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

NCT03437278 | Completed Phase 2 Results DMC FDA Drug

• 2 other identifiers: CQGE031C22022017-004207-52
• Study Type: interventional
• Target: 49
• Locations: 10 countries
• Sites: 20

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.

Conditions

Chronic Spontaneous Urticaria

Keywords

Anti-IgE; Chronic spontaneous urticaria

Outcome Measures

Primary Outcomes (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.

  Baseline, week 24

Secondary Outcomes (11)

• Change From Baseline of Weekly Urticaria Activity Score (UAS7) at Weeks 12 and 40

  Baseline, weeks 12 and 40

• Percentage of Participants With Complete Response in Weekly Urticaria Activity Score (UAS7)

  Weeks 12, 24 and 40

• Change From Baseline of Weekly Itch Severity Score (ISS7)

  Baseline, weeks 12, 24 and 40

• Percentage of Participants With Complete Response in Weekly Itch Severity Score (ISS7)

  Weeks 12, 24 and 40

• Change From Baseline of Weekly Hives Severity Score (HSS7)

  Baseline, weeks 12, 24 and 40

Study Arms (3)

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).

Drug: Ligelizumab

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).

Drug: Ligelizumab

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.

Drug: Ligelizumab; Drug: Placebo

Interventions

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.

Also known as: QGE031

Ligelizumab 120 mg; Ligelizumab 24 mg; Placebo + Ligelizumab 120 mg

Placebo DRUG

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

Placebo + Ligelizumab 120 mg

Eligibility Criteria

• Age: 12 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (20)

Novartis Investigative Site

Rosario, Santa Fe Province, S2000BRH, Argentina

Location

Novartis Investigative Site

Bahía Blanca, B8000JRB, Argentina

Location

Novartis Investigative Site

Buenos Aires, C1125ABE, Argentina

Location

Novartis Investigative Site

Brussels, 1200, Belgium

Location

Novartis Investigative Site

Montreal, Quebec, H3H 1P3, Canada

Location

Novartis Investigative Site

Québec, Quebec, G1V 4W2, Canada

Location

Novartis Investigative Site

Berlin, 13353, Germany

Location

Novartis Investigative Site

Mainz, 55131, Germany

Location

Novartis Investigative Site

Budapest, HUN, 1037, Hungary

Location

Novartis Investigative Site

Nashik, Maharashtra, 422 101, India

Location

Novartis Investigative Site

New Delhi, National Capital Territory of Delhi, 110 060, India

Location

Novartis Investigative Site

Bikaner, Rajasthan, 334001, India

Location

Novartis Investigative Site

Saint Petersburg, 191015, Russia

Location

Novartis Investigative Site

Saint Petersburg, 191123, Russia

Location

Novartis Investigative Site

Smolensk, 214019, Russia

Location

Novartis Investigative Site

Barcelona, Barcelona, 08006, Spain

Location

Novartis Investigative Site

Esplugues de Llobregat, Barcelona, 08950, Spain

Location

Novartis Investigative Site

Taipei, Taiwan, 10002, Taiwan

Location

Novartis Investigative Site

Aydin, Turkey, 09100, Turkey (Türkiye)

Location

Novartis Investigative Site

Ankara, 06100, Turkey (Türkiye)

Location

Related Publications (1)

• 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.

  PMID: 37492920 DERIVED

Related Links

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

MeSH Terms

Conditions

Chronic Urticaria

Interventions

ligelizumab

Condition Hierarchy (Ancestors)

Urticaria; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Study Director
• Organization: Novartis Pharmaceuticals

Novartis.email@Novartis.com

+ 1 862 778 8300

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: 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.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: This was a Phase 2b dose-finding, randomized, double-blind, parallel group, placebo controlled multicenter study in adolescent patients

Study Record Dates

• First Submitted: January 31, 2018
• First Posted: February 19, 2018
• Study Start: August 1, 2018
• Primary Completion: October 23, 2020
• Study Completion: February 3, 2021
• Last Updated: January 13, 2026
• Results First Posted: August 24, 2021

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will share

Locations

IN (3); CA (2); DE (2); TU (2); AR (3); BE (1); RU (3); HU (1); ES (2); TW (1)