40 |
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| Secondary | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | 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. | All participants who received at least one dose of study treatment. | Posted | Count of Participants | Participants | From the start of treatment to 20 weeks after end of treatment, assessed up to maximum duration of 40 weeks |
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Adverse events were reported from the start treatment to 20 weeks after end of treatment, assessed up to maximum duration of 40 weeks.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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). | 0 | 24 | 1 | 24 | 18 | 24 |
| EG001 | 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). | 0 | 13 | 0 | 13 | 11 | 13 |
| EG002 | 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. | 0 | 12 | 1 | 12 | 9 | 12 |
| EG003 | Total | Total | 0 | 49 | 2 | 49 | 38 | 49 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pulmonary valve incompetence | Cardiac disorders | MedDRA (23.1) | Systematic Assessment |
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| Tricuspid valve incompetence | Cardiac disorders | MedDRA (23.1) | Systematic Assessment |
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| Suicide attempt | Psychiatric disorders | MedDRA (23.1) | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Eye pruritus | Eye disorders | MedDRA (23.1) | Systematic Assessment |
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| Abdominal pain | Gastrointestinal disorders | MedDRA (23.1) | Systematic Assessment |
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| Abdominal pain upper | Gastrointestinal disorders | MedDRA (23.1) | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA (23.1) | Systematic Assessment |
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| Gastritis | Gastrointestinal disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Odynophagia | Gastrointestinal disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Administration site erythema | General disorders and administration site conditions | MedDRA (23.1) | Systematic Assessment |
| |
| Injection site erythema | General disorders and administration site conditions | MedDRA (23.1) | Systematic Assessment |
| |
| Injection site pain | General disorders and administration site conditions | MedDRA (23.1) | Systematic Assessment |
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| Injection site reaction | General disorders and administration site conditions | MedDRA (23.1) | Systematic Assessment |
| |
| Pyrexia | General disorders and administration site conditions | MedDRA (23.1) | Systematic Assessment |
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| Hyperbilirubinaemia | Hepatobiliary disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Gastrointestinal infection | Infections and infestations | MedDRA (23.1) | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (23.1) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (23.1) | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA (23.1) | Systematic Assessment |
| |
| Post procedural infection | Infections and infestations | MedDRA (23.1) | Systematic Assessment |
| |
| Respiratory tract infection viral | Infections and infestations | MedDRA (23.1) | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA (23.1) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (23.1) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (23.1) | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA (23.1) | Systematic Assessment |
| |
| Animal bite | Injury, poisoning and procedural complications | MedDRA (23.1) | Systematic Assessment |
| |
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA (23.1) | Systematic Assessment |
| |
| Face injury | Injury, poisoning and procedural complications | MedDRA (23.1) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Fibromyalgia | Musculoskeletal and connective tissue disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Medial tibial stress syndrome | Musculoskeletal and connective tissue disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Intercostal neuralgia | Nervous system disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Migraine | Nervous system disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Dysmenorrhoea | Reproductive system and breast disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Menstruation irregular | Reproductive system and breast disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Angioedema | Skin and subcutaneous tissue disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Chronic spontaneous urticaria | Skin and subcutaneous tissue disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Onycholysis | Skin and subcutaneous tissue disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (23.1) | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA (23.1) | Systematic Assessment |
|
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.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Novartis Pharmaceuticals | + 1 862 778 8300 | Novartis.email@Novartis.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Mar 11, 2021 | Jul 28, 2021 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D000080223 | Chronic Urticaria |
| ID | Term |
|---|---|
| D014581 | Urticaria |
| D017445 | Skin Diseases, Vascular |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D006969 | Hypersensitivity, Immediate |
| D006967 | Hypersensitivity |
| D007154 | Immune System Diseases |
| D002908 | Chronic Disease |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
Not provided
Not provided
| ID | Term |
|---|---|
| C000598891 | ligelizumab |
Not provided
Not provided
Not provided
| Male |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
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).
| OG002 | 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. |
|
|
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.
|
|
| OG002 | 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. |
|
|
|
|
| OG002 | 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. |
|
|
|
|
| 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). |
| OG002 | 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. |
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