Efficacy and Safety Study of DX-88 to Treat Acute Attacks of Hereditary Angioedema (HAE)
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ClinicalTrials.gov Identifier: NCT00262080 |
Recruitment Status :
Completed
First Posted : December 6, 2005
Results First Posted : May 11, 2010
Last Update Posted : June 11, 2021
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Hereditary Angioedema (HAE) | Drug: ecallantide Drug: Phosphate Buffer Saline (PBS), | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 91 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Quadruple (Participant, Care Provider, 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 |
Actual Study Start Date : | December 31, 2005 |
Actual Primary Completion Date : | December 31, 2005 |
Actual Study Completion Date : | February 28, 2007 |

Arm | Intervention/treatment |
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Experimental: DX-88 (ecallantide)
DX-88 (ecallantide) 30 mg given as three 10 mg/mL subcutaneous injections.
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Drug: ecallantide
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.
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Drug: Phosphate Buffer Saline (PBS),
given as three 1mL subcutaneous injections. |
- Treatment Outcome Score at 4 Hours Post-Dose [ Time Frame: 4 hours post-dose (DOUBLE-BLIND PART) ]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 [ Time Frame: baseline, 4 hours post-dose (DOUBLE-BLIND PART) ]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) ]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".
- Number of Patients With Symptom Complexes of Treated Attack at Baseline(DOUBLE-BLIND PART) [ Time Frame: Baseline ]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) ]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) ]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) ]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)

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Ages Eligible for Study: | 10 Years and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00262080
United States, Maryland | |
Institute for Asthma and Allergy | |
Wheaton, Maryland, United States, 20902 |
Study Director: | Study Director | Takeda |
Responsible Party: | Shire |
ClinicalTrials.gov Identifier: | NCT00262080 |
Other Study ID Numbers: |
EDEMA3 (DX-88/14) |
First Posted: | December 6, 2005 Key Record Dates |
Results First Posted: | May 11, 2010 |
Last Update Posted: | June 11, 2021 |
Last Verified: | June 2021 |
Angioedema Angioedemas, Hereditary Ecallantide Vascular Diseases Cardiovascular Diseases Urticaria Skin Diseases, Vascular Skin Diseases Hypersensitivity, Immediate Hypersensitivity Immune System Diseases Hereditary Complement Deficiency Diseases |
Primary Immunodeficiency Diseases Genetic Diseases, Inborn Immunologic Deficiency Syndromes Analgesics Sensory System Agents Peripheral Nervous System Agents Physiological Effects of Drugs Anti-Inflammatory Agents, Non-Steroidal Analgesics, Non-Narcotic Anti-Inflammatory Agents Antirheumatic Agents |