Safety and Efficacy Study of Repeated Doses of DX-88 (Ecallantide) to Treat Attacks of Hereditary Angioedema (HAE)
- Registration Number
- NCT00456508
- Lead Sponsor
- Shire
- 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 147
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description DX-88 (ecallantide) ecallantide 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.
- Primary Outcome Measures
Name Time Method Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hrs Post Dosing 4 hrs post dose after every episode 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
Name Time Method Treatment Outcome Score (TOS) at 4 Hrs Post Dosing, Based on the Patient Assessment of Baseline Severity of Symptoms 4 hrs post dose after every episode 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 15 min - 4 hrs post dose after every episode 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.
Trial Locations
- Locations (41)
Aaron Davis
🇺🇸Scottsdale, Arizona, United States
Arkansas Children's Hospital
🇺🇸Little Rock, Arkansas, United States
Little Rock Allergy & Asthma Clinic
🇺🇸Little Rock, Arkansas, United States
Alta Bates Summit Comprehensive Cancer Center
🇺🇸Berkeley, California, United States
Pacific Coast Allergy
🇺🇸Crescent City, California, United States
Jacob Offenberger
🇺🇸Granada Hills, California, United States
UCLA David Geffen School of Medicine, Department of Medicine
🇺🇸Los Angeles, California, United States
Asthma and Allergy Associates, P.C.
🇺🇸Colorado Springs, Colorado, United States
Christiana Hospital, Christiana Care Health Services
🇺🇸Newark, Delaware, United States
Georgetown University Medical Center, Georgetown University Hospital
🇺🇸Washington, District of Columbia, United States
Scroll for more (31 remaining)Aaron Davis🇺🇸Scottsdale, Arizona, United States