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

This study has been completed.
Sponsor:
Information provided by:
Shire
ClinicalTrials.gov Identifier:
NCT00262080
First received: December 5, 2005
Last updated: August 1, 2016
Last verified: August 2016

December 5, 2005
August 1, 2016
December 2005
December 2005   (final data collection date for primary outcome measure)
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 [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.
Treatment outcome score at 4 hours as determined by patient reported outcome
Complete list of historical versions of study NCT00262080 on ClinicalTrials.gov Archive Site
  • 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 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".
  • Change in symptom severity at 4 hours
  • Time to onset of significant improvement
  • Number of Patients With Symptom Complexes of Treated Attack at Baseline(DOUBLE-BLIND PART) [ Time Frame: Baseline ] [ Designated as safety issue: No ]
    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
  • Treatment Outcome Score at 4 Hours Post-Dose Over Multiple Treatment Episodes [ Time Frame: 4 hours post-dose (REPEAT-DOSING 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 [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.
  • Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hours Post-dose Over Multiple Treatment Episodes [ Time Frame: baseline, 4 hours post-dose (REPEAT-DOSING PART) ] [ 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.
  • Time to Significant Improvement in Overall Response Over Multiple Treatment Episodes [ Time Frame: 4 hours post-dose (REPEAT-DOSING 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 (ie,immediately before treatment) using the following 5-category scale from significant improvement (Score = 100)to significant worsening (Score = -100)
Not Provided
 
Efficacy and Safety Study of DX-88 to Treat Acute Attacks of Hereditary Angioedema (HAE)
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
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.
Not Provided
Interventional
Phase 3
Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Hereditary Angioedema (HAE)
  • Drug: ecallantide
    dose of 30 mg (10 mg/ml) given as 3 subcutaneous injections.
    Other Name: DX-88
  • Drug: Phosphate Buffer Saline (PBS),
    given as three 1mL subcutaneous injections.
  • Experimental: DX-88 (ecallantide)
    DX-88 (ecallantide) 30 mg given as three 10 mg/mL subcutaneous injections.
    Intervention: Drug: ecallantide
  • Placebo Comparator: Placebo
    Phosphate Buffer Saline (PBS), pH 7.0 given as 3 subcutaneous injections.
    Intervention: Drug: Phosphate Buffer Saline (PBS),

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
91
February 2007
December 2005   (final data collection date for primary outcome measure)

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
Both
10 Years and older   (Child, Adult, Senior)
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT00262080
EDEMA3 (DX-88/14)
Yes
Not Provided
Not Provided
Bill Pullman, MD, PhD, Executive Vice President, Chief Development Officer, Dyax Corp.
Shire
Not Provided
Not Provided
Shire
August 2016

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP