Efficacy and Safety Study of DX-2930 to Prevent Acute Angioedema Attacks in Patients With Type I and Type II HAE

NCT ID: NCT02586805

Last Updated: 2021-06-02

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

125 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-03-03

Study Completion Date

2017-04-13

Brief Summary

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This is a phase 3, multicenter, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of DX-2930 in preventing acute angioedema attacks in patients with Type I and Type II HAE.

Detailed Description

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Conditions

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Hereditary Angioedema (HAE)

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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DX-2930 300 mg every 2 weeks

300 mg DX-2930 administered every 2 weeks by subcutaneous injection.

Group Type EXPERIMENTAL

DX-2930 - 300mg/2wk

Intervention Type DRUG

300 mg DX-2930 administered every 2 weeks by subcutaneous injection.

DX-2930 300 mg every 4 weeks

300 mg DX-2930 administered every 4 weeks by subcutaneous injection

Group Type EXPERIMENTAL

DX-2930 - 300mg/4wk

Intervention Type DRUG

300 mg DX-2930 administered every 4 weeks by subcutaneous injection. To maintain the study blind, subjects will be given placebo injections every other 2 weeks when they are not receiving drug.

DX-2930 150 mg every 4 weeks

150 mg DX-2930 administered every 4 weeks by subcutaneous injection

Group Type EXPERIMENTAL

DX-2930 - 150mg/4wk

Intervention Type DRUG

150 mg DX-2930 administered every 4 weeks by subcutaneous injection. To maintain the study blind, subjects will be given placebo injections every other 2 weeks when they are not receiving drug.

Placebo

Placebo administered every 2 weeks by subcutaneous injection.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo administered every 2 weeks by subcutaneous injection.

Interventions

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DX-2930 - 300mg/2wk

300 mg DX-2930 administered every 2 weeks by subcutaneous injection.

Intervention Type DRUG

DX-2930 - 300mg/4wk

300 mg DX-2930 administered every 4 weeks by subcutaneous injection. To maintain the study blind, subjects will be given placebo injections every other 2 weeks when they are not receiving drug.

Intervention Type DRUG

DX-2930 - 150mg/4wk

150 mg DX-2930 administered every 4 weeks by subcutaneous injection. To maintain the study blind, subjects will be given placebo injections every other 2 weeks when they are not receiving drug.

Intervention Type DRUG

Placebo

Placebo administered every 2 weeks by subcutaneous injection.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Males and females 12 years of age or older at time of screening
* Documented diagnosis of HAE, Type I or II
* Baseline rate of at least 1 Investigator-confirmed HAE attack per 4 weeks
* Adult subjects and caregivers of subjects under the age of 18 are willing and able to read, understand, and sign an informed consent form. Subjects age 12 to 17, whose caregiver provides informed consent, are willing and able to read, understand an dsign an assent form.
* Males and femailes who are fertile and sexually active must adhere to contraception requirements.

Exclusion Criteria

* Concomitant diagnosis of another form of chronic, recurrent angioedema, such as acquired angioedema, idiopathic angioedema, or recurrent angioedema associated with urticaria.
* Participation in a prior DX-2930 study
* Treatment with any other investigational drug or exposure to an investigational device within 4 weeks prior screening
* Exposure to angiotensin-converting enzyme (ACE) inhibitors or any estrogen-containing medications within 4 weeks prior to screening.
* Exposure to androgens within 2 weeks prior to entering the run-in period.
* Use of long-term prophylactic therapy for HAE within 2 weeks prior to entering the run-in period.
* Use of short-term prophylaxis for HAE within 7 days prior to entering the run-in period.
* Any of the following liver function test abnormalities: alanine aminotransferase (ALT) \> 3x upper limit of normal, or aspartate aminotransferase (AST) \> 3x upper limit of normal, or total bilirubin \> 2x upper limit of normal (unless the bilirubin elevation is a result of Gilbert's syndrome).
* Pregnancy or breastfeeding.
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Dyax Corp.

INDUSTRY

Sponsor Role collaborator

Shire

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Shire Physician

Role: STUDY_DIRECTOR

Shire

Locations

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Clinical Research Center of Alabama, LLC

Birmingham, Alabama, United States

Site Status

Medical Research of Arizona

Scottsdale, Arizona, United States

Site Status

UC San Diego School of Medicine

San Diego, California, United States

Site Status

AIRE Medical of Los Angeles

Santa Monica, California, United States

Site Status

Allergy & Asthma Clinical Research

Walnut Creek, California, United States

Site Status

IMMUNOe International Health & Research Centers

Centennial, Colorado, United States

Site Status

Asthma and Allergy Associates, P.C.

Colorado Springs, Colorado, United States

Site Status

University of South Florida

Tampa, Florida, United States

Site Status

University of Kansas Medical Center

Kansas City, Kansas, United States

Site Status

Institute of Asthma & Allergy, P.C.

Chevy Chase, Maryland, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

University of Michigan Hospital and Health System

Ann Arbor, Michigan, United States

Site Status

Midwest Immunology Clinic

Plymouth, Minnesota, United States

Site Status

Washington University School of Medicine

St Louis, Missouri, United States

Site Status

Hudson-Essex Allergy, LLC

Belleville, New Jersey, United States

Site Status

Atlantic Research Center, LLC

Ocean City, New Jersey, United States

Site Status

Winthrop University Hospital

Mineola, New York, United States

Site Status

The Mount Sinai Medical Center

New York, New York, United States

Site Status

American Health Research

Charlotte, North Carolina, United States

Site Status

Duke Asthma, Allergy and Airway Center

Durham, North Carolina, United States

Site Status

Bernstein Clinical Research Center, LLC

Cincinnati, Ohio, United States

Site Status

Optimed Research, LTD

Columbus, Ohio, United States

Site Status

Toledo Institute of Clinical Research

Toledo, Ohio, United States

Site Status

Penn State Milton S. Hershey Medical Center

Hershey, Pennsylvania, United States

Site Status

Austin Regional Clinic

Austin, Texas, United States

Site Status

AARA Research Center

Dallas, Texas, United States

Site Status

Intermountain Clinical Research

Draper, Utah, United States

Site Status

Allergy Associates of Utah

Murray, Utah, United States

Site Status

Virginia Commonwealth University

Richmond, Virginia, United States

Site Status

Marycliff Allergy Specialists

Spokane, Washington, United States

Site Status

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, United States

Site Status

Ottawa Allergy Research Corporation

Ottawa, Ontario, Canada

Site Status

Gordon Sussman Clinical Research Inc.

Toronto, Ontario, Canada

Site Status

Clinique Specialisee en Allergie de la Capitale

Québec, Quebec, Canada

Site Status

Charité - University of Berlin

Berlin, , Germany

Site Status

Hautklinik und Poliklinik der Universitätsmedizin

Mainz, , Germany

Site Status

HZRM Hamophilie-Zentrum Rhein Main

Mörfelden-Walldorf, , Germany

Site Status

Hospital L. Sacco, Milan University

Milan, , Italy

Site Status

Triumpharma

Amman, , Jordan

Site Status

Sociedad Alergologica

San Juan, PR, Puerto Rico

Site Status

Barts Health NHS Trust Clinical Research Centre

London, , United Kingdom

Site Status

Countries

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United States Canada Germany Italy Jordan Puerto Rico United Kingdom

References

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Lumry WR, Maurer M, Weller K, Riedl MA, Watt M, Yu M, Devercelli G, Meunier J, Banerji A; HELP OLE Study Group. Long-term lanadelumab treatment improves health-related quality of life in patients with hereditary angioedema. Ann Allergy Asthma Immunol. 2023 Jul;131(1):101-108.e3. doi: 10.1016/j.anai.2023.03.028. Epub 2023 Apr 5.

Reference Type DERIVED
PMID: 37028510 (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)

Craig TJ, Zaragoza-Urdaz RH, Li HH, Yu M, Ren H, Juethner S, Anderson J; HELP and HELP OLE Study Investigators. Effectiveness and safety of lanadelumab in ethnic and racial minority subgroups of patients with hereditary angioedema: results from phase 3 studies. Allergy Asthma Clin Immunol. 2022 Sep 24;18(1):85. doi: 10.1186/s13223-022-00721-y.

Reference Type DERIVED
PMID: 36153561 (View on PubMed)

Banerji A, Riedl MA, Bernstein JA, Cicardi M, Longhurst HJ, Zuraw BL, Busse PJ, Anderson J, Magerl M, Martinez-Saguer I, Davis-Lorton M, Zanichelli A, Li HH, Craig T, Jacobs J, Johnston DT, Shapiro R, Yang WH, Lumry WR, Manning ME, Schwartz LB, Shennak M, Soteres D, Zaragoza-Urdaz RH, Gierer S, Smith AM, Tachdjian R, Wedner HJ, Hebert J, Rehman SM, Staubach P, Schranz J, Baptista J, Nothaft W, Maurer M; HELP Investigators. Effect of Lanadelumab Compared With Placebo on Prevention of Hereditary Angioedema Attacks: A Randomized Clinical Trial. JAMA. 2018 Nov 27;320(20):2108-2121. doi: 10.1001/jama.2018.16773.

Reference Type DERIVED
PMID: 30480729 (View on PubMed)

Provided Documents

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Document Type: Study Protocol: Original

View Document

Document Type: Study Protocol: Amendment 1

View Document

Document Type: Study Protocol: Amendment 2

View Document

Document Type: Study Protocol: Amendment 3

View Document

Document Type: Statistical Analysis Plan: Original

View Document

Document Type: Statistical Analysis Plan: Amendment 1

View Document

Other Identifiers

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2015-003943-20

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

DX-2930-03

Identifier Type: -

Identifier Source: org_study_id

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