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

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.


Study Type: Interventional  (Clinical Trial)
Estimated Enrollment: 50 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: This is 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: Patients, investigator staff and personnel performing the study assessments will remain 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 will also remain blinded until final database lock. The study drug must be prepared by an independent unblinded pharmacist (or authorized delegate) and administered by an independent unblinded administrator.
Primary Purpose: Treatment
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)
Estimated Study Start Date: May 1, 2018
Estimated Primary Completion Date: March 17, 2020
Estimated Study Completion Date: March 17, 2020

Arms:
- Experimental: A. Ligelizumab high dose
- Experimental: B.Ligelizumab low dose
- Placebo Comparator: C. Placebo