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

Hereditary Angioedema (HAE) Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00262080
• Other study ID #: EDEMA3 (DX-88/14)
• Status: Completed
• Phase: Phase 3
• Start date: December 31, 2005
• Est. completion date: February 28, 2007

Study information

• Verified date: June 2021
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 91
• Est. completion date: February 28, 2007
• Est. primary completion date: December 31, 2005
• Accepts healthy volunteers: No
• Gender: All
• Age group: 10 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Angioedema
• Hereditary Angioedema (HAE)

Intervention

Drug:
• ecallantide
dose of 30 mg (10 mg/ml) given as 3 subcutaneous injections.
• Phosphate Buffer Saline (PBS),
given as three 1mL subcutaneous injections.

Locations

Country	Name	City	State
United States	Institute for Asthma and Allergy	Wheaton	Maryland

Sponsors (1)

Lead Sponsor	Collaborator
Shire

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Number of Patients With Symptom Complexes of Treated Attack at Baseline(DOUBLE-BLIND PART)	Patient-reported severity of symptom complexes at baseline, by symptom complex and treatment group. Patients were to have at least one symptom complex that was moderate or severe. Patients could present with multiple symptom complexes, some of which could be mild. Mild=noticeable but do not impact daily living activities; Moderate=treatment or intervention is highly desirable and activities of daily living are impacted; Severe=require treatment or intervention due to inability to perform activities of daily living. The results are for number of patients with symptom complexes including mild, moderate and severe, provided the patients have at least one symptom complex that was moderate or severe	Baseline
Other	Treatment Outcome Score at 4 Hours Post-Dose Over Multiple Treatment Episodes	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.	4 hours post-dose (REPEAT-DOSING PART)
Other	Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hours Post-dose Over Multiple Treatment Episodes	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.	baseline, 4 hours post-dose (REPEAT-DOSING PART)
Other	Time to Significant Improvement in Overall Response Over Multiple Treatment Episodes	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 (ie,immediately before treatment) using the following 5-category scale from significant improvement (Score = 100)to significant worsening (Score = -100)	4 hours post-dose (REPEAT-DOSING PART)
Primary	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.	4 hours post-dose (DOUBLE-BLIND PART)
Secondary	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.	baseline, 4 hours post-dose (DOUBLE-BLIND PART)
Secondary	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".	4 hours post-dose (DOUBLE-BLIND PART)