Efficacy Study of DX-88 (Ecallantide) to Treat Acute Attacks of Hereditary Angioedema (HAE)

May 14, 2021 updated by: Shire

EDEMA4: A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Assess the Efficacy and Safety of DX-88 (Ecallantide) for the Treatment of Acute Attacks of Hereditary Angioedema

The purpose of this study is to evaluate the efficacy and safety of DX-88 (ecallantide) versus placebo in the treatment of moderate to severe acute attacks of hereditary angioedema.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

This is a randomized placebo-controlled trial.

The study is designed to assess the efficacy and safety of 30 mg subcutaneous ecallantide versus placebo in the treatment of moderate to severe acute attacks of hereditary angioedema. This study is conducted under Special Protocol Assessment with the FDA and is designed to provide pivotal efficacy data on ecallantide. These data are intended to support the marketing authorization of ecallantide in the treatment of acute attacks of hereditary angioedema. Efficacy and safety of ecallantide will be evaluated in this study.

Study Type

Interventional

Enrollment (Actual)

96

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Ontario
      • Ottawa, Ontario, Canada, K1Y 4G2
        • Allergy and Asthma Research Center
      • Toronto, Ontario, Canada, M4V 1R2
        • University of Toronto
  • Jordan
      • Amman, Jordan, 1194
        • Jordan University Hospital
  • United States
    • Arizona
      • Scottsdale, Arizona, United States, 85251
        • Aaron J. Davis
    • Arkansas
      • Little Rock, Arkansas, United States, 72202
        • Arkansas Children's Hospital
      • Little Rock, Arkansas, United States, 72205
        • Little Rock Allergy & Asthma Clinic
    • California
      • Berkeley, California, United States, 94704
        • Alta Bates Comprehensive Cancer Center
      • Crescent City, California, United States, 95531
        • Pacific Coast Allergy
      • Granada Hills, California, United States, 91344
        • Jacob Offenberger
      • Los Angeles, California, United States, 90095-1680
        • UCLA David Geffen School of Medicine, Department of Medicine
    • Colorado
      • Colorado Springs, Colorado, United States, 80907
        • Asthma and Allergy Associates, P.C.
    • Delaware
      • Newark, Delaware, United States, 19718
        • Christiana Hospital
    • District of Columbia
      • Washington, District of Columbia, United States, 20007
        • Georgetown University Hospital
    • Florida
      • Miami, Florida, United States, 33136
        • University of Miami, General Clinical Research Center
      • Tampa, Florida, United States, 33613
        • University of South Florida
      • West Palm Beach, Florida, United States, 33401
        • Roberson Allergy and Asthma
    • Georgia
      • Atlanta, Georgia, United States, 30342
        • Family Allergy & Asthma Center, PC
      • Columbus, Georgia, United States, 31904
        • Allergy Center of Brookstone
    • Illinois
      • Chicago, Illinois, United States, 60612
        • University Consultants in Allergy and Immunology
    • Indiana
      • Muncie, Indiana, United States, 47304
        • Muncie Allergy Center
    • Kansas
      • Overland Park, Kansas, United States, 66210
        • Kansas City Allergy & Asthma
    • Maryland
      • Wheaton, Maryland, United States, 20902
        • Institute for Asthma and Allergy
    • Massachusetts
      • Chestnut Hill, Massachusetts, United States, 02467
        • Brigham and Women's Hospital
    • Michigan
      • Clinton Township, Michigan, United States, 48038
        • Asthma and Allergy Institute of Michigan
      • Ypsilanti, Michigan, United States, 48197
        • Respiratory Medicine Research Institute of Michigan, PLC
    • Nevada
      • Las Vegas, Nevada, United States, 89102
        • Nevada Access to Research and Education Society
      • Reno, Nevada, United States, 89503
        • University of Nevada School of Medicine
    • New Jersey
      • Newark, New Jersey, United States, 07103
        • UMDNJ-New Jersey Medical School
    • New Mexico
      • Albuquerque, New Mexico, United States, 87109
        • Allergy Partners of Albuquerque
    • New York
      • Mineola, New York, United States, 11501
        • Winthrop University Hospital
    • North Carolina
      • Asheville, North Carolina, United States, 28801
        • Allergy Partners of Western North Carolina
    • Ohio
      • Cincinnati, Ohio, United States, 45267
        • University of Cincinnati
      • Columbus, Ohio, United States, 43235
        • Optimed Research, LLC
    • Pennsylvania
      • Easton, Pennsylvania, United States, 18045
        • Valley Clinical Research Center
      • Hershey, Pennsylvania, United States, 17033
        • Penn State Milton S. Hershey Medical Center
      • Philadelphia, Pennsylvania, United States, 19107
        • Asthma Allergy and Pulmonary Associates
      • Pittsburgh, Pennsylvania, United States, 15213
        • Childrens Hospital of Pittsburgh
    • Tennessee
      • Bristol, Tennessee, United States, 37620
        • Highlands Allergy and Asthma Center, PC
    • Texas
      • Bryan, Texas, United States, 77802
        • The Paull Allergy and Asthma Clinic, P.A.
      • Dallas, Texas, United States, 75231
        • AARA Research Center
      • Galveston, Texas, United States, 77555-1083
        • University of Texas Medical School
      • Houston, Texas, United States, 77030
        • Baylor Clinic, Baylor College of Medicine
    • Utah
      • Salt Lake City, Utah, United States, 84132-2409
        • University of Utah
    • Virginia
      • Norfolk, Virginia, United States, 23507
        • Clinical Research Associates of Tidewater
    • Washington
      • Tacoma, Washington, United States, 98405
        • Puget Sound Allergy, Asthma, & Immunology

