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Safety and Efficacy Study of Repeated Doses of DX-88 (Ecallantide) to Treat Attacks of Hereditary Angioedema (HAE)

Primary Purpose

Hereditary Angioedema (HAE)

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
ecallantide
Sponsored by
Shire
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hereditary Angioedema (HAE)

Eligibility Criteria

10 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • 10 years of age or older
  • Documented diagnosis of HAE (Type I or II)
  • Willing and able to give informed consent
  • Acute HAE attack at time of presentation

Exclusion Criteria:

  • Receipt of an investigational drug or device, within 30 days prior to study treatment, other than DX-88 (ecallantide)
  • Pregnancy or breastfeeding
  • Receipt of non-investigational C1-INH or DX-88 within 72 hours of treatment
  • Patients eligible for current, ongoing clinical trial in which DX 88 (ecallantide) is offered

Sites / Locations

  • Aaron Davis
  • Arkansas Children's Hospital
  • Little Rock Allergy & Asthma Clinic
  • Alta Bates Summit Comprehensive Cancer Center
  • Pacific Coast Allergy
  • Jacob Offenberger
  • UCLA David Geffen School of Medicine, Department of Medicine
  • Asthma and Allergy Associates, P.C.
  • Christiana Hospital, Christiana Care Health Services
  • Georgetown University Medical Center, Georgetown University Hospital
  • University of Miami, General Clinical Research Center
  • University of South Florida, Asthma, Allergy and Immunology Clinical Research Unit
  • Roberson Allergy and Asthma
  • Family Allergy & Asthma Center, PC
  • Allergy Center of Brookstone
  • University Consultants in Allergy and Immunology
  • Muncie Allergy Center
  • Kansas City Allergy & Asthma
  • Institute for Asthma and Allergy
  • Brigham and Women's Hospital
  • Asthma and Allergy Institutes of Michigan
  • Respiratory Medicine Research Institute of Michigan, PLC
  • Nevada Access to Research and Education Society
  • University of Nevada School of Medicine
  • UMDNJ- New Jersey Medical School
  • Allergy Partners of Albuquerque
  • Winthrop University Hospital
  • Allergy Partners of Western North Carolina
  • University of Cincinnati, Division of Internal Medicine
  • Optimed Research, LLC
  • Penn State Milton S Hershey Medical Center
  • Asthma Allergy and Pulmonary Associates
  • Children's Hospital of Pittsburgh
  • Highlands Allergy and Asthma Center, PC
  • The Paull Allergy and Asthma Clinic, P.A
  • AARA Research Center
  • University of Utah Health Sciences Center
  • Clinical Research Associates of Tidewater
  • Puget Sound Allergy, Asthma, & Immunology
  • Allergy and Asthma Research Centre
  • Jordan University Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

DX-88 (ecallantide)

Arm Description

DX-88 (ecallantide) Patients were treated with DX-88 (ecallantide) when they experienced an HAE attack. 30 mg dose of ecallantide given via 3 SC injections; a second 30 mg dose can be administered if needed. Patients were to be assessed until 4 hrs post-dose. Patients were asked to return for 3 follow-up visits: 7 days, 28 days and 90 days post-dose.

Outcomes

Primary Outcome Measures

Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hrs Post Dosing
Mean Symptom Complex Severity (MSCS) score is a validated point-in-time measure of symptom severity. At baseline and 4 hrs, 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.

Secondary Outcome Measures

Treatment Outcome Score (TOS) at 4 Hrs Post Dosing, Based on the Patient Assessment of Baseline Severity of Symptoms
The Treatment Outcome Score (TOS)is a validated measure of response to therapy. Response assessment for each symptom complex (internal head/neck, stomach/GI, genital/buttocks, external head/neck or cutaneous) was to be weighted based on the severity of symptom complexes at baseline. Severity assessment at baseline was rated on a categorical scale (1=mild, 2=moderate, 3=severe) for symptoms at each affected symptom complex. Response assessment of each symptom complex post-dosing relative to baseline used a scale (100=significant improvement, 50=improvement, 0=same). The weighted values were used to calculate the composite TOS. A TOS greater than 0 denotes an improvement in symptoms compared with baseline severity.
Time to Significant Improvement
Time to significant improvement in overall response based on the period from 15 minutes after dosing through 4 hrs post dosing. Significant improvement was defined as a response of "a lot better or resolved" in the overall response assessment.

