NCT03580356

Brief Summary

The purpose of this study was to establish efficacy and safety of ligelizumab in adolescent and adult subjects with CSU who remained symptomatic despite standard of care treatment by demonstrating better efficacy over omalizumab and over placebo. The study population consisted of 1,079 male and female subjects aged ≥ 12 years who were diagnosed with CSU and who remained symptomatic despite the use of H1-antihistamines. This was a multi-center, randomized, double-blind, active- and placebo-controlled, parallel-group study. There was a screening period of up to 28 days, a 52 week double-blind treatment period, and a 12 week post-treatment follow-up period.

Trial Health

98
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Strong global presence with extensive site network
Enrollment
1,078

participants targeted

Target at P75+ for phase_3

Timeline
Completed

Started Oct 2018

Typical duration for phase_3

Geographic Reach
26 countries

172 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

June 26, 2018

Completed
13 days until next milestone

First Posted

Study publicly available on registry

July 9, 2018

Completed
3 months until next milestone

Study Start

First participant enrolled

October 20, 2018

Completed
2.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 22, 2021

Completed
12 months until next milestone

Study Completion

Last participant's last visit for all outcomes

June 14, 2022

Completed
1.8 years until next milestone

Results Posted

Study results publicly available

March 25, 2024

Completed
Last Updated

January 7, 2025

Status Verified

January 1, 2025

Enrollment Period

2.7 years

First QC Date

June 26, 2018

Results QC Date

December 14, 2022

Last Update Submit

January 6, 2025

Conditions

Keywords

Anti-IgECSUChronic Spontaneous Urticariahives severity scoreitch severity scoreurticaria activity score

Outcome Measures

Primary Outcomes (2)

  • Mean Change From Baseline in UAS7 at Week 12 (Multiple Imputation) of Adult Subjects

    The Urticaria Activity Score (UAS) is sum of the Hive Severity Score (HSS) and the Itch Severity Score (ISS). UAS7 is sum of the HSS7 and the ISS7 scores. Possible range of weekly UAS7 score is 0 to 42. Complete UAS7 response is UAS7 = 0. Hives Severity Score (HSS) scale is 0 to 3. A weekly score (HSS7) is derived by adding up the average daily scores of the 7 days preceding the visit. Possible range of the weekly score is therefore 0 to 21. Hives Severity Score scale: 0 - None 1 - Mild (1-6 hives/12 hours) 2 - Moderate (7-12 hives/12 hours) 3 - Severe (\>12 hives/12 hours). Itch Severity Score (ISS) scale is 0 to 3. Score (ISS7) is derived by adding up average daily scores of 7 days preceding visit. Possible range of weekly score is therefore 0 to 21. Itch Severity Score scale: 0 - None 1 - Mild (minimal awareness, easily tolerated) 2 - Moderate (definite awareness, bothersome but tolerable) 3 - Severe (difficult to tolerate). Negative change from baseline indicates improvement

    Baseline, Week 12

  • Mean Change From Baseline in UAS7 at Week 12 (Observed Data) of Adolescent Subjects (FAS)

    The Urticaria Activity Score (UAS) is sum of the Hive Severity Score (HSS) and the Itch Severity Score (ISS). UAS7 is sum of the HSS7 and the ISS7 scores. Possible range of weekly UAS7 score is 0 to 42. Complete UAS7 response is UAS7 = 0. Hives Severity Score (HSS) scale is 0 to 3. A weekly score (HSS7) is derived by adding up the average daily scores of the 7 days preceding the visit. Possible range of the weekly score is therefore 0 to 21. Hives Severity Score scale: 0 - None 1 - Mild (1-6 hives/12 hours) 2 - Moderate (7-12 hives/12 hours) 3 - Severe (\>12 hives/12 hours). Itch Severity Score (ISS) scale is 0 to 3. Score (ISS7) is derived by adding up average daily scores of 7 days preceding visit. Possible range of weekly score is therefore 0 to 21. Itch Severity Score scale: 0 - None 1 - Mild (minimal awareness, easily tolerated) 2 - Moderate (definite awareness, bothersome but tolerable) 3 - Severe (difficult to tolerate). Negative change from baseline indicates improvement

    Baseline, Week 12

Secondary Outcomes (6)

  • Number and Percentage of Subjects With UAS7=0 Response at Week 12 (Multiple Imputation - Adults, Observed Data for Adolescents)

