CSL312 (Garadacimab) in the Prevention of Hereditary Angioedema Attacks

NCT ID: NCT04656418

Last Updated: 2023-06-29

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

64 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-01-27

Study Completion Date

2022-06-07

Brief Summary

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This is a multicenter, double-blind, randomized, placebo-controlled, parallel-arm study to investigate the efficacy and safety of subcutaneous administration of CSL312 (garadacimab) in the prophylactic treatment of hereditary angioedema.

Detailed Description

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Conditions

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Hereditary Angioedema

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

PREVENTION

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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CSL312

Participants received a CSL312 loading dose of 400 mg as two 200 mg SC injections in Month 1 along with CSL312 of 200 mg subcutaneous (SC) injections, once monthly from Months 2 to 6.

Group Type EXPERIMENTAL

CSL312

Intervention Type BIOLOGICAL

Fully human immunoglobulin G subclass 4/lambda recombinant monoclonal antibody

Placebo

Participants received a CSL312 matched loading dose of placebo as two SC injections in Month 1 along with CSL312 matched placebo SC injections, once monthly from Months 2 to 6.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Buffer without active ingredient

Interventions

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CSL312

Fully human immunoglobulin G subclass 4/lambda recombinant monoclonal antibody

Intervention Type BIOLOGICAL

Placebo

Buffer without active ingredient

Intervention Type DRUG

Other Intervention Names

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Factor XIIa inhibitor monoclonal antibody garadacimab

Eligibility Criteria

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Inclusion Criteria

* Male or female ≥ 12 years of age; diagnosed with clinically confirmed C1-INH hereditary angioedema; experience ≥ 3 attacks during the 3 months before screening.

Note: For subjects taking any prophylactic HAE therapy during the 3 months before Screening, ≥ 3 HAE attacks may be documented over 3 consecutive months before commencing the prophylactic therapy.

Exclusion Criteria

* Concomitant diagnosis of another form of angioedema such as idiopathic or acquired angioedema, recurrent angioedema associated with urticarial or hereditary angioedema type 3
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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CSL Behring

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Study Director

Role: STUDY_DIRECTOR

CSL Behring LLC

Locations

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Clinical Research Center of Alabama

Birmingham, Alabama, United States

Site Status

Medical Research of Arizona

Scottsdale, Arizona, United States

Site Status

Raffi Tachdjian MD, Inc.

Santa Monica, California, United States

Site Status

Allergy and Asthma Clinical Research

Walnut Creek, California, United States

Site Status

Institute of Asthma and Allergy

Chevy Chase, Maryland, United States

Site Status

Bernstein Clinical Research Center LLC

Cincinnati, Ohio, United States

Site Status

Pennsylvania State University

Hershey, Pennsylvania, United States

Site Status

AARA Research Center

Dallas, Texas, United States

Site Status

University of Alberta - Research Transition Facility

Edmonton, Alberta, Canada

Site Status

Ottawa Allergy Research Corp

Ottawa, Ontario, Canada

Site Status

Gordon Sussman Clinical Research Inc.

Toronto, Ontario, Canada

Site Status

Clinique specialisee en allergie de la Capitale

Québec, , Canada

Site Status

Universitätsklinikum Frankfurt Goethe-Universität

Frankfurt am Main, Hesse, Germany

Site Status

Charité Universitätsmedizin Berlin

Berlin, , Germany

Site Status

Universitätsklinikum Leipzig

Leipzig, , Germany

Site Status

CRC Clinical Research / Hautklinik und Poliklinik der Universitätsklinik Mainz

Mainz, , Germany

Site Status

HZRM Hämophilie Zentrum Rhein Main GmbH

Mörfelden-Walldorf, , Germany

Site Status

Semmelweis University

Budapest, , Hungary

Site Status

Barzilai University Medical Center

Ashkelon, , Israel

Site Status

Hiroshima University Hospital

Hiroshima, Hiroshima, Japan

Site Status

Kobe University Hospital

Kobe, Hyōgo, Japan

Site Status

St.Marianna University School of Medicine Hospital

Kawasaki-shi, Kanagawa, Japan

Site Status

Saitama Medical Center

Saitama, Kawagoe-shi, Japan

Site Status

Saga University Hospital

Saga, Saga-shi, Japan

Site Status

Koga Community Hospital

Yaizu-shi, Shizuoka, Japan

Site Status

Juntendo University Hospital

Bunkyo, Tokyo, Japan

Site Status

Saiyu Soka Hospital

Saitama, , Japan

Site Status

Amsterdam UMC, Location AMC

Amsterdam, , Netherlands

Site Status

Countries

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United States Canada Germany Hungary Israel Japan Netherlands

References

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Craig TJ, Reshef A, Li HH, Jacobs JS, Bernstein JA, Farkas H, Yang WH, Stroes ESG, Ohsawa I, Tachdjian R, Manning ME, Lumry WR, Saguer IM, Aygoren-Pursun E, Ritchie B, Sussman GL, Anderson J, Kawahata K, Suzuki Y, Staubach P, Treudler R, Feuersenger H, Glassman F, Jacobs I, Magerl M. Efficacy and safety of garadacimab, a factor XIIa inhibitor for hereditary angioedema prevention (VANGUARD): a global, multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2023 Apr 1;401(10382):1079-1090. doi: 10.1016/S0140-6736(23)00350-1. Epub 2023 Feb 28.

Reference Type DERIVED
PMID: 36868261 (View on PubMed)

Beard N, Frese M, Smertina E, Mere P, Katelaris C, Mills K. Interventions for the long-term prevention of hereditary angioedema attacks. Cochrane Database Syst Rev. 2022 Nov 3;11(11):CD013403. doi: 10.1002/14651858.CD013403.pub2.

Reference Type DERIVED
PMID: 36326435 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2020-000570-25

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CSL312_3001

Identifier Type: -

Identifier Source: org_study_id

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