E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Chronic Spontaneous Urticaria |
|
E.1.1.1 | Medical condition in easily understood language |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Skin and Connective Tissue Diseases [C17] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10072757 |
E.1.2 | Term | Chronic spontaneous urticaria |
E.1.2 | System Organ Class | 10040785 - Skin and subcutaneous tissue disorders |
|
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The primary objective of this study is to assess the change in the Urticaria Activity Score (UAS7) between baseline and Week 24 |
|
E.2.2 | Secondary objectives of the trial |
- To evaluate the efficacy of ligelizumab doses with respect to UAS7 change from baseline
- To evaluate the efficacy of ligelizumab doses on complete response in Urticaria Activity Score (UAS7=0), Hives Severity Score (HSS=0), Itch Severity score (ISS=0)
- To investigate the effects on ISS7 and HSS7 when compared to baseline
- To investigate the pharmacokinetics and Pharmacodynamics of ligelizumab
- Change from baseline in the Children Dermatology Life Quality Index
- To evaluate the safety (including immunogenicity) and tolerability of ligelizumab versus placebo in patients with CSU |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Parent or legal guardian’s written informed consent and child’s assent
- Male and female adolescent patients aged ≥ 12 to <18 years at the time of screening
- CSU diagnosis ≥ 6 months and presence of itch and hives for at least 6 consecutive weeks at any time prior to enrollment
- Diagnosis of CSU refractory to approved doses of H1-antihistamines at the time of randomization
- Other protocol-defined criteria may apply |
|
E.4 | Principal exclusion criteria |
- Clearly defined underlying etiology for chronic urticarias other than CSU
- Any other skin disease associated with chronic itching that might confound the study evaluations and results
- Previous exposure to omalizumab
- History of anaphylaxis
- Other protocol-defined criteria may apply |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Change in the Urticaria Activity Score (UAS7) between baseline and Week 24 |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
- UAS7 change from baseline over time
- Rate of complete responders (UAS7=0, HSS7=0, ISS7 =0) over time
- Itch severity score and Hives severity score change from baseline over time
- Model-based estimate of clearance and volume of distribution using at least 7 samples
- Summary statistics of change in Total IgE over time
- Children Dermatology Life Quality Index change from baseline over time
- Adverse events, ECG-intervals and interpretation, vital signs (blood pressure, pulse rate) and clinical laboratory evaluation |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
- At each protocol defined study visit |
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | Yes |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
|
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 3 |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 14 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Argentina |
Belgium |
Canada |
Estonia |
Germany |
Hungary |
India |
Russian Federation |
Spain |
Switzerland |
Taiwan |
Turkey |
United States |
|
E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
Completion of the study will be considered when all patients will have completed 24 weeks of treatment epoch and the full follow up epoch or have prematurely withdrawn from the study |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 7 |
E.8.9.1 | In the Member State concerned days | 27 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 2 |
E.8.9.2 | In all countries concerned by the trial days | 0 |