Clinical trial NCT01723072: Impact of Omalizumab on Quality of Life Measures and Angioedema Occurrence in Patients With CSU Refractory to Therapy
Official title: A Randomized, Double-blind, Placebo-controlled, 28-week Treatment With a 8 Week Follow-up to Investigate the Impact of Omalizumab on Quality of Life Measures and the Incidence and Severity of Angioedema Despite H1-antihistamine Therapy.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Chronic Spontaneous Urticaria
Angioedema
Keywords: Chronic spontanous urticaria, refractory CSU, Angioedema
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2012-11-07
Enrollment: 91
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This study will assess the impact of omalizumab on the quality of life improvement when added to the standard therapy in refractory patients suffering from chronic spontaneous urticaria and angioedema.

Detailed description: This study will be conducted using a double-blind, placebo-controlled, randomized, multicenter design to investigate the impact of omalizumab on Quality of Life (QoL) in Chronic Spontaneous Urticaria (CSU) and will assess its effectiveness in reducing and/or inhibiting the occurrence, time-course and severity of angioedema episodes.

Omalizumab is administered subcutaneously every 4 weeks as an add-on therapy to the current approved treatment of adult CSU patients. The treatment period is 28 weeks which is followed by an 8-week follow-up. The study aims to enroll approximately 70 patients.

The study is divided into a 2-week screening period, a 28-week double-blind treatment phase with omalizumab or placebo-omalizumab, and an 8-week follow-up phase.

Eligibility criteria: Inclusion Criteria:

* Diagnosis of chronic spontaneous urticaria (CSU) refractory to H1-antihistamine treatment
* Presence of itch an hives for more than 6 weeks
* UAS7 score of more than 14 (range 0-42)
* Patient has history of insufficient response to 4x of the approved dose of H1-antihistamines
* CSU diagnosis for more than 6 months
* Angioedema at least 4x in the last 6 months

Exclusion Criteria:

* Patients with non urticaria associated angioedema
* History of hypersensitivity to omalizumab or the rescue medication or to drugs of similar chemical structure
* Evidence of parasitic infection
* Previous treatment with omalizumab within the last 6 months prior to screening
* History of anaphylactic shock
* Woman who are pregnant or breast feeding
* Other protocol-defined inclusion/exclusion criteria may apply.

Primary outcomes:
measure: Mean Change From Baseline Using Chronic Urticaria Quality of Life Questionnaire (CU-Q2oL) Total Scores During the Study: Unadjusted Analysis and ANCOVA (LOCF) (FAS)
timeFrame: Baseline, 4, 12, 20, 28 and 36 weeks
Secondary outcomes:
measure: Number of Angioedema Burdened Days by Study Phase (Observed Cases With Imputation)
timeFrame: Baseline, week 28; and the follow-up period (weeks 29-36)
measure: Mean Time Interval Between Successive Angioedema Episodes of the First 15 Episodes
timeFrame: Baseline to week 28
measure: Change of Angioedema Activity Score (AAS) Total Week Sum Scores: Unadjusted Analysis and ANCOVA (Observed Cases With Imputation)
timeFrame: Baseline to week 28; and follow-up period (to week 36)
measure: Diameter: Acute Swelling Episodes Within the Screening Period (Week -2 to -1)
timeFrame: week -2 to -1
measure: Diameter: Acute Swelling Episodes at End of Treatment (Weeks 25 to 28)
timeFrame: weeks 25 to 28
measure: Diameter: Acute Swelling Episodes at End of Follow-up(Weeks 33 to 36)
timeFrame: weeks 33 to 36
measure: Shortness of Breath: Acute Swelling Episodes Within the Screening Period (Weeks -2 to -1)
timeFrame: weeks -2 to -1
measure: Shortness of Breath: Acute Swelling Episodes at End of Treatment Period (Weeks 25 to 28)
timeFrame: weeks 25 to 28
measure: Shortness of Breath: Acute Swelling Episodes at End of Follow-up Period (Weeks 33 to 36)
timeFrame: weeks 33 to 36
measure: Change of AE-Q2oL Scores From Baseline to Week 28: Unadjusted Analysis and ANCOVA (Observed Cases)
timeFrame: baseline to week 28; and then follow-up to week 36
measure: Rescue Medication During the Treatment Period
timeFrame: baseline to 28 weeks
measure: Days of Rescue Medication During the Treatment Period
timeFrame: baseline to 28 weeks
measure: Days of Rescue Medication During the Follow-up Period
timeFrame: weeks 33 to 36
measure: Change of UAS7 Total Scores From Baseline to Week 28: Unadjusted Analysis and ANCOVA (Observed Cases)
timeFrame: baseline to week 28; and then follow-up to week 36
measure: Change of DLQI Scores From Baseline to Week 28: Unadjusted Analysis and ANCOVA (Observed Cases)
timeFrame: baseline to week 28; and follow-up to week 36
Other outcomes:
Locations:
city: Mannheim
country: Germany
facility: Novartis Investigative Site
state: Baden-Wuerttemberg
city: Regensburg
country: Germany
facility: Novartis Investigative Site
state: Bavaria
city: Koeln
country: Germany
facility: Novartis Investigative Site
state: Nordrhein-Westfalen
city: Aachen
country: Germany
facility: Novartis Investigative Site
city: Augsburg
country: Germany
facility: Novartis Investigative Site
city: Berlin
country: Germany
facility: Novartis Investigative Site
city: Bochum
country: Germany
facility: Novartis Investigative Site
city: Bonn
country: Germany
facility: Novartis Investigative Site
city: Darmstadt
country: Germany
facility: Novartis Investigative Site
city: Dresden
country: Germany
facility: Novartis Investigative Site
city: Duesseldorf
country: Germany
facility: Novartis Investigative Site
city: Erlangen
country: Germany
facility: Novartis Investigative Site
city: Essen
country: Germany
facility: Novartis Investigative Site
city: Freiburg
country: Germany
facility: Novartis Investigative Site
city: Gera
country: Germany
facility: Novartis Investigative Site
city: Halle
country: Germany
facility: Novartis Investigative Site
city: Hannover
country: Germany
facility: Novartis Investigative Site
city: Homburg
country: Germany
facility: Novartis Investigative Site
city: Kiel
country: Germany
facility: Novartis Investigative Site
city: Mainz
country: Germany
facility: Novartis Investigative Site
city: Muenchen
country: Germany
facility: Novartis Investigative Site
city: Muenchen
country: Germany
facility: Novartis Investigative Site
city: Oldenburg
country: Germany
facility: Novartis Investigative Site
city: Selters
country: Germany
facility: Novartis Investigative Site
city: Wiesbaden
country: Germany
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References:
citation: Staubach P, Metz M, Chapman-Rothe N, Sieder C, Brautigam M, Canvin J, Maurer M. Effect of omalizumab on angioedema in H1 -antihistamine-resistant chronic spontaneous urticaria patients: results from X-ACT, a randomized controlled trial. Allergy. 2016 Aug;71(8):1135-44. doi: 10.1111/all.12870. Epub 2016 Mar 31.