Clinical Trial: C1-INH Compared to Placebo at the Time of Prodromal Symptoms for Hereditary Angioedema (HAE) Exacerbation

Study Status: Withdrawn
Recruit Status: Withdrawn
Study Type: Interventional

Official Title: Randomized, Double-blind, Placebo-controlled, Dose-finding Study to Determine the Efficacy of 1000u, and 1500u of C1-INH Compared to Placebo at the Time of Prodromal Symptoms

Brief Summary: The study hypothesis is that treatment of Hereditary Angioedema at the time of prodromal symptoms will decrease morbidity associated with the disease

Detailed Summary:

Section 3 Study Design and Methods:

Our proposal is to perform a randomized double-blind, three-arm, three-way cross-over study in which subjects with HAE would be placed into one of three different treatment groups based on computer randomization. Subjects will be randomized to receive placebo, 1000 units, or 1500 units of C1-INH at the onset of prodromal symptoms although they would be blinded to which one they were receiving. Randomization will be to one of the two doses or placebo and the sequence of further treatments will be determined randomly in a double-blinded fashion by a person who is not involved in the study. The randomized drug will be available 24 hours and 7 days a week. They would have to come to the study office to receive the blinded drug, and would need to seek treatment within 6 hours of the onset of the prodrome. Following the blinded treatment, the subject would need to be observed for at least 30 minutes. If a subject develops an acute HAE exacerbation in any of the three treatment periods, they would have access to 20 units/kg of open label C1-INH for acute treatment also received at the study office, and the time from onset of symptoms of an acute attack until the time the subject sought open-label treatment could not exceed 12 hours. Following open label rescue, the subject would need to be observed for at least 1 hour or until symptoms started to improve. The subject would also be expected to complete a symptom diary card over the next 24 hours after receiving open-label C1-INH to monitor severity and duration of symptoms (24 Hour Prodrome/Open-Label Diary Appendix IV) associated with the acute attack. The number, duration, and severity of acute HAE attacks would be compared for each treatment period. Each treatment period would last 16 weeks so each subject would be studied for a total of 48 weeks. Because safety has been established when C1-
Sponsor: Penn State University

Current Primary Outcome: percentage of full blown HAE attacks occurring within 24 hours following treatment at the prodromal [ Time Frame: 24 hours ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

Original Secondary Outcome:

Information By: Penn State University

Dates:
Date Received: June 25, 2010
Date Started: July 2010
Date Completion:
Last Updated: November 29, 2012
Last Verified: November 2012