    Week 12

  • Mean Change From Baseline in ISS7 at Week 12 (Multiple Imputation) of Adult Subjects (FAS)

    Baseline, Week 12

  • Mean Change From Baseline in ISS7 at Week 12 (Observed Data) of Adolescent Subjects, (FAS)

    Baseline, Week 12

  • Number and Percentage of Participants With DLQI Score of 0 - 1 at Week 12 (Multiple Imputation - Adults, Observed Data for Adolescents)

    Week 12

  • Cumulative Number of Weeks of AAS7=0 up to Week 12 (Multiple Imputation) of Adult Subjects (FAS)

    Baseline, Week 12

  • +1 more secondary outcomes

Study Arms (4)

Ligelizumab 120 mg

EXPERIMENTAL

Ligelizumab 120 mg arm: 1 injection of 1.0 mL ligelizumab + 1 injection of 1.0 mL ligelizumab placebo q4w

Biological: Ligelizumab

Ligelizumab 72 mg

EXPERIMENTAL

Ligelizumab 72 mg arm: 1 injection of 0.6 mL ligelizumab + 1 injection of 1.0 mL ligelizumab placebo q4w

Biological: Ligelizumab

Omalizumab 300 mg

ACTIVE COMPARATOR

Omalizumab 300 mg arm: 2 injections of 1.2 mL omalizumab q4w

Biological: Omalizumab

Placebo

PLACEBO COMPARATOR

Placebo-ligelizumab arm: 2 injections of 1.0mL of ligelizumab placebo from Week 0 through Week 20; 1 injection of 1.0mL of ligelizumab 120 mg + 1 injection of 1.0 mL ligelizumab placebo from Week 24 through Week 48

Other: Placebo

Interventions

LigelizumabBIOLOGICAL

Liquid in vial

Ligelizumab 120 mgLigelizumab 72 mg
OmalizumabBIOLOGICAL

Lyophilized powder for solution in vial

Omalizumab 300 mg
PlaceboOTHER

Liquid in vial

Placebo

Eligibility Criteria

Age12 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Signed informed consent must be obtained prior to participation in the study. The subject's, parent's or legal guardian's signed written informed consent and child's assent, if appropriate, must be obtained before any 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 Informed Consent Form (ICF) at the next study visit.
  • Male and female subjects ≥ 12 years of age at the time of screening.
  • CSU diagnosis for ≥ 6 months.
  • Diagnosis of CSU refractory to H1-AH at approved doses at the time of randomization, as defined by all of the following:
  • The presence of itch and hives for ≥ 6 consecutive weeks at any time prior to Visit 1 (Day - 28 to Day -14) despite current use of non-sedating H1-antihistamine
  • UAS7 score (range 0-42) ≥ 16 and HSS7 (range 0-21) ≥ 8 during the 7 days prior to randomization (Visit 110, Day 1)
  • Subjects must be on H1-antihistamine at only locally label approved doses for treatment of CSU starting at Visit 1 (Day -28 to Day -14)
  • Willing and able to complete a daily symptom eDiary for the duration of the study and adhere to the study visit schedules.

You may not qualify if:

  • History of hypersensitivity to any of the study drugs or their excipients or to drugs of similar chemical classes (i.e. to murine, chimeric or human antibodies).
  • Subjects having a clearly defined cause of their chronic urticaria, other than CSU. This includes, but is not limited to, the following: symptomatic dermographism (urticaria factitia), cold-, heat-, solar-, pressure-, delayed pressure-, aquagenic-, cholinergic- or contact-urticaria.
  • Diseases, other than chronic urticaria, with urticarial or angioedema symptoms such as urticarial vasculitis, erythema multiforme, cutaneous mastocytosis (urticaria pigmentosa) and hereditary or acquired angioedema (eg, due to C1 inhibitor deficiency).
  • Subjects with evidence of helminthic parasitic infection as evidenced by stools being positive for a pathogenic organism according to local guidelines. All subjects will be screened at Visit 1. If stool testing is positive for pathogenic organism, the subject will not be randomized and will not be allowed to rescreen.
  • Any other skin disease associated with chronic itching that might influence in the investigators opinion the study evaluations and results (e.g. atopic dermatitis, bullous pemphigoid, dermatitis herpetiformis, senile pruritus, etc.).
  • Prior exposure to ligelizumab or omalizumab.
  • H1-AH used as background medication at greater than locally label-approved doses after visit 1

