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 ]

The number and percentage of subjects with any AE, any related AE, any SAE, any related SAE, any severe AE, and any related severe AE as well as the total number of events for each category will be summarized.


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