Clinical Trial: Long-term Safety and Efficacy Study of DX-2930 to Prevent Acute Angioedema Attacks in Patients With Type I and Type II HAE
Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional
Official Title: HELP Study ExtensionTM: An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of DX-2930 for Prevention Against Acute Attacks of Hereditary Angioedema (HAE)
Brief Summary: This study is an open-label, long term safety and efficacy study to evaluate DX-2930 in preventing acute angioedema attacks in patients with Type I and Type II HAE.
Detailed Summary:
Sponsor: Shire
Current Primary Outcome: Evaluate the long-term safety of repeated subcutaneous (SC) administrations of DX-2930 through analyses based on treatment-emergent Adverse Events (AEs) [ Time Frame: 14 months ]
Original Primary Outcome: Number of subjects with treatment related adverse events [ Time Frame: 1 year for rollover subjects; 6 months for non-rollover subjects ]
Current Secondary Outcome:
- Evaluate the long-term efficacy of DX-2930 in preventing HAE attacks in accordance with the HAE Attack Assessment and Reporting Procedures (HAARP). [ Time Frame: Through study completion (approximately 58 weeks) ]
- Characterize the outer bounds of dosing frequency for DX-2930 by assessing the duration of time between a rollover subject's first and second open-label dose. [ Time Frame: Through study completion (approximately 58 weeks) ]
Original Secondary Outcome:
- Time to first HAE attack for rollover subjects [ Time Frame: Through study completion (approximately 34 weeks) ]
- Mean rate of HAE attacks for all subjects [ Time Frame: 14 days post-second dose to Day 182 for rollover subjects; Day 14 to Day 182 for non-rollover subjects ]
- Mean rate of acute therapy usage for all subjects [ Time Frame: 14 days post-second dose to Day 182 for rollover subjects; Day 14 to Day 182 for non-rollover subjects ]
- Mean rate of moderate or severe attacks for all subjects [ Time Frame: 14 days post-second dose to Day 182 for rollover subjects; Day 14 to Day 182 for non-rollover subjects ]The site will determine the severity of attacks based on specific definitions described in the HAE Attack Assessment and Reporting Procedures (HAARP).
- Mean rate of high-morbidity attacks for all subjects [ Time Frame: 14 days post-second dose to Day 182 for rollover subjects; Day 14 to Day 182 for non-rollover subjects ]A high morbidity HAE attack is defined as any attack that has at least one of the following characteristics: severe, results in hospitalization (except hospitalization for observation < 24 hours), hemodynamically significant (systolic blood pressure < 90, requires IV hydration, or associated with syncope or near-syncope) or laryngeal.
Information By: Shire
Dates:
Date Received: April 8, 2016
Date Started: September 2016
Date Completion: February 2018
Last Updated: March 13, 2017
Last Verified: March 2017