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (183)

Allervie Clinical Research

Birmingham, Alabama, 35209, United States

Location

Medical Resch of Arizona-Div of Allergy Asthma and Immunology

Scottsdale, Arizona, 85251, United States

Location

Atria Clinical Research Asthma and Allergy Institute

Little Rock, Arkansas, 72209, United States

Location

DeMera Allergy Asthma and Immun Ctr

Fresno, California, 93720, United States

Location

California Allergy and Asthma Medical Group

Los Angeles, California, 90025, United States

Location

Jonathan Corren Inc

Los Angeles, California, 90025, United States

Location

Allergy and Asthma Consultants

Redwood City, California, 94063, United States

Location

Allergy and Asthma Associates of Santa Clara Vally Center

San Jose, California, 95117, United States

Location

Sarasota Clinical Research

Sarasota, Florida, 34233, United States

Location

Olympian Clinical Research

Tampa, Florida, 33609, United States

Location

Idaho Research

Eagle, Idaho, 83616, United States

Location

Midwest Allergy Sinus Asthma SC

Normal, Illinois, 61761, United States

Location

John Hopkins University

Baltimore, Maryland, 21204, United States

Location

Institute for Asthma and Allergy PC

Chevy Chase, Maryland, 20815, United States

Location

Chesapeake Clinical Research

White Marsh, Maryland, 21162, United States

Location

Mayo Clinic Rochester

Rochester, Minnesota, 55905, United States

Location

Midwest Clinical Research LcLC

St Louis, Missouri, 63141, United States

Location

Montana Medical Research

Missoula, Montana, 59808, United States

Location

The Asthma and Allergy Center PC

Papillion, Nebraska, 68046, United States

Location

Icahn School Of Med At Mount Sinai

New York, New York, 10029, United States

Location

Toledo Institute of Clinical Research

Toledo, Ohio, 43617, United States

Location

Allergy Asthma and Clinical Research

Oklahoma City, Oklahoma, 73120, United States

Location

PCR DBA Columbia Asthma and Allergy

Clackamas, Oregon, 97015, United States

Location

Allergy and Asthma Specialists PC

Blue Bell, Pennsylvania, 19422, United States

Location

Allergy and Clinical Immunology Associates

Pittsburgh, Pennsylvania, 15241, United States

Location

National Allergy and Asthma Research LLS

North Charleston, South Carolina, 29420, United States

Location

Bellaire Dermatology Associates

Bellaire, Texas, 77401, United States

Location

Western Sky Medical Research

El Paso, Texas, 79924, United States

Location

North Texas Inst For Clinical Tri

Fort Worth, Texas, 76132, United States

Location

Allergy and Asthma Research Center PA

San Antonio, Texas, 78229, United States

Location

Quality Assurance Research Center

San Antonio, Texas, 78230, United States

Location

Allergy and Asthma Care of Waco

Waco, Texas, 76712, United States

Location

Allergy Associates of Utah

Sandy City, Utah, 84093, United States

Location

Bellingham Asthma Allergy and Immunology

Bellingham, Washington, 98225, United States

Location

Novartis Investigative Site

CABA, Buenos Aires, C1181ACH, Argentina

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Novartis Investigative Site

CABA, Buenos Aires, C1414AIF, Argentina

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Novartis Investigative Site

Mendoza, Mendoza Province, M5500AWD, Argentina

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Santa Fe, Rosario, S2000DBS, Argentina

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Rosario, Santa Fe Province, S2000BRH, Argentina

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Rosario, Santa Fe Province, S2000JKR, Argentina

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Novartis Investigative Site

Buenos Aires, C1425DKG, Argentina

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CABA, 1035, Argentina

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Salta, 4400, Argentina

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Adelaide, South Australia, 5000, Australia

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East Melbourne, Victoria, 3002, Australia

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Parkville, Victoria, 3050, Australia

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Brussels, 1070, Belgium

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Brussels, 1090, Belgium

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Brussels, 1200, Belgium

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Ghent, 9000, Belgium

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Leuven, 3000, Belgium

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Loverval, 6280, Belgium

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Salvador, Estado de Bahia, 40110-060, Brazil

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Rio de Janeiro, Rio de Janeiro, 21941-913, Brazil

