Study to Investigate the Efficacy and Safety of QGE031 in Adolescent Patients With Chronic Spontaneous Urticaria (CSU)
Condition: Chronic Spontaneous Urticaria
MeSH terms: Urticaria; Chronic Urticaria
Intervention: Ligelizumab; Placebo
Intervention description: 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 0 mg per 1 ml liquid injection once every 4 weeks.
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Intervention model description: This was a Phase 2b dose-finding, randomized, double-blind, parallel group, placebo controlled multicenter study in adolescent patients
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking description: 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.
Enrollment: 49.0
Sex: All
Minimum age: 12 Years
Maximum age: 17 Years
Healthy volunteers: No
Keywords: Anti-IgE; Chronic spontaneous urticaria

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.
    

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