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PI indicated departure from institution, inability to complete study.
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| Name | Class |
|---|---|
| Dyax Corp. | INDUSTRY |
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The objective of this study is to evaluate the safety and tolerability of a single Subcutaneous (SC) dose of Ecallantide in children and adolescents with Hereditary Angioedema (HAE).
This pilot study is an open-label, non-randomized, single-arm study to evaluate the tolerability and safety of a single SC administration of ecallantide in up to approximately 10 pediatric subjects with HAE during an initial acute attack. The study is planned to enroll subjects 2 through 15 years of age who present with an acute cutaneous, abdominal, or laryngeal HAE attack. No more than 3 study sites will be included until a goal of 10 patients is achieved.
After treatment for an initial attack, one additional open label treatment with ecallantide will be offered to subjects contingent upon having been treated previously and presenting with a subsequent acute cutaneous, abdominal, or laryngeal attack of HAE at least 7 days after initial treatment. Open-label treatment for a second HAE attack will continue until 10 patients have been treated for an initial attack. Safety evaluations will be performed at each subsequent ecallantide-treated attack as for the initial treated attack
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Ecallantide | Experimental | Study Medication, Dose, and Mode of Administration: Single dose of ecallantide subcutaneous dosing:
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Ecallantide subcutaneous dosing | Drug | For acute attacks of Hereditary Angioedema in children and adolescents, Ecallantide will be administered. |
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| Measure | Description | Time Frame |
|---|---|---|
| Measurement of time to symptomatic improvement from acute attacks of hereditary angioedema | Time to symptom relief. Time to minimal symptoms. An improvement assessment will also be performed at each of the following timepoints: pre -treatment, every 30 minutes for the first 2 hours and then hourly through discharge and at the 28 day clinic visit. Incidence of need for rescue medication. Incidence of worsening despite use of ecallantide. | 28 days |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Partcipants with Adverse Events | Study Partcipants will be monitored for adverse events, changes in laboratory values, physical exam, vital sign changes and ECG changes. Vital signs, including body temperature, heart rate and sitting blood pressure, will be assessed at screening, Pre-treatment, every 30 minutes for the first 2 hours and then hourly through discharge and at the 28 day clinic visit. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Mark A Davis-Lorton, MD | Winthrop University Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Winthrop-University Hosptial Clinical Trials Center | Mineola | New York | 11501 | United States |
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| ID | Term |
|---|---|
| D056829 | Hereditary Angioedema Types I and II |
| D054179 | Angioedemas, Hereditary |
| ID | Term |
|---|---|
| D000799 | Angioedema |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D014581 | Urticaria |
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| 28 Days |
| D017445 |
| Skin Diseases, Vascular |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D006969 | Hypersensitivity, Immediate |
| D006967 | Hypersensitivity |
| D007154 | Immune System Diseases |
| D000081208 | Hereditary Complement Deficiency Diseases |
| D000081207 | Primary Immunodeficiency Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007153 | Immunologic Deficiency Syndromes |