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Alphaville Barueri, São Paulo, 06454010, Brazil

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São José do Rio Preto, São Paulo, 15090 000, Brazil

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São Paulo, São Paulo, 05437 010, Brazil

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Vitacura, Santiago Metropolitan, 7640881, Chile

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Osorno, 5311297, Chile

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Santiago, 8420383, Chile

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Tallinn, 10138, Estonia

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Tartu, 50406, Estonia

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Helsinki, 00180, Finland

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Clermont-Ferrand, 63003, France

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Grenoble, 38043, France

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Nice, 06202, France

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Paris, 75970, France

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Rouen, 76031, France

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Bad Bentheim, 48455, Germany

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Dresden, 01307, Germany

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Düsseldorf, 40225, Germany

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Frankfurt, 60590, Germany

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Gera, 07548, Germany

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Göttingen, 37075, Germany

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Halle S, 06120, Germany

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Hamburg, 22303, Germany

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Hamburg, 22391, Germany

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Hanover, 30625, Germany

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Heidelberg, 69120, Germany

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Leipzig, 04103, Germany

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München, 80377, Germany

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Osnabrück, 49074, Germany

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Quedlinburg, 06484, Germany

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Simmern, 55469, Germany

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Stade, 21682, Germany

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Stuttgart, 70178, Germany

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Tübingen, 72076, Germany

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Bangalore, Karnataka, 560004, India

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Mangalore, Karnataka, 575002, India

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Nashik, Maharashtra, 422 101, India

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New Delhi, National Capital Territory of Delhi, 110 060, India

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Bikaner, Rajasthan, 334001, India

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Afula, 1834111, Israel

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Haifa, 3339419, Israel

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Jerusalem, 9112001, Israel

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Kfar Saba, 44281, Israel

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Ramat Gan, 52621, Israel

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Rehovot, 7610001, Israel

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Cagliari, CA, 09042, Italy

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Catania, CT, 95123, Italy

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Florence, FI, 50122, Italy

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Florence, FI, 50134, Italy

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Rozzano, MI, 20089, Italy

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Modena, MO, 41124, Italy

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Siena, SI, 53100, Italy

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Nagoya, Aichi-ken, 454-0012, Japan

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Chikushino-shi, Fukuoka, 818 0083, Japan

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Amagasaki, Hyōgo, 660 8550, Japan

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Kawasaki, Kanagawa, 211-0063, Japan

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Yokohama, Kanagawa, 220-6208, Japan

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Yokohama, Kanagawa, 221-0825, Japan

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Kamimashi-gun, Kumamoto, 861-3106, Japan

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Sakai, Osaka, 593-8324, Japan

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Izumo, Shimane, 693 8501, Japan

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Itabashi-ku, Tokyo, 173-8610, Japan

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Machida, Tokyo, 194-0013, Japan

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Setagaya-ku, Tokyo, 158-0097, Japan

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Shinagawa Ku, Tokyo, 141 8625, Japan

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Fukuoka, 819 0167, Japan

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Hiroshima, 734-8551, Japan

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Kobe, 650-0017, Japan

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Novartis Investigative Site

Beirut, 166378, Lebanon

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El Achrafiyé, 166830, Lebanon

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Saida, 652, Lebanon

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Guadalajara, Jalisco, 44130, Mexico

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Villahermosa, Tabasco, 86035, Mexico

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Rotterdam, South Holland, 3015 GD, Netherlands

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Amersfroort, 3818 ES, Netherlands

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Breda, 4818 CK, Netherlands

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Utrecht, 3584CX, Netherlands

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Lipa City, Batangas, 4217, Philippines

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City of Taguig, National Capital Region, 1634, Philippines

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Makati City, 1220, Philippines

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Pasig, 1605, Philippines

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Quezon, 1102, Philippines

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Bydgoszcz, 85-094, Poland

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Gdansk, 80-214, Poland

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Krosno, 38-400, Poland

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Lodz, 90-153, Poland

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Lodz, 90-436, Poland

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Ossy, 42 624, Poland

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Sopot, 81 756, Poland

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Warsaw, 02-507, Poland

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Bucharest, District 2, 020762, Romania

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Brasov, 500283, Romania

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Cluj-Napoca, 400162, Romania

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Craiova, 200642, Romania

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Iași, 700381, Romania

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Chelyabinsk, 454048, Russia

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Kazan', 420012, Russia

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Moscow, 115478, Russia

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Novartis Investigative Site

Saint Petersburg, 194223, Russia

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Novartis Investigative Site

Saint Petersburg, 195112, Russia

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Novartis Investigative Site

Saint Petersburg, 197198, Russia

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Stavropol, 355000, Russia

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Kežmarok, 060 01, Slovakia

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Novartis Investigative Site

Komárno, 945 01, Slovakia

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Levice, 934 01, Slovakia

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Nové Zámky, 940 34, Slovakia

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Považská Bystrica, 017 26, Slovakia

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Svidník, 08901, Slovakia

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Topoľčany, 95501, Slovakia

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Žilina, 010 01, Slovakia

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Córdoba, Andalusia, 14004, Spain

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Granada, Andalusia, 18014, Spain

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Barcelona, Catalonia, 08035, Spain

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Alcorcón, Madrid, 28922, Spain

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Pozuelo de Alarcón, Madrid, 28223, Spain

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Pamplona, Navarre, 31008, Spain

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San Cristóbal de La Laguna, Santa Cruz De Tenerife, 38320, Spain

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Valencia, Valencia, 46014, Spain

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Valencia, Valencia, 46015, Spain

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Madrid, 28041, Spain

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Valencia, 46026, Spain

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Taichung, 407219, Taiwan

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Taipei, 10002, Taiwan

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Taoyuan District, 33305, Taiwan

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Tunis, Tunisie, 1007, Tunisia

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Sfax, Tunusia, 3029, Tunisia

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Sousse, 4000, Tunisia

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London, SE1 7EH, United Kingdom

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Hanoi, 100000, Vietnam

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Novartis Investigative Site

Ho Chi Minh City, 7000, Vietnam

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Related Publications (1)

  • Maurer M, Ensina LF, Gimenez-Arnau AM, Sussman G, Hide M, Saini S, Grattan C, Fomina D, Rigopoulos D, Berard F, Canonica GW, Rockmann H, Irani C, Szepietowski JC, Leflein J, Bernstein JA, Peter JG, Kulthanan K, Godse K, Ardusso L, Ukhanova O, Staubach P, Sinclair R, Gogate S, Thomsen SF, Tanus T, Ye YM, Burciu A, Barve A, Modi D, Scosyrev E, Hua E, Letzelter K, Varanasi V, Patekar M, Severin T; PEARL-1 and PEARL-2 trial investigators. Efficacy and safety of ligelizumab in adults and adolescents with chronic spontaneous urticaria: results of two phase 3 randomised controlled trials. Lancet. 2024 Jan 13;403(10422):147-159. doi: 10.1016/S0140-6736(23)01684-7. Epub 2023 Nov 23.

MeSH Terms

Conditions

Chronic Urticaria

Interventions

ligelizumabOmalizumab

Condition Hierarchy (Ancestors)

UrticariaSkin Diseases, VascularSkin DiseasesSkin and Connective Tissue DiseasesHypersensitivity, ImmediateHypersensitivityImmune System DiseasesChronic DiseaseDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Antibodies, Anti-IdiotypicAntibodiesImmunoglobulinsImmunoproteinsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsAntibodies, Monoclonal, HumanizedAntibodies, MonoclonalSerum GlobulinsGlobulins

Results Point of Contact

Title
Study Director
Organization
Novartis Pharmaceuticals

Study Officials

  • Novartis Pharmaceuticals

    Novartis Pharmaceuticals

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
TRIPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR
Masking Details
Patients, 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. The study drug was prepared by an independent unblinded pharmacist (or authorized delegate) and administered by an independent unblinded study drug administrator. Neither the unblinded pharmacist nor the unblinded study drug administrator was involved in any assessments.
Purpose
TREATMENT
Intervention Model
PARALLEL
Model Details: This was a Phase III multi-center, randomized, double-blind, active- and placebo-controlled, parallel-group study. There was a screening period of up to 28 days, a 52 week double-blind treatment period, and a 12 week post-treatment follow-up period.
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 26, 2018

First Posted

July 9, 2018

Study Start

October 20, 2018

Primary Completion

June 22, 2021

Study Completion

June 14, 2022

Last Updated

January 7, 2025

Results First Posted

March 25, 2024

Record last verified: 2025-01

Data Sharing

IPD Sharing
Will share

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

More information

Locations