Safety and Efficacy Study of Repeated Doses of DX-88 (Ecallantide) to Treat Attacks of Hereditary Angioedema (HAE)
|ClinicalTrials.gov Identifier: NCT00456508|
Recruitment Status : Completed
First Posted : April 5, 2007
Results First Posted : December 28, 2012
Last Update Posted : February 20, 2018
|Condition or disease||Intervention/treatment||Phase|
|Hereditary Angioedema (HAE)||Drug: ecallantide||Phase 3|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||147 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Open-label Patient Continuation of DX-88 (Ecallantide) for Acute Hereditary Angioedema Attacks|
|Study Start Date :||April 1, 2007|
|Actual Primary Completion Date :||June 1, 2010|
|Actual Study Completion Date :||September 1, 2010|
Experimental: DX-88 (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.
solution for SC injection, one 30 mg dose per HAE attack
Other Name: DX-88
- Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hrs Post Dosing [ Time Frame: 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.
- Treatment Outcome Score (TOS) at 4 Hrs Post Dosing, Based on the Patient Assessment of Baseline Severity of Symptoms [ Time Frame: 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 [ Time Frame: 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.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00456508
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|Study Director:||Bill Pullman, MD, PhD||Dyax Corp.|