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Chronic Spontaneous UrticariaPhase 2NCT03437278

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

A Phase 2 study testing Ligelizumab in people with Chronic Spontaneous Urticaria, run by Novartis Pharmaceuticals at 20 sites in 10 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

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). Read more about this study

Who is it for?
Ages 12 to 17 years
Conditions the study is for
Chronic Spontaneous Urticaria
What is being tested?
Ligelizumab
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
49 participants
Where
20 sitesin 10 countries
Study dates
1 Aug 2018to 3 Feb 2021
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 12 to 17 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • 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:
  • The presence of itch and hives for at least 6 consecutive weeks at any time prior to enrollment despite current use of non-sedating H1-antihistamines during this time period
  • UAS7 score (range 0 - 42) ≥ 16 and HSS7 (range 0 - 21) ≥ 8 during 7 days prior to randomization (Day 1)
  • In-clinic UAS ≥ 4 on at least one of the screening visit days or Day 1 or a medical record of the presence of hives (confirmed and documented by a physician); patients must have been on H1-antihistamines for treatment of CSU at the time of in-clinic UAS at screening visit and/or time of the medical record of hives (for at least 3 days prior to the in-clinic UAS or medical record) • Patients must have been on H1-antihistamines for treatment of CSU for at least the 3 consecutive days immediately prior to the first screening visit and must have documented current use on the day of the initial screening visit
Show all 9Show fewer
  • CSU diagnosis for ≥ 6 months
  • 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.

Who cannot join

  • Clearly defined underlying etiology for chronic urticarias other than CSU. This includes the following:
  • Inducible urticaria: urticaria factitia, cold-, heat-, solar-, pressure-, delayed pressure-, aquagenic-, cholinergic-, or contact-urticaria
  • Diseases with possible symptoms of urticaria or angioedema such as urticarial vasculitis, erythema multiforme, cutaneous mastocytosis (urticaria pigmentosa), and hereditary or acquired angioedema (e.g., due to C1 inhibitor deficiency)
  • 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

Trial locations and contacts

20 sites in 10 countries.

20 sites in 10 countries

Argentina3 sites
  • Novartis Investigative Site

    Bahía Blanca, B8000JRB

  • Novartis Investigative Site

    Buenos Aires, C1125ABE

  • Novartis Investigative Site

    Rosario, Santa Fe Province, S2000BRH

Other trials in Argentina

India3 sites
  • Novartis Investigative Site

    Bikaner, Rajasthan, 334001

  • Novartis Investigative Site

    Nashik, Maharashtra, 422 101

  • Novartis Investigative Site

    New Delhi, National Capital Territory of Delhi, 110 060

Other trials in India

Russia3 sites
  • Novartis Investigative Site

    Saint Petersburg, 191015

  • Novartis Investigative Site

    Saint Petersburg, 191123

  • Novartis Investigative Site

    Smolensk, 214019

Other trials in Russia

Canada2 sites
  • Novartis Investigative Site

    Montreal, Quebec, H3H 1P3

  • Novartis Investigative Site

    Québec, Quebec, G1V 4W2

Other trials in Canada

Germany2 sites
  • Novartis Investigative Site

    Berlin, 13353

  • Novartis Investigative Site

    Mainz, 55131

Other trials in Germany

Spain2 sites
  • Novartis Investigative Site

    Barcelona, Barcelona, 08006

  • Novartis Investigative Site

    Esplugues de Llobregat, Barcelona, 08950

Other trials in Spain

Turkey (Türkiye)2 sites
  • Novartis Investigative Site

    Ankara, 06100

  • Novartis Investigative Site

    Aydin, Turkey, 09100

Other trials in Turkey (Türkiye)

Belgium1 site
  • Novartis Investigative Site

    Brussels, 1200

Other trials in Belgium

Hungary1 site
  • Novartis Investigative Site

    Budapest, HUN, 1037

Other trials in Hungary

Taiwan1 site
  • Novartis Investigative Site

    Taipei, Taiwan, 10002

Other trials in Taiwan

About this study

Official title: A Multicenter, Randomized, Double-blind, Placebo-controlled Phase 2b Dose-finding Study to Investigate the Efficacy and Safety of Ligelizumab (QGE031) in Adolescent Patients With Chronic Spontaneous Urticaria (CSU)

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.

Read the detailed description

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.

Being tested
Ligelizumab, Placebo
Keywords
Anti-IgE, Chronic spontaneous urticaria
Medical subject headings
Chronic Urticaria

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • LigelizumabDrug

    Also called QGE031

    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.

  • PlaceboDrug

    Placebo 0 mg per 1 ml liquid injection once every 4 weeks.

Study groups in NCT03437278
Study groupRoleWhat they receive
Ligelizumab 120 mgExperimentalParticipants 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).
Receives: Drug: Ligelizumab
Ligelizumab 24 mgExperimentalParticipants 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).
Receives: Drug: Ligelizumab
Placebo + Ligelizumab 120 mgPlacebo comparatorParticipants 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.
Receives: Drug: Ligelizumab; Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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.

    Measured: Baseline, week 24

Secondary outcomes (11)
  1. 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.

    Measured: Baseline, weeks 12 and 40

  2. 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.

    Measured: Weeks 12, 24 and 40

  3. 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.

    Measured: Baseline, weeks 12, 24 and 40

  4. 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.

    Measured: Weeks 12, 24 and 40

  5. 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.

    Measured: Baseline, weeks 12, 24 and 40

  6. 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.

    Measured: Weeks 12, 24 and 40

  7. 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.

    Measured: Baseline, weeks 12, 24 and 40

  8. 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.

    Measured: Baseline, weeks 12, 24 and 40

  9. 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).

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

  10. 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).

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

  11. 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.

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

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted19 Feb 2018
  2. Study start1 Aug 2018
  3. Primary completion23 Oct 2020
  4. Study completion3 Feb 2021

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
This was a Phase 2b dose-finding, randomized, double-blind, parallel group, placebo controlled multicenter study in adolescent patients
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
49 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03437278
Sponsor's protocol ID
CQGE031C2202
Other IDs
2017-004207-52, 2017
Status last verified
Dec 2025
First submitted
31 Jan 2018
Last updated
13 Jan 2026
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: 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

Publications

  • 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.

    PubMed 37492920

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Ligelizumab in people with Chronic Spontaneous Urticaria, run by Novartis Pharmaceuticals at 20 sites in 10 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 9 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 13 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 20 sites in Argentina, India, Russia, Canada, Germany and 5 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 24 Aug 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change From Baseline of Weekly Urticaria Activity Score (UAS7) at Week 24 (time frame: Baseline, week 24). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03437278, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Jan 2026.

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