Full Information

First Posted
April 4, 2007
Last Updated
May 14, 2021
Sponsor
Shire
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1. Study Identification

Unique Protocol Identification Number
NCT00456508
Brief Title
Safety and Efficacy Study of Repeated Doses of DX-88 (Ecallantide) to Treat Attacks of Hereditary Angioedema (HAE)
Official Title
Open-label Patient Continuation of DX-88 (Ecallantide) for Acute Hereditary Angioedema Attacks
Study Type
Interventional

2. Study Status

Record Verification Date
May 2021
Overall Recruitment Status
Completed
Study Start Date
April 1, 2007 (Actual)
Primary Completion Date
June 1, 2010 (Actual)
Study Completion Date
September 1, 2010 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Shire

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
The purpose of this study is to evaluate the efficacy and safety of repeated doses of ecallantide in the treatment of acute attacks of hereditary angioedema and to allow HAE patients continued access to ecallantide. In addition, patients enrolled in DX-88/20 (EDEMA4) trial will be followed up and treated for subsequent attacks in this trial.
Detailed Description
This is an open label trial. The study is designed to assess the efficacy and safety of 30 mg subcutaneous ecallantide in the treatment of acute attacks of hereditary angioedema. This study is designed to provide efficacy and safety data on repeated use of 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hereditary Angioedema (HAE)

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
147 (Actual)

8. Arms, Groups, and Interventions

Arm Title
DX-88 (ecallantide)
Arm Type
Experimental
Arm Description
DX-88 (ecallantide) Patients were treated with DX-88 (ecallantide) when they experienced an HAE attack. 30 mg dose of ecallantide given via 3 SC injections; a second 30 mg dose can be administered if needed. Patients were to be assessed until 4 hrs post-dose. Patients were asked to return for 3 follow-up visits: 7 days, 28 days and 90 days post-dose.
Intervention Type
Drug
Intervention Name(s)
ecallantide
Other Intervention Name(s)
DX-88
Intervention Description
solution for SC injection, one 30 mg dose per HAE attack
Primary Outcome Measure Information:
Title
Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hrs Post Dosing
Description
Mean Symptom Complex Severity (MSCS) score is a validated point-in-time measure of symptom severity. At baseline and 4 hrs, 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.
Time Frame
4 hrs post dose after every episode
Secondary Outcome Measure Information:
Title
Treatment Outcome Score (TOS) at 4 Hrs Post Dosing, Based on the Patient Assessment of Baseline Severity of Symptoms
Description
The Treatment Outcome Score (TOS)is a validated measure of response to therapy. Response assessment for each symptom complex (internal head/neck, stomach/GI, genital/buttocks, external head/neck or cutaneous) was to be weighted based on the severity of symptom complexes at baseline. Severity assessment at baseline was rated on a categorical scale (1=mild, 2=moderate, 3=severe) for symptoms at each affected symptom complex. Response assessment of each symptom complex post-dosing relative to baseline used a scale (100=significant improvement, 50=improvement, 0=same). The weighted values were used to calculate the composite TOS. A TOS greater than 0 denotes an improvement in symptoms compared with baseline severity.
Time Frame
4 hrs post dose after every episode
Title
Time to Significant Improvement
Description
Time to significant improvement in overall response based on the period from 15 minutes after dosing through 4 hrs post dosing. Significant improvement was defined as a response of "a lot better or resolved" in the overall response assessment.
Time Frame
15 min - 4 hrs post dose after every episode

10. Eligibility

Sex
All
Minimum Age & Unit of Time
10 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 10 years of age or older Documented diagnosis of HAE (Type I or II) Willing and able to give informed consent Acute HAE attack at time of presentation Exclusion Criteria: Receipt of an investigational drug or device, within 30 days prior to study treatment, other than DX-88 (ecallantide) Pregnancy or breastfeeding Receipt of non-investigational C1-INH or DX-88 within 72 hours of treatment Patients eligible for current, ongoing clinical trial in which DX 88 (ecallantide) is offered
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Study Director
Organizational Affiliation
Takeda
Official's Role
Study Director
Facility Information:
Facility Name
Aaron Davis
City
Scottsdale
State/Province
Arizona
ZIP/Postal Code
85251
Country
United States
Facility Name
Arkansas Children's Hospital
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72202
Country
United States
Facility Name
Little Rock Allergy & Asthma Clinic
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72205
Country
United States
Facility Name
Alta Bates Summit Comprehensive Cancer Center
City
Berkeley
State/Province
California
ZIP/Postal Code
94704
Country
United States
Facility Name
Pacific Coast Allergy
City
Crescent City
State/Province
California
ZIP/Postal Code
95531
Country
United States
Facility Name
Jacob Offenberger
City
Granada Hills
State/Province
California
ZIP/Postal Code
91344
Country
United States
Facility Name
UCLA David Geffen School of Medicine, Department of Medicine
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095-1680
Country
United States
Facility Name
Asthma and Allergy Associates, P.C.
City
Colorado Springs
State/Province
Colorado
ZIP/Postal Code
80907
Country
United States
Facility Name
Christiana Hospital, Christiana Care Health Services
City
Newark
State/Province
Delaware
ZIP/Postal Code
19718
Country
United States
Facility Name
Georgetown University Medical Center, Georgetown University Hospital
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20007
Country
United States
Facility Name
University of Miami, General Clinical Research Center
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
University of South Florida, Asthma, Allergy and Immunology Clinical Research Unit
City
Tampa
State/Province
Florida
ZIP/Postal Code
33613
Country
United States
Facility Name
Roberson Allergy and Asthma
City
West Palm Beach
State/Province
Florida
ZIP/Postal Code
33401
Country
United States
Facility Name
Family Allergy & Asthma Center, PC
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30342
Country
United States
Facility Name
Allergy Center of Brookstone
City
Columbus
State/Province
Georgia
ZIP/Postal Code
31904
Country
United States
Facility Name
University Consultants in Allergy and Immunology
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
Muncie Allergy Center
City
Muncie
State/Province
Indiana
ZIP/Postal Code
47304
Country
United States
Facility Name
Kansas City Allergy & Asthma
City
Overland Park
State/Province
Kansas
ZIP/Postal Code
66210
Country
United States
Facility Name
Institute for Asthma and Allergy
City
Wheaton
State/Province
Maryland
ZIP/Postal Code
20902
Country
United States
Facility Name
Brigham and Women's Hospital
City
Chestnut Hill
State/Province
Massachusetts
ZIP/Postal Code
02467
Country
United States
Facility Name
Asthma and Allergy Institutes of Michigan
City
Clinton Township
State/Province
Michigan
ZIP/Postal Code
48038
Country
United States
Facility Name
Respiratory Medicine Research Institute of Michigan, PLC
City
Ypsilanti
State/Province
Michigan
ZIP/Postal Code
48197
Country
United States
Facility Name
Nevada Access to Research and Education Society
City
Las Vegas
State/Province
Nevada
ZIP/Postal Code
89102
Country
United States
Facility Name
University of Nevada School of Medicine
City
Reno
State/Province
Nevada
ZIP/Postal Code
89503
Country
United States
Facility Name
UMDNJ- New Jersey Medical School
City
Newark
State/Province
New Jersey
ZIP/Postal Code
07103
Country
United States
Facility Name
Allergy Partners of Albuquerque
City
Albuquerque
State/Province
New Mexico
ZIP/Postal Code
87109
Country
United States
Facility Name
Winthrop University Hospital
City
Mineola
State/Province
New York
ZIP/Postal Code
11501
Country
United States
Facility Name
Allergy Partners of Western North Carolina
City
Asheville
State/Province
North Carolina
ZIP/Postal Code
28801
Country
United States
Facility Name
University of Cincinnati, Division of Internal Medicine
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45267
Country
United States
Facility Name
Optimed Research, LLC
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43235
Country
United States
Facility Name
Penn State Milton S Hershey Medical Center
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
Facility Name
Asthma Allergy and Pulmonary Associates
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19107
Country
United States
Facility Name
Children's Hospital of Pittsburgh
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
Facility Name
Highlands Allergy and Asthma Center, PC
City
Bristol
State/Province
Tennessee
ZIP/Postal Code
37620
Country
United States
Facility Name
The Paull Allergy and Asthma Clinic, P.A
City
Bryan
State/Province
Texas
ZIP/Postal Code
77802
Country
United States
Facility Name
AARA Research Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75231
Country
United States
Facility Name
University of Utah Health Sciences Center
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132-2409
Country
United States
Facility Name
Clinical Research Associates of Tidewater
City
Norfolk
State/Province
Virginia
ZIP/Postal Code
23507
Country
United States
Facility Name
Puget Sound Allergy, Asthma, & Immunology
City
Tacoma
State/Province
Washington
ZIP/Postal Code
98405
Country
United States
Facility Name
Allergy and Asthma Research Centre
City
Ottawa
State/Province
Ontario
ZIP/Postal Code
K1Y 4G2
Country
Canada
Facility Name
Jordan University Hospital
City
Amman
ZIP/Postal Code
1194
Country
Jordan

12. IPD Sharing Statement

Citations:
PubMed Identifier
24712435
Citation
Rubinstein E, Stolz LE, Sheffer AL, Stevens C, Bousvaros A. Abdominal attacks and treatment in hereditary angioedema with C1-inhibitor deficiency. BMC Gastroenterol. 2014 Apr 9;14:71. doi: 10.1186/1471-230X-14-71.
Results Reference
derived
PubMed Identifier
23878046
Citation
MacGinnitie AJ, Davis-Lorton M, Stolz LE, Tachdjian R. Use of ecallantide in pediatric hereditary angioedema. Pediatrics. 2013 Aug;132(2):e490-7. doi: 10.1542/peds.2013-0646. Epub 2013 Jul 22.
Results Reference
derived
PubMed Identifier
23548529
Citation
Sheffer AL, MacGinnitie AJ, Campion M, Stolz LE, Pullman WE. Outcomes after ecallantide treatment of laryngeal hereditary angioedema attacks. Ann Allergy Asthma Immunol. 2013 Mar;110(3):184-188.e2. doi: 10.1016/j.anai.2012.12.007. Epub 2013 Jan 5.
Results Reference
derived
PubMed Identifier
23548526
Citation
Li HH, Campion M, Craig TJ, Soteres DF, Riedl M, Lumry WR, MacGinnitie AJ, Shea EP, Bernstein JA. Analysis of hereditary angioedema attacks requiring a second dose of ecallantide. Ann Allergy Asthma Immunol. 2013 Mar;110(3):168-72. doi: 10.1016/j.anai.2012.12.004. Epub 2013 Jan 8.
Results Reference
derived

Learn more about this trial

Safety and Efficacy Study of Repeated Doses of DX-88 (Ecallantide) to Treat Attacks of Hereditary Angioedema (HAE)

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