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Pharmacokinetics, Safety, and Tolerability of Subcutaneous GAMUNEX-C in Pediatric Subjects With Primary Immunodeficiency (KIDS)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Grifols Therapeutics Inc.
ClinicalTrials.gov Identifier:
NCT01465958
First received: October 24, 2011
Last updated: November 26, 2013
Last verified: November 2013

October 24, 2011
November 26, 2013
November 2011
October 2013   (final data collection date for primary outcome measure)
Pharmacokinetics of SC GAMUNEX-C compared to IV GAMUNEX-C in pediatric (2-16 years old) patients with Primary Immunodeficiency [ Time Frame: 4 to 5 weeks for IV administration; 12 weeks for SC administration ] [ Designated as safety issue: No ]
Pharmacokinetics of SC GAMUNEX-C compared to IV GAMUNEX-C in pediatric (2-16 years old) patients with Primary Immunodeficiency [ Time Frame: 4 to 5 weeks for IV administration; 12-weeks for SC administration ] [ Designated as safety issue: No ]
Complete list of historical versions of study NCT01465958 on ClinicalTrials.gov Archive Site
Safety (including incidence of adverse events and site infusion reactions) of SC GAMUNEX-C in pediatric (2-16 years old) patients with Primary Immunodeficiency [ Time Frame: 12 weeks for SC administration ] [ Designated as safety issue: Yes ]
Safety (including incidence of adverse events and site infusion reactions) of SC GAMUNEX-C in pediatric (2-16 years old) patients with Primary Immunodeficiency [ Time Frame: Throughout the study for up to 37 weeks depending on individual infusion interval and IV infusion history ] [ Designated as safety issue: Yes ]
Not Provided
Not Provided
 
Pharmacokinetics, Safety, and Tolerability of Subcutaneous GAMUNEX-C in Pediatric Subjects With Primary Immunodeficiency
An Open-label, Single-sequence, Crossover Study to Evaluate the Pharmacokinetics, Safety and Tolerability of Subcutaneous GAMUNEX®-C in Pediatric Subjects With Primary Immunodeficiency

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.

Not Provided
Interventional
Phase 4
Allocation: Non-Randomized
Endpoint Classification: Pharmacokinetics Study
Intervention Model: Crossover Assignment
Masking: Open Label
Primary Purpose: Treatment
Primary Immunodeficiency
  • Biological: 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
  • Biological: 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
  • Active Comparator: Intravenous GAMUNEX®-C
    Intervention: Biological: GAMUNEX®-C
  • Experimental: Subcutaneous GAMUNEX®-C
    Intervention: Biological: GAMUNEX®-C
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
18
October 2013
October 2013   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • Aged 2-16 years old, inclusive.
  • Documented and confirmed pre