Abstract | PURPOSE: This phase 4, multicenter, open-labeled, single-sequence, crossover study in pediatric patients (ages 2 to 16) with primary immunodeficiency disease (PID) evaluated the pharmacokinetics, safety, and tolerability for subcutaneously (SC) administered 10 % caprylate/chromatography purified human immune globulin injection (IGIV-C, GAMUNEX®) compared with intravenously (IV) administered IGIV-C. METHODS: This study included a screening phase, run-in phase (where required), IV treatment phase, SC treatment phase, and end of study/early termination visit. Eligible patients receiving a stable dose of IGIV-C entered into the IV phase to receive two IV infusions of IGIV-C (200-600 mg/kg per infusion) every 3-4 weeks. The weekly SC dose of IGIV-C was calculated using a conversion factor of 1.37 times the prior IV dose. RESULTS: Twelve subjects between the ages of 2 and 16 years participated in the clinical study with the median age being 11 years old. The adjusted weekly mean AUC0-τ,IV was 216,873.7 h*mg/dL for the IV phase versus a mean AUC0-τ,SC of 230,830.0 h*mg/dL for the SC phase. The mean (range) C trough was 997.2 (784-1320) mg/dL in the IV phase and 1325.0 (1077-1690) mg/dL in the SC phase. During the SC phase, 100.0 % of the patients (n = 11) experienced treatment-emergent adverse events (TEAEs) that were local infusion reactions and 9 patients (81.8 %) had TEAEs that were non- infusion site reactions. The majority of TEAEs were mild or moderate in severity. CONCLUSION: In pediatric patients with PID, SC-administered IGIV-C provides comparable overall serum exposure to total IgG to that produced by IV-administered IGIV-C. We have concluded that weekly SC administration of 10 % IGIV-C based on a dose conversion factor of 1.37 is safe and well-tolerated in pediatric patients with PID. TRIAL REGISTRATION: ClinicalTrials.gov identifier: NCT01465958. https://clinicaltrials.gov/ct2/show/NCT01465958?term=NCT01465958.&rank=1.
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Authors | Jennifer Heimall, Junliang Chen, Joseph A Church, Rhonda Griffin, Isaac Melamed, Gary I Kleiner |
Journal | Journal of clinical immunology
(J Clin Immunol)
Vol. 36
Issue 6
Pg. 600-9
(08 2016)
ISSN: 1573-2592 [Electronic] Netherlands |
PMID | 27342758
(Publication Type: Clinical Trial, Journal Article, Multicenter Study, Research Support, Non-U.S. Gov't)
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Chemical References |
- Antibodies, Neutralizing
- Immunoglobulins, Intravenous
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Topics |
- Adolescent
- Antibodies, Neutralizing
(blood)
- Area Under Curve
- Bacterial Infections
(etiology)
- Child
- Child, Preschool
- Drug Monitoring
- Female
- Humans
- Immunoglobulins, Intravenous
(pharmacology, therapeutic use)
- Immunologic Deficiency Syndromes
(blood, diagnosis, drug therapy)
- Injection Site Reaction
(etiology)
- Male
- Treatment Outcome
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