Pharmacokinetics, Safety, and Tolerability of Subcutaneous GAMUNEX-C in Pediatric Subjects With Primary Immunodeficiency (KIDS)
The purpose of this open-label study is to evaluate the pharmacokinetics, safety, and tolerability of subcutaneously (SC; under the skin) administered GAMUNEX-C compared to intravenously (IV; through the vein) administered GAMUNEX-C in subjects 2-16 years of age with Primary Immunodeficiency.
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Pharmacokinetics Study
Intervention Model: Crossover Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||An Open-label, Single-sequence, Crossover Study to Evaluate the Pharmacokinetics, Safety and Tolerability of Subcutaneous GAMUNEX®-C in Pediatric Subjects With Primary Immunodeficiency|
- Steady-state Area Under the Curve (AUC) for Serum Total Immunoglobulin (IgG) [ Time Frame: 4 to 5 weeks for IV administration; 12 weeks for SC administration ] [ Designated as safety issue: No ]Steady-state area under the curve (AUC): For the IV phase, the mean adjusted AUC was calculated for all 11 subjects, which included subjects on both 3 and 4 week intravenous (IV) dosing schedules and who had sufficient immunoglobulin G (IgG) data. For the SC phase, the mean AUC was calculated for 10 subjects on weekly subcutaneous (SC) administration and who had sufficient IgG data.
- Mean Trough of Serum Total IgG [ Time Frame: 4 - 5 weeks of IV administration and 12 weeks for SC administration ] [ Designated as safety issue: No ]Mean trough serum total IgG values were calculated for each subject for the IV Phase (IV #1 and IV #2) and the SC phase (SC Weeks #9 and #12, and End of Treatment/Early termination visit). Mean trough concentration values of serum total IgG during the IV and SC phases were calculated based on the IgG population (subjects who received any amount of study drug and had serum total IgG concentration data).
|Study Start Date:||November 2011|
|Study Completion Date:||October 2013|
|Primary Completion Date:||October 2013 (Final data collection date for primary outcome measure)|
|Active Comparator: Intravenous GAMUNEX-C||
GAMUNEX-C Immune Globulin Injection (Human), 10%, Caprylate/Chromatography Purified, Intravenous Administration: 200-600 mg/kg per intravenous infusion every 3-4 weeks
|Experimental: Subcutaneous GAMUNEX-C||
GAMUNEX-C Immune Globulin Injection (Human), 10%, Caprylate/Chromatography Purified, Subcutaneous Administration: weekly subcutaneous infusion at a mg/kg dose based on subject's intravenous dose and dosing interval x 1.37 conversion factor
This study was a multi-center, open-label, single-sequence, crossover study to evaluate the pharmacokinetics (PK), safety and tolerability of SC-administered GAMUNEX-C in pediatric PI subjects (ages 2-16). The study consisted of a Screening Phase, Run-in Phase, two treatment phases (an IV Phase and a SC Phase), and an End of Study/Early Termination (EOS/ET) visit. Run-in phase: 3 - 4 months, IV Phase: ~ 4 - 5 weeks, SC Phase: 12 weeks, and End of Study/Early Termination (EOS/ET) visit: one week after SC Week #12.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01465958
|United States, California|
|Childrens Hospital Los Angeles|
|Los Angeles, California, United States, 90027|
|United States, Colorado|
|IMMUNOe International Research Centers|
|Centennial, Colorado, United States, 80112|
|United States, Florida|
|Joe DiMaggio Children's Hospital|
|Hollywood, Florida, United States, 33021|
|United States, Pennsylvania|
|The Children's Hospital of Philadelphia|
|Philadelphia, Pennsylvania, United States, 19104|