Clinical Trial: Double-Blind, Multiple Ascending Dose Study to Assess Safety, Tolerability and Pharmacokinetics of DX-2930 in Hereditary Angioedema (HAE) Subjects

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

Official Title: A Phase 1b, Double-Blind, Multiple Ascending Dose Study to Assess Safety, Tolerability and Pharmacokinetics of DX-2930 in Hereditary Angioedema Subjects

Brief Summary: The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetic profile of multiple subcutaneous administrations of DX-2930 across a range of doses in Hereditary Angioedema subjects.

Detailed Summary:
Sponsor: Shire

Current Primary Outcome:

  • Number of Patients With Treatment-Emergent Adverse Events (TEAE) [ Time Frame: through 4 months ]
    As per the DX-2930-02 clinical protocol, an AE was considered treatment-emergent if the onset time was after administration of study drug through the Day 120 post-dose final follow-up visit or, in the event that onset time preceded study drug administration, the AE increased in severity during the 120-day post-dose follow-up period.
  • Proportion of Patients With Treatment-Emergent Adverse Events (TEAE) [ Time Frame: through 4 months ]
    As per the DX-2930-02 clinical protocol, an AE was considered treatment-emergent if the onset time was after administration of study drug through the Day 120 post-dose final follow-up visit or, in the event that onset time preceded study drug administration, the AE increased in severity during the 120-day post-dose follow-up period.
  • Number of Patients With Serious Adverse Events (SAEs) [ Time Frame: through 4 months ]

    As per the DX-2930-02 clinical protocol, a SAE was any adverse experience occurring at any dose that resulted in any of the following outcomes:

    • Death
    • Life-threatening experience: "life-threatening" referred to a situation in which the subject was at risk of death at the time of the event, it did not refer to an event that might have caused death if it were more severe.
    • Required inpatient hospitalization or prolongation of existing hospitalization: this did not include hospitalization

      Original Primary Outcome: proportion of patients with non-serious and serious adverse events [ Time Frame: through 4 months ]

      Current Secondary Outcome:

      • Maximum Plasma Concentration (Cmax) [ Time Frame: Pharmacokinetic samples were drawn on Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120. ]
      • Time to Maximum Plasma Concentration (Tmax) [ Time Frame: Pharmacokinetic samples were drawn on Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120. ]
      • Area Under the Plasma Concentration-time Curve (AUC) [ Time Frame: Pharmacokinetic samples were drawn on Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120. ]
      • Apparent Clearance (CL/F) [ Time Frame: Pharmacokinetic samples were drawn on Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120. ]
      • Apparent Volume of Distribution (Vd/F) [ Time Frame: Pharmacokinetic samples were drawn on Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120. ]
      • Terminal Elimination Half-life (t1/2) [ Time Frame: Pharmacokinetic samples were drawn on Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120. ]


      Original Secondary Outcome: DX-2930 plasma level [ Time Frame: through 4 months ]

      Information By: Shire

      Dates:
      Date Received: March 17, 2014
      Date Started: April 2014
      Date Completion:
      Last Updated: June 20, 2016
      Last Verified: June 2016