Clinical Trial: A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary Immunodeficiency

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

Official Title: A Phase IV, Multicenter, Open-Label Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases (PID) in Children and Adolescents

Brief Summary: The main objective is to determine the efficacy of Gammaplex by measuring the number of serious acute bacterial infections during treatment with Gammaplex over a 12 month period. The secondary objectives are to assess the safety and tolerability of Gammaplex and to compare the data collected from adult subjects with PID from the GMX01 study

Detailed Summary:
Sponsor: Bio Products Laboratory

Current Primary Outcome: Adverse Events [ Time Frame: 12 months ]

Number of subjects with serious, acute, bacterial infections as a measure of efficacy


Original Primary Outcome: Number of subjects with serious, acute bacterial infections. [ Time Frame: 12 months ]

Current Secondary Outcome:

  • Therapeutic Efficacy [ Time Frame: From week 15 onwards ]
    Number and proportion of subjects who maintain trough IgG levels at least as high as the average of the 2 previous trough levels before the first Gammaplex infusion
  • Therapeutic Efficacy [ Time Frame: 12 months ]
    Number of days off school
  • Therapeutic Efficacy [ Time Frame: 12 months ]
    Number of days in hospital
  • Therapeutic Efficacy [ Time Frame: 12 months ]
    Visits to physicians and/or emergency room
  • Therapeutic Efficacy [ Time Frame: 12 months ]
    Number of days on therapeutic antibiotics


Original Secondary Outcome:

  • Number and proportion of IGIV naive subjects with a trough IgG level of greater than or equal to 300 mg/dL above the diagnostic (i.e. endogenous) level. [ Time Frame: Week 15 onwards ]
  • Number and proportion of subjects with trough IgG levels of less than or equal to 600 mg/dL [ Time Frame: Week 15 onwards ]
  • Therapeutic efficacy of Gammaplex [ Time Frame: 12 months ]
    Number of infections, days off school, days in hospital, visits to physicians &/or emergency room, days on antibiotics
  • Infusion associated adverse events [ Time Frame: 12 months ]
    Number and percent of infusions associated with 1 or more adverse events beginning during the infusion or within 72 hours of infusion end.
  • Tolerability of Gammaplex [ Time Frame: 12 months ]
    Nature, severity and frequency of Adverse Events
  • Vital signs [ Time Frame: 12 months ]
    Change in vital signs
  • Clinical laboratory testing [ Time Frame: 12 months ]
    Change in clinical laboratory tests including Direct Coombs' Test
  • Viral safety [ Time Frame: 12 months ]
    Assessment of transmission of viruses
  • Physical examination [ Time Frame: 12 months ]
    Clinically significant changes from baseline will be recorded as adverse events


Information By: Bio Products Laboratory

Dates:
Date Received: January 27, 2011
Date Started: March 2011
Date Completion:
Last Updated: December 15, 2014
Last Verified: December 2014