Efficacy Study of DX-88 (Ecallantide) to Treat Acute Attacks of Hereditary Angioedema (HAE)
- Conditions
- Hereditary Angioedema (HAE)
- Interventions
- Registration Number
- NCT00457015
- Lead Sponsor
- Shire
- Brief Summary
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.
- 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 96
- 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
- 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 & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo Phosphate Buffer Saline (PBS), pH 7.0 Placebo, Phosphate Buffer Saline (PBS), pH 7.0 given as 3 subcutaneous injections. DX-88 (ecallantide) ecallantide DX-88 (ecallantide) 30 mg given as three 10 mg/mL subcutaneous injections.
- Primary Outcome Measures
Name Time Method Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hours Post-dose 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.
- Secondary Outcome Measures
Name Time Method Treatment Outcome Score at 4 Hours Post-Dose 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.
Patients With Significant Improvement in Overall Response 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."
Patients With a Successful Response at 4 Hours Post-dosing, Based on the Change From Baseline in the MSCS Score 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.
Proportion of Patients Maintaining a Significant Improvement in Overall Response Through 24 Hours 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".
Trial Locations
- Locations (45)
Kansas City Allergy & Asthma
🇺🇸Overland Park, Kansas, United States
Arkansas Children's Hospital
🇺🇸Little Rock, Arkansas, United States
University Consultants in Allergy and Immunology
🇺🇸Chicago, Illinois, United States
Nevada Access to Research and Education Society
🇺🇸Las Vegas, Nevada, United States
University of Cincinnati
🇺🇸Cincinnati, Ohio, United States
University of Utah
🇺🇸Salt Lake City, Utah, United States
Muncie Allergy Center
🇺🇸Muncie, Indiana, United States
Brigham and Women's Hospital
🇺🇸Chestnut Hill, Massachusetts, United States
Pacific Coast Allergy
🇺🇸Crescent City, California, United States
Jacob Offenberger
🇺🇸Granada Hills, California, United States
Georgetown University Hospital
🇺🇸Washington, District of Columbia, United States
Aaron J. Davis
🇺🇸Scottsdale, Arizona, United States
Alta Bates Comprehensive Cancer Center
🇺🇸Berkeley, California, United States
Little Rock Allergy & Asthma Clinic
🇺🇸Little Rock, Arkansas, United States
University of Nevada School of Medicine
🇺🇸Reno, Nevada, United States
Respiratory Medicine Research Institute of Michigan, PLC
🇺🇸Ypsilanti, Michigan, United States
Clinical Research Associates of Tidewater
🇺🇸Norfolk, Virginia, United States
UMDNJ-New Jersey Medical School
🇺🇸Newark, New Jersey, United States
Asthma and Allergy Associates, P.C.
🇺🇸Colorado Springs, Colorado, United States
Christiana Hospital
🇺🇸Newark, Delaware, United States
Allergy Center of Brookstone
🇺🇸Columbus, Georgia, United States
Puget Sound Allergy, Asthma, & Immunology
🇺🇸Tacoma, Washington, United States
UCLA David Geffen School of Medicine, Department of Medicine
🇺🇸Los Angeles, California, United States
Baylor Clinic, Baylor College of Medicine
🇺🇸Houston, Texas, United States
Optimed Research, LLC
🇺🇸Columbus, Ohio, United States
University of Miami, General Clinical Research Center
🇺🇸Miami, Florida, United States
Roberson Allergy and Asthma
🇺🇸West Palm Beach, Florida, United States
Asthma and Allergy Institute of Michigan
🇺🇸Clinton Township, Michigan, United States
Winthrop University Hospital
🇺🇸Mineola, New York, United States
Childrens Hospital of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
The Paull Allergy and Asthma Clinic, P.A.
🇺🇸Bryan, Texas, United States
Allergy and Asthma Research Center
🇨🇦Ottawa, Ontario, Canada
University of Toronto
🇨🇦Toronto, Ontario, Canada
Family Allergy & Asthma Center, PC
🇺🇸Atlanta, Georgia, United States
Institute for Asthma and Allergy
🇺🇸Wheaton, Maryland, United States
Allergy Partners of Albuquerque
🇺🇸Albuquerque, New Mexico, United States
Allergy Partners of Western North Carolina
🇺🇸Asheville, North Carolina, United States
Valley Clinical Research Center
🇺🇸Easton, Pennsylvania, United States
Penn State Milton S. Hershey Medical Center
🇺🇸Hershey, Pennsylvania, United States
Asthma Allergy and Pulmonary Associates
🇺🇸Philadelphia, Pennsylvania, United States
Highlands Allergy and Asthma Center, PC
🇺🇸Bristol, Tennessee, United States
AARA Research Center
🇺🇸Dallas, Texas, United States
University of Texas Medical School
🇺🇸Galveston, Texas, United States
Jordan University Hospital
🇯🇴Amman, Jordan
University of South Florida
🇺🇸Tampa, Florida, United States