Efficacy Study of DX-88 (Ecallantide) to Treat Acute Attacks of Hereditary Angioedema (HAE)
Drug: Phosphate Buffer Saline (PBS), pH 7.0
|Study Design:||Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
|Official Title:||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|
- Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hours Post-dose [ Time Frame: baseline, 4 hours post-dose ] [ Designated as safety issue: No ]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.
- Treatment Outcome Score at 4 Hours Post-Dose [ Time Frame: 4 hours post-dose ] [ Designated as safety issue: No ]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 [ Time Frame: 4 hours post-dose ] [ Designated as safety issue: No ]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 [ Time Frame: baseline, 4 hours post-dosing ] [ Designated as safety issue: No ]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 [ Time Frame: 24 hours post-dosing ] [ Designated as safety issue: No ]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".
|Study Start Date:||April 2007|
|Study Completion Date:||June 2008|
|Primary Completion Date:||June 2008 (Final data collection date for primary outcome measure)|
Experimental: DX-88 (ecallantide)
DX-88 (ecallantide) 30 mg given as three 10 mg/mL subcutaneous injections.
dose of 30 mg (10 mg/ml) given as 3 subcutaneous injections.
Other Name: 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.
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00457015
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|Study Director:||Patrick Horn, MD, PhD||Dyax Corp.|