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

NCT ID: NCT03437278

Last Updated: 2026-01-13

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 49 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-08-01
• Study Completion Date: 2021-02-03

Brief 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). 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.

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.

Conditions

Chronic Spontaneous Urticaria

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

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).

Group Type: EXPERIMENTAL

Ligelizumab

Intervention Type: 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.

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).

Group Type: EXPERIMENTAL

Ligelizumab

Intervention Type: 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.

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.

Group Type: PLACEBO_COMPARATOR

Ligelizumab

Intervention Type: 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.

Placebo

Intervention Type: DRUG

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

Interventions

Ligelizumab

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.

Intervention Type: DRUG

Placebo

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

Intervention Type: DRUG

Other Intervention Names

QGE031

Eligibility Criteria

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:

• 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
• 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.

Exclusion Criteria

• 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

• Minimum Eligible Age: 12 Years
• Maximum Eligible Age: 17 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Novartis Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Novartis Investigative Site

Rosario, Santa Fe Province, Argentina

Novartis Investigative Site

Bahía Blanca, , Argentina

Novartis Investigative Site

Buenos Aires, , Argentina

Novartis Investigative Site

Brussels, , Belgium

Novartis Investigative Site

Montreal, Quebec, Canada

Novartis Investigative Site

Québec, Quebec, Canada

Novartis Investigative Site

Berlin, , Germany

Novartis Investigative Site

Mainz, , Germany

Novartis Investigative Site

Budapest, HUN, Hungary

Novartis Investigative Site

Nashik, Maharashtra, India

Novartis Investigative Site

New Delhi, National Capital Territory of Delhi, India

Novartis Investigative Site

Bikaner, Rajasthan, India

Novartis Investigative Site

Saint Petersburg, , Russia

Novartis Investigative Site

Saint Petersburg, , Russia

Novartis Investigative Site

Smolensk, , Russia

Novartis Investigative Site

Barcelona, Barcelona, Spain

Novartis Investigative Site

Esplugues de Llobregat, Barcelona, Spain

Novartis Investigative Site

Taipei, Taiwan, Taiwan

Novartis Investigative Site

Aydin, Turkey, Turkey (Türkiye)

Novartis Investigative Site

Ankara, , Turkey (Türkiye)

Countries

Argentina; Belgium; Canada; Germany; Hungary; India; Russia; Spain; Taiwan; Turkey (Türkiye)

References

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.

Reference Type: DERIVED

PMID: 37492920 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

https://www.novctrd.com/ctrdweb/patientsummary/patientsummaries?patientSummaryId=985

A Plain Language Trial Summary is available on novctrd.com

https://www.novctrd.com/ctrdweb/ppatientsummary/ppatientsummaries?periodicPatientSummaryId=514

A Pediatric Plain Language Trial Summary is available on novctrd.com

Other Identifiers

2017-004207-52

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CQGE031C2202

Identifier Type: -

Identifier Source: org_study_id