A Study of the Efficacy and Safety of Omalizumab (Xolair) in Patients With Chronic Idiopathic Urticaria (CIU)/Chronic Spontaneous Urticaria (CSU) Who Remain Symptomatic Despite Antihistamine (H1) Treatment

NCT ID: NCT01287117

Last Updated: 2013-11-27

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

319 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-02-28

Study Completion Date

2012-10-31

Brief Summary

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The study is a global Phase III, multicenter, randomized, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy and safety of omalizumab administered subcutaneously as an add-on therapy for the treatment of adolescent and adult patients aged 12-75 who have been diagnosed with refractory CIU and who remain symptomatic despite standard-dose H1 antihistamine treatment.

Detailed Description

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Type I Error Rate Control Plan

Primary Outcome Measure

In order to maintain an overall type I error rate of 0.05 (2-sided) across the 3 omalizumab dose levels, the testing of the primary Outcome Measure was conducted in the following hierarchical order. A p-value \< 0.05 is only considered statistically significant if statistical significance was claimed at the previous stage.

* Stage 1: Omalizumab 300-mg group vs. placebo
* Stage 2: Omalizumab 150-mg group vs. placebo
* Stage 3: Omalizumab 75-mg group vs. placebo

Secondary Outcome Measures

A hierarchical analysis of the following secondary Outcome Measures was performed for each dose found to be significant in the primary Outcome Measure. A p-value \< 0.05 is only considered statistically significant if statistical significance was claimed at the previous stage.

* Stage 1: Change from baseline to Week 12 in the urticaria activity score over 7 days (UAS7)
* Stage 2: Change from Baseline to Week 12 in the weekly number of hives score
* Stage 3: Time to minimally important difference (MID) response in the weekly itch severity score by Week 12
* Stage 4: Percentage of participants with a UAS7 score ≤ 6 at Week 12
* Stage 5: Percentage of weekly itch severity score MID responders at Week 12
* Stage 6: Change from Baseline to Week 12 in the weekly size of the largest hive score
* Stage 7: Change from Baseline in the overall dermatology life quality index (DLQI) score at Week 12
* Stage 8: Change from Baseline in the overall dermatology life quality index (DLQI) score at Week 12
* Stage 9: Percentage of complete responders (UAS7 = 0) at Week 12

Conditions

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Chronic Idiopathic Urticaria

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Investigators Outcome Assessors

Study Groups

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Placebo

Participants received placebo subcutaneously every 4 weeks during the 24 week treatment period.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo was supplied as a lyophilized, sterile powder in a single-use vial without study drug.

Omalizumab 75 mg

Participants received omalizumab 75 mg subcutaneously every 4 weeks during the 24 week treatment period.

Group Type EXPERIMENTAL

Omalizumab

Intervention Type DRUG

Omalizumab was supplied as a lyophilized, sterile powder in a single-use vial.

Omalizumab 150 mg

Participants received omalizumab 150 mg subcutaneously every 4 weeks during the 24 week treatment period.

Group Type EXPERIMENTAL

Omalizumab

Intervention Type DRUG

Omalizumab was supplied as a lyophilized, sterile powder in a single-use vial.

Omalizumab 300 mg

Participants received omalizumab 300 mg subcutaneously every 4 weeks during the 24 week treatment period.

Group Type EXPERIMENTAL

Omalizumab

Intervention Type DRUG

Omalizumab was supplied as a lyophilized, sterile powder in a single-use vial.

Interventions

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Omalizumab

Omalizumab was supplied as a lyophilized, sterile powder in a single-use vial.

Intervention Type DRUG

Placebo

Placebo was supplied as a lyophilized, sterile powder in a single-use vial without study drug.

Intervention Type DRUG

Other Intervention Names

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Xolair

Eligibility Criteria

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Inclusion Criteria

* Diagnosis of Chronic Idiopathic Urticaria (CIU)/Chronic Spontaneous Urticaria (CSU) refractory to H1 antihistamines at the time of randomization.

Exclusion Criteria

* Treatment with an investigational agent within 30 days prior to screening.
* Weight \< 20 kg (44 lbs).
* Clearly defined underlying etiology for chronic urticarias other than CIU.
* Evidence of parasitic infection.
* Atopic dermatitis, bullous pemphigoid, dermatitis herpetiformis, senile pruritus, or other skin disease associated with itch.
* Previous treatment with omalizumab within a year prior to screening.
* Routine doses of the following medications within 30 days prior to screening: Systemic or cutaneous (topical) corticosteroids (prescription or over the counter), hydroxychloroquine, methotrexate, cyclosporine, or cyclophosphamide.
* Intravenous (IV) immunoglobulin G (IVIG), or plasmapheresis within 30 days prior to screening.
* Regular (daily/every other day) doxepin (oral) use within 6 weeks prior to screening.
* Any H2 antihistamine use within 7 days prior to screening.
* Any leukotriene receptor antagonist (LTRA) (montelukast or zafirlukast) within 7 days prior to screening.
* Any H1 antihistamines at greater than approved doses within 3 days prior to screening.
* Patients with current malignancy, history of malignancy, or currently under work-up for suspected malignancy except non-melanoma skin cancer that has been treated or excised and is considered resolved.
* Hypersensitivity to omalizumab or any component of the formulation.
* History of anaphylactic shock.
* Presence of clinically significant cardiovascular, neurological, psychiatric, metabolic, or other pathological conditions that could interfere with the interpretation of the study results and or compromise the safety of the patients.
* Evidence of current drug or alcohol abuse.
* Nursing women or women of childbearing potential, unless they meet the following definition of post-menopausal: 12 months of natural amenorrhea or 6 months of spontaneous amenorrhea with serum follicle-stimulating hormone (FSH) levels \> 40 mIU/mL or 6 weeks post surgical bilateral oophorectomy (with or without hysterectomy) or hysterectomy or are using one or more of the following acceptable methods of contraception: surgical sterilization, hormonal contraception, and double-barrier methods.
Minimum Eligible Age

12 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Genentech, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Clinical Trials

Role: STUDY_DIRECTOR

Genentech, Inc.

Locations

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Birmingham, Alabama, United States

Site Status

Huntington Beach, California, United States

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Long Beach, California, United States

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Los Angeles, California, United States

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Palmdale, California, United States

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Sacramento, California, United States

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San Jose, California, United States

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Studio City, California, United States

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Centennial, Colorado, United States

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Washington D.C., District of Columbia, United States

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Coral Gables, Florida, United States

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Sarasota, Florida, United States

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Tallahassee, Florida, United States

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Columbus, Georgia, United States

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Springfield, Illinois, United States

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Indianapolis, Indiana, United States

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Baltimore, Massachusetts, United States

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Boston, Massachusetts, United States

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Burlington, Massachusetts, United States

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St Louis, Missouri, United States

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Edison, New Jersey, United States

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Skillman, New Jersey, United States

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Staten Island, New York, United States

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The Bronx, New York, United States

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Durham, North Carolina, United States

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Columbus, Ohio, United States

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Toledo, Ohio, United States

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Tulsa, Oklahoma, United States

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Portland, Oregon, United States

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Pittsburgh, Pennsylvania, United States

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Upland, Pennsylvania, United States

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Charleston, South Carolina, United States

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Fort Worth, Texas, United States

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Houston, Texas, United States

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Waco, Texas, United States

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Salt Lake City, Utah, United States

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Sandy City, Utah, United States

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Springfield, Virginia, United States

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La Crosse, Wisconsin, United States

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Madison, Wisconsin, United States

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Copenhagen, , Denmark

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Odense, , Denmark

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Bordeaux, , France

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Marseille, , France

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Reims, , France

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Berlin, , Germany

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Berlin, , Germany

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Berlin, , Germany

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Dresden, , Germany

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Freiburg im Breisgau, , Germany

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Heidelberg, , Germany

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München, , Germany

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Münster, , Germany

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Perugia, , Italy

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Roma, , Italy

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Terni, , Italy

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Krakow, , Poland

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Lodz, , Poland

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Lublin, , Poland

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Wroclaw, , Poland

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Barcelona, , Spain

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Barcelona, , Spain

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Pamplona, , Spain

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Ankara, , Turkey (Türkiye)

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Bursa, , Turkey (Türkiye)

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Istanbul, , Turkey (Türkiye)

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Countries

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Russia United States Denmark France Germany Italy Poland Spain Turkey (Türkiye)

References

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Casale TB, Trzaskoma B, Holden M, Bernstein JA, Maurer M. Does angioedema in patients with chronic spontaneous urticaria impact response to omalizumab? World Allergy Organ J. 2024 Aug 5;17(8):100943. doi: 10.1016/j.waojou.2024.100943. eCollection 2024 Aug.

Reference Type DERIVED
PMID: 39193419 (View on PubMed)

Ferrer M, Gimenez-Arnau A, Saldana D, Janssens N, Balp MM, Khalil S, Risson V. Predicting Chronic Spontaneous Urticaria Symptom Return After Omalizumab Treatment Discontinuation: Exploratory Analysis. J Allergy Clin Immunol Pract. 2018 Jul-Aug;6(4):1191-1197.e5. doi: 10.1016/j.jaip.2018.04.003. Epub 2018 Apr 12.

Reference Type DERIVED
PMID: 29655772 (View on PubMed)

Goldstein S, Gabriel S, Kianifard F, Ortiz B, Skoner DP. Clinical features of adolescents with chronic idiopathic or spontaneous urticaria: Review of omalizumab clinical trials. Ann Allergy Asthma Immunol. 2017 Apr;118(4):500-504. doi: 10.1016/j.anai.2017.02.003.

Reference Type DERIVED
PMID: 28390587 (View on PubMed)

Saini SS, Omachi TA, Trzaskoma B, Hulter HN, Rosen K, Sterba PM, Courneya JP, Lackey A, Chen H. Effect of Omalizumab on Blood Basophil Counts in Patients with Chronic Idiopathic/Spontaneous Urticaria. J Invest Dermatol. 2017 Apr;137(4):958-961. doi: 10.1016/j.jid.2016.11.025. Epub 2016 Dec 6. No abstract available.

Reference Type DERIVED
PMID: 27939380 (View on PubMed)

Gimenez-Arnau AM, Spector S, Antonova E, Trzaskoma B, Rosen K, Omachi TA, Stull D, Balp MM, Murphy T. Improvement of sleep in patients with chronic idiopathic/spontaneous urticaria treated with omalizumab: results of three randomized, double-blind, placebo-controlled studies. Clin Transl Allergy. 2016 Aug 18;6:32. doi: 10.1186/s13601-016-0120-0. eCollection 2016.

Reference Type DERIVED
PMID: 27540466 (View on PubMed)

Zazzali JL, Kaplan A, Maurer M, Raimundo K, Trzaskoma B, Solari PG, Antonova E, Mendelson M, Rosen KE. Angioedema in the omalizumab chronic idiopathic/spontaneous urticaria pivotal studies. Ann Allergy Asthma Immunol. 2016 Oct;117(4):370-377.e1. doi: 10.1016/j.anai.2016.06.024. Epub 2016 Jul 14.

Reference Type DERIVED
PMID: 27424128 (View on PubMed)

Casale TB, Bernstein JA, Maurer M, Saini SS, Trzaskoma B, Chen H, Grattan CE, Gimenez-Arnau A, Kaplan AP, Rosen K. Similar Efficacy with Omalizumab in Chronic Idiopathic/Spontaneous Urticaria Despite Different Background Therapy. J Allergy Clin Immunol Pract. 2015 Sep-Oct;3(5):743-50.e1. doi: 10.1016/j.jaip.2015.04.015. Epub 2015 Jun 6.

Reference Type DERIVED
PMID: 26054553 (View on PubMed)

Saini SS, Bindslev-Jensen C, Maurer M, Grob JJ, Bulbul Baskan E, Bradley MS, Canvin J, Rahmaoui A, Georgiou P, Alpan O, Spector S, Rosen K. Efficacy and safety of omalizumab in patients with chronic idiopathic/spontaneous urticaria who remain symptomatic on H1 antihistamines: a randomized, placebo-controlled study. J Invest Dermatol. 2015 Jan;135(1):67-75. doi: 10.1038/jid.2014.306. Epub 2014 Jul 21.

Reference Type DERIVED
PMID: 25046337 (View on PubMed)

Other Identifiers

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GA00887

Identifier Type: -

Identifier Source: secondary_id

Q4881g

Identifier Type: -

Identifier Source: org_study_id