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

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)

ClinicalTrials.gov Identifier: NCT03437278

Novartis Reference Number: CQGE031C2202

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All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation. 

Study Description

This clinical study is designed to evaluate the pharmacokinetics, safety and efficacy of ligelizumab in children from 12 to <18 years of age, with chronic spontaneous urticaria.

The study will generate data to be analyzed in a separate modelling and simulation study with the objective of i) estimating the relative potency of ligelizumab in adolescent patients compared with adults and ii) proposing a posology for the future treatment of adolescent patients.

The patient population will be treated with ligelizumab as an add-on therapy to approved doses of H1- antihistamines following the guideline on treatment of CSU .

The study consists of 3 distinct study periods: Screening period (up to 4 weeks) Treat period: Day 1 patients are randomized into one of the three treatment arms in 1:2:1 fashion to ligelizumab high dose q4w vs. ligelizumab low dose q4w vs. placebo. During the 24 weeks of treatment, doses are administered on Day 1 then 4, 8, 12, 16, and 20 weeks after randomization. Subjects randomized to placebo will receive placebo on Day 1, Weeks 4 and 8; thereafter they will receive ligelizumab (high dose) at Weeks 12, 16 and 20 such that the same number of patients will, by the end of the study, receive the high dose as the low dose. Safety is assessed every 4 weeks; efficacy is primarily assessed using daily itch and hives scores summed into the weekly UAS7. The treatment period (24 weeks) is followed by a follow-up period of 16 weeks to a maximum of Week 40.

Condition 
Chronic Spontaneous Urticaria
Phase 
Phase 2
Overall status 
Active, not recruiting
Enrollment count 
49 participants
Start date 
Aug 01, 2018
Completion date 
Jun 15, 2021
Gender 
All
Age(s)
12 Years - 18 Years (Child, Adult)

Interventions

Drug
Ligelizumab
Ligelizumab comes in 120 mg per 1 mL liquid in vial. If the patient is on the high or low dose of ligelizumab, the patient will receive 1 injection of ligelizumab every 4 weeks from Day 1 to Week 20 (inclusive).
Drug
Placebo
Placebo comes in 00 mg per 1 mL liquid in vial. The patient will receive 1 injection of placebo at Day 1, Week 4, Week 8; followed by one injection of ligelizumab at Week 12, Week 16 and Week 20

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

Study Locations

Argentina
Novartis Investigative Site
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Rosario, S2000BRH
Santa Fe
Argentina
Novartis Investigative Site
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Bahia Blanca, B8000JRB
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Argentina
Novartis Investigative Site
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Buenos Aires, C1125ABE
-
Argentina
Belgium
Novartis Investigative Site
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Bruxelles, 1200
-
Belgium
Canada
Novartis Investigative Site
-
Montreal, H3H 1P3
Quebec
Canada
Novartis Investigative Site
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Quebec, G1V 4W2
-
Canada
Germany
Novartis Investigative Site
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Berlin, 13353
-
Germany
Novartis Investigative Site
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Mainz, 55131
-
Germany
Hungary
Novartis Investigative Site
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Budapest, 1037
HUN
Hungary
India
Novartis Investigative Site
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New Delhi, 110 060
Delhi
India
Novartis Investigative Site
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Nashik, 422 101
Maharashtra
India
Novartis Investigative Site
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Bikaner, 334001
Rajasthan
India
Russian Federation
Novartis Investigative Site
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Saint Petersburg, 191123
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Russian Federation
Novartis Investigative Site
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Smolensk, 214019
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Russian Federation
Novartis Investigative Site
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St Petersburg, 191015
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Russian Federation
Spain
Novartis Investigative Site
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Esplugues de Llobregat, 08950
Barcelona
Spain
Novartis Investigative Site
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Barcelona, 08006
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Spain
Taiwan
Novartis Investigative Site
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Taipei, 10002
-
Taiwan
Turkey
Novartis Investigative Site
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Ankara, 06100
-
Turkey
Novartis Investigative Site
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Aydin, 09100
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Turkey

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Call 1-999-669-6682 or email [email protected]