Efficacy and Safety Study of DX-88 to Treat Acute Attacks of Hereditary Angioedema (HAE)

Phase 3CompletedNCT00262080

Shire91 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Hereditary Angioedema (HAE)

Interventions

ecallantideDRUG

dose of 30 mg (10 mg/ml) given as 3 subcutaneous injections.

Phosphate Buffer Saline (PBS),DRUG

given as three 1mL subcutaneous injections.

Eligibility

Sex: ALLMin age: 10 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Age 10 and older * Documented diagnosis of HAE, Type I or II * Executed informed consent * Presentation for treatment within 8 hours of patient recognition of moderate to severe HAE attack Exclusion Criteria: * Receipt of investigational drug or device, other than DX-88, within 30 days of treatment * Receipt of non-investigational C1-INH (C1 esterase inhibitor) within 7 days of treatment * Diagnostic of acquired angioedema, estrogen-dependent angioedema or drug induced angioedema * Pregnancy or breastfeeding * Patients who have received DX-88 within 7 days of presentation for dosing in the Double-blind Phase

Locations (1)

Institute for Asthma and Allergy

Wheaton, Maryland, United States

Outcomes

Primary Outcomes

Treatment Outcome Score at 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 \[100\] 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.

Time frame: 4 hours post-dose (DOUBLE-BLIND PART)

Secondary Outcomes

Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hours Post-dose

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 frame: baseline, 4 hours post-dose (DOUBLE-BLIND PART)

Time to Significant Improvement in Overall Response

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 little 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".

Time frame: 4 hours post-dose (DOUBLE-BLIND PART)

Data from ClinicalTrials.gov

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