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

10 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • 10 years of age or older
  • Executed informed consent
  • Documented diagnosis of HAE (Type I or II)
  • Presentation at the site within 8 hours of patient recognition of an moderate to severe HAE acute attack

Exclusion Criteria:

  • Receipt of an investigational drug or device, within 30 days prior to study treatment
  • Receipt of non-investigational C1-INH within 7 days of treatment
  • Receipt of DX-88 (ecallantide) within 3 days prior to study treatment
  • Diagnosis of acquired angioedema (AAE), estrogen-dependent angioedema or drug-induced angioedema (including angiotensin-converting enzyme inhibitor induced angioedema)
  • Pregnancy or breastfeeding

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: DX-88 (ecallantide)
DX-88 (ecallantide) 30 mg given as three 10 mg/mL subcutaneous injections.
Drug: ecallantide
dose of 30 mg (10 mg/ml) given as 3 subcutaneous injections.
Other Names:
  • DX-88
Placebo Comparator: Placebo
Placebo, Phosphate Buffer Saline (PBS), pH 7.0 given as 3 subcutaneous injections.
Drug: Phosphate Buffer Saline (PBS), pH 7.0
given as three 1mL subcutaneous injections.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hours Post-dose
Time Frame: baseline, 4 hours post-dose
The Mean Symptom Complex Severity (MSCS) score is a validated, comprehensive point-in-time measure of symptom severity. At baseline and 4 hours, patients rated the severity on a categorical scale (0 = normal, 1 = mild, 2 = moderate, 3 = severe) for symptoms at each affected anatomical location. Ratings were averaged to obtain the MSCS score. A decrease in MSCS score reflected an improvement in symptoms; clinically meaningful improvement was indicated by a reduction in the score of 0.30 or more.
baseline, 4 hours post-dose

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Treatment Outcome Score at 4 Hours Post-Dose
Time Frame: 4 hours post-dose
Treatment Outcome Score (TOS) is a validated, comprehensive measure of symptom response to treatment. At 4 hours , patient assessment of response characterized by their change from baseline in symptom severity and collected by anatomic site of attack involvement, was recorded on a categorical scale (significant improvement [100; best value]to significant worsening [-100; worst value]). Clinically meaningful improvement was indicated by a TOS of 30 or higher.
4 hours post-dose
Patients With Significant Improvement in Overall Response
Time Frame: 4 hours post-dose
Patients were to be asked to perform an overall response assessment at intervals during the first 4 hours post-dose. Assessments were to be made relative to baseline (ie, immediately before initial dosing) using a 5-category scale. Categories were: significant improvement = "a lot better or resolved"; improvement = "a little better"; same = response unchanged; worsening = "a little worse"; significant worsening = "a lot worse". Significant improvement is the first time that a patient responded to the overall response assessment as "a lot better or resolved."
4 hours post-dose
Patients With a Successful Response at 4 Hours Post-dosing, Based on the Change From Baseline in the MSCS Score
Time Frame: baseline, 4 hours post-dosing
A successful response was defined as improvement in existing laryngeal symptom complex,stabilization of an existing peripheral symptom complex, or a change from baseline in the MSCS score at 4 hours of at least -1.0.
baseline, 4 hours post-dosing
Proportion of Patients Maintaining a Significant Improvement in Overall Response Through 24 Hours
Time Frame: 24 hours post-dosing
Maintenance of significant improvement was defined as achieving and maintaining a significant improvement in overall response through 24 hours after dosing. Patient response categories were: significant improvement = "a lot better or resolved"; improvement = "a little better"; same = response unchanged; worsening = "a little worse"; significant worsening = "a lot worse".
24 hours post-dosing

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Shire

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 1, 2007

Primary Completion (Actual)

June 1, 2008

Study Completion (Actual)

June 1, 2008

Study Registration Dates

First Submitted

April 4, 2007

First Submitted That Met QC Criteria

April 4, 2007

First Posted (Estimate)

April 5, 2007

Study Record Updates

Last Update Posted (Actual)

June 8, 2021

Last Update Submitted That Met QC Criteria

May 14, 2021

Last Verified

May 1, 2021

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • EDEMA4 (DX-88/20)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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