Clinical Trial: Study to Assess the Tolerability and Safety of Ecallantide in Children and Adolescents With Hereditary Angioedema
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: A Multicenter, Open-Label Study to Assess the Tolerability and Safety of a Single, Subcutaneous Administration of Ecallantide in Children and Adolescents With Hereditary Angioede
Brief Summary: 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).
Detailed Summary:
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
Sponsor: Mark Davis-Lorton, MD
Current Primary Outcome: Measurement of time to symptomatic improvement from acute attacks of hereditary angioedema [ Time Frame: 28 days ]
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.
Original Primary Outcome: Measurement of time to symptomatic improvement from acute attacks of hereditary angioedema [ Time Frame: 28 days ]
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 worstening despite use of ecallantide.
Current Secondary Outcome: Number of Partcipants with Adverse Events [ Time Frame: 28 Days ]
Original Secondary Outcome: Same as current
Information By: Winthrop University Hospital
Dates:
Date Received: April 4, 2013
Date Started: August 2013
Date Completion: December 2017
Last Updated: January 3, 2017
Last Verified: January 2017
