Efficacy and Safety Study of DX-88 to Treat Acute Attacks of Hereditary Angioedema (HAE)
The purpose of this study is to determine if a subcutaneous dose of DX-88 (ecallantide; an investigational product) is safe and relieves symptoms of HAE in patients suffering from moderate to severe acute attacks of HAE.
Hereditary Angioedema (HAE)
Drug: Phosphate Buffer Saline (PBS),
|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:||A Double-blind, Placebo-controlled Study (72 Patients, Randomized 1:1) Followed by a Repeat-dosing Phase to Assess the Efficacy and Safety of DX-88 (Ecallantide; Recombinant Plasma Kallikrein Inhibitor) for the Treatment of Acute Attacks of Hereditary Angioedema|
- Treatment Outcome Score at 4 Hours Post-Dose [ Time Frame: 4 hours post-dose (DOUBLE-BLIND PART) ] [ 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  to significant worsening [-100]). The response at each anatomic site was weighted by baseline severity and then the weighted scores across all involved sites were averaged to calculate the TOS. Clinically meaningful improvement was indicated by a TOS of 30 or higher.
- Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hours Post-dose [ Time Frame: baseline, 4 hours post-dose (DOUBLE-BLIND PART) ] [ Designated as safety issue: No ]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 (minimally important difference) was indicated by a reduction in the score of 0.30 or more.
- Time to Significant Improvement in Overall Response [ Time Frame: 4 hours post-dose (DOUBLE-BLIND PART) ] [ Designated as safety issue: No ]The overall response assessment is a patient-reported assessment of global response to therapy. Patients are asked to perform an overall response assessment at regular intervals, relative to baseline. Patients were asked "overall how are you feeling" compared to how they felt before study drug. Answer options were "a lot worse", "a litte worse", "same", "a little better" or "a lot better or resolved". Significant improvement was the first time that the patient responded to the assessment as "a little better or resolved". The results are by the number of patients reproting.
|Study Start Date:||December 2005|
|Study Completion Date:||February 2007|
|Primary Completion Date:||December 2005 (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
Phosphate Buffer Saline (PBS), pH 7.0 given as 3 subcutaneous injections.
Drug: Phosphate Buffer Saline (PBS),
given as three 1mL subcutaneous injections.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00262080
|United States, Maryland|
|Institute for Asthma and Allergy|
|Wheaton, Maryland, United States, 20902|