Clinical Trial: Efficacy and Safety Study of DX-2930 to Prevent Acute Angioedema Attacks in Patients With Type I and Type II HAE

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: HELP Study: A Multicenter, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study to Evaluate DX-2930 For Long-Term Prophylaxis Against Acute Attacks of Hereditar

Brief Summary: This is a phase 3, multicenter, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of DX-2930 in preventing acute angioedema attacks in patients with Type I and Type II HAE.

Detailed Summary:
Sponsor: Shire

Current Primary Outcome: Number of Investigator confirmed HAE attacks per week observed in each DX-2930 treatment arm versus placebo arm [ Time Frame: Efficacy assessment period (Day 0 through Day 182) ]

Original Primary Outcome: Number of HAE attacks per week observed in each DX-2930 treatment arm versus placebo arm [ Time Frame: Efficacy assessment period (Day 14 through Day 182) ]

Current Secondary Outcome:

  • Number per week of HAE attacks requiring acute attack therapy use for each DX-2930 treatment arm versus placebo arm [ Time Frame: Efficacy assessment period (Day 0 through Day 182) ]
  • Number per week of moderate or severe HAE attacks for each DX-2930 treatment arm versus placebo arm [ Time Frame: Efficacy assessment period (Day 0 through Day 182) ]
  • Number of Investigator confirmed HAE attacks for each DX-2930 treatment arm versus placebo arm occurring on Day 14 through Day 182 [ Time Frame: Day 14 through Day 182 ]


Original Secondary Outcome:

  • Time to first HAE attack for each DX-2930 treatment arm versus placebo arm [ Time Frame: Efficacy assessment period (Day 14 through Day 182) ]
  • Number per week of HAE attacks requiring acute attack therapy use for each DX-2930 treatment arm versus placebo arm [ Time Frame: Efficacy assessment period (Day 14 through Day 182) ]
  • Number per week of moderate or severe HAE attacks for each DX-2930 treatment arm versus placebo arm [ Time Frame: Efficacy assessment period (Day 14 through Day 182) ]
  • Reduction in high-morbidity HAE attacks for each DX-2930 treatment arm versus placebo arm [ Time Frame: Efficacy assessment period (Day 14 through Day 182) ]
    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: October 23, 2015
Date Started: March 3, 2016
Date Completion:
Last Updated: May 23, 2017
Last Verified: May 2017