Clinical Trial: Pharmacokinetics, Efficacy, and Safety Study of RI-002 (IGIV) in Subjects With Primary Immunodeficiency Diseases (PIDD)

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

Official Title: An Open Label, Multicenter, Study to Evaluate the Pharmacokinetics, Efficacy and Safety of RI-002 (IGIV) in Subjects With Primary Immunodeficiency Diseases (PIDD)

Brief Summary: This is a Phase III, multicenter, open-label study of RI-002 administered as an intravenous infusion of RI-002 (IGIV) every 21 or 28 days in approximately 60 subjects with Primary Immunodeficiency Diseases (PIDD).

Detailed Summary: Primary immunodeficiency diseases (PIDDs) are genetically determined disorders of the immune system resulting in greatly enhanced susceptibility to infectious disease, autoimmunity and malignancy. As most subjects with PIDDs present with infections, the differential diagnosis and initial investigations for an underlying immune defect are typically guided by the clinical presentation. In subjects with PIDDs, individual infections are not necessarily more severe than those that occur in a normal host. Rather, the clinical features suggestive of an immune defect may be the recurring and/or chronic nature of infections with common pathogens that may result in end organ damage, such as bronchiectasis. Several immune globulin products have already been approved by the FDA.
Sponsor: ADMA Biologics, Inc.

Current Primary Outcome: Number of Serious Bacterial Infections (SBIs) Per Subject Per Year (FDA Guidance for Industry (2008)) [ Time Frame: One year ]

The primary objective of this study was to demonstrate that RI-002 (IGIV) reduces the frequency of serious bacterial infections (SBIs), as defined by the Diagnostic Criteria for Serious Infection Types guideline, in subjects with primary humoral immunodeficiency.


Original Primary Outcome: Demonstrate that RI-002 prevents the frequency of serious bacterial infections(FDA Guidance for Industry (2008)) [ Time Frame: Up to 1 year ]

Current Secondary Outcome:

  • Incidence of All Infections (Serious and Non-serious) [ Time Frame: Up to 1 Year ]
  • Number of Days Lost From Work/School/Daycare and Usual Activities Due to Infections and Their Treatment - Combined Days Lost [ Time Frame: Up to 1 year ]
  • Number of Days Lost From Work/School/Daycare and Usual Activities Due to Infections and Their Treatment - Per Subject-Year [ Time Frame: Up to 1 year ]
  • Number of Unscheduled Visits to Physician/ER Due to Infections - Total Number of Visits [ Time Frame: Up to 1 year ]
  • Number of Unscheduled Visits to Physician/ER Due to Infections - Per Subject-Year [ Time Frame: Up to 1 year ]
  • Time to Resolution of Infections - Duration Per Infection [ Time Frame: Up to 1 year ]
  • Time to Resolution of Infections - Infection Days Per Subject [ Time Frame: Up to 1 year ]
  • Number of Hospitalizations Due to Infections [ Time Frame: Up to 1 year ]
  • Number of Hospitalizations Due to Infections - Per Subject-Year [ Time Frame: Up to 1 year ]
  • Days of Hospitalization Due to Infections [ Time Frame: Up to 1 year ]
  • Days of Hospitalization Due to Infections - Per Subject-Year [ Time Frame: Up to 1 year ]
  • Number of Days of Antibiotic Therapy (Prophylaxis and Treatment of Infection) [ Time Frame: Up to 1 year ]
    Summary of the days of antibiotic therapy in the study for prophylaxis, as treatment for infections, and combined
  • Number of Days of Antibiotic Therapy (Prophylaxis and Treatment of Infection) - Per Subject-Year [ Time Frame: Up to 1 year ]
    Summary of the days of antibiotic therapy in the study for prophylaxis, as treatment for infections, and combined, per subject-year of treatment with RI-002
  • Correlation Between Trough Level of RI-002 and Serious and Non-serious Infections [ Time Frame: Up to 1 year ]
    The relationship between trough IgG concentrations and the number of infections of any kind/seriousness was evaluated using Pearson linear correlation coefficients using forward analysis (outcomes after infusion) and backward analysis (outcomes prior to infusion; outcomes post last infusion were excluded)
  • Trough Total IgG and Specific Antibody Levels - IgG [ Time Frame: Up to 1 year ]
    Summary of trough total IgG concentration prior to specified infusion
  • Trough Total IgG and Specific Antibody Levels - Haemophilus Influenzae Type B [ Time Frame: Up to 1 year ]
    Summary of trough antibody concentrations prior to specified infusion for Haemophilus influenzae type B
  • Trough Total IgG and Specific Antibody Levels - Respiratory Syncytial Virus (RSV) [ Time Frame: Up to 1 year ]
    Summary of trough antibody concentrations prior to specified infusion for Respiratory Syncytial Virus (RSV)
  • Trough Total IgG and Specific Antibody Levels - Tetanus [ Time Frame: Up to 1 year ]
    Summary of trough antibody concentrations prior to specified infusion for Tetanus
  • Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 1 [ Time Frame: Up to 1 year ]
    Summary of trough antibody concentrations prior to specified infusion for Streptococcus Pneumoniae, Serotype 1
  • Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 3 [ Time Frame: Up to 1 year ]
    Summary of trough antibody concentrations prior to specified infusion for Streptococcus Pneumoniae, Serotype 3
  • Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 4 [ Time Frame: Up to 1 year ]
    Summary of trough antibody concentrations prior to specified infusion for Streptococcus Pneumoniae, Serotype 4
  • Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 5 [ Time Frame: Up to 1 year ]
    Summary of trough antibody concentrations prior to specified infusion for Streptococcus Pneumoniae, Serotype 5
  • Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 6B [ Time Frame: Up to 1 year ]
    Summary of trough antibody concentrations prior to specified infusion for Streptococcus Pneumoniae, Serotype 6B
  • Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 7F [ Time Frame: Up to 1 year ]
  • Incidence of All Infections (Serious and Non-serious) [ Time Frame: Up to 1 Year ]
  • Number of days lost from work/school/usual activities due to infections and their treatment [ Time Frame: Up to 1 year ]
  • Number of unscheduled visits to physician/ER due to infections [ Time Frame: Up to 1 year ]
  • Time to resolution of infections [ Time Frame: Up to 1 year ]
  • Number of hospitalizations and days of hospitalizations due to infections [ Time Frame: Up to 1 year ]
  • Number of days of antibiotic therapy [ Time Frame: Up to 1 year ]
  • Relationship among dose of RI-002 and serious and non-serious infections [ Time Frame: Up to 1 year ]
  • Evaluate trough total IgG and specific antibody levels [ Time Frame: Up to 1 year ]
  • Relationship among trough level of RI-002 and serious and non-serious infections [ Time Frame: Up to 1 year ]


Information By: ADMA Biologics, Inc.

Dates:
Date Received: March 6, 2013
Date Started: February 2014
Date Completion:
Last Updated: August 10, 2016
Last Verified: August 2016