Clinical Trial: Efficacy Study of DX-88 (Ecallantide) to Treat Acute Attacks of Hereditary Angioedema (HAE)

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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 Heredita

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 Summary:

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.

Current Primary Outcome: Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hours Post-dose [ Time Frame: baseline, 4 hours post-dose ]

Original Primary Outcome: Change in symptom severity

Current Secondary Outcome:

• Treatment Outcome Score at 4 Hours Post-Dose [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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".

Original Secondary Outcome:

• Patient reported symptom change
• Time to improvement

Dates:
Date Received: April 4, 2007
Date Started: April 2007
Date Completion:
Last Updated: June 28, 2016
Last Verified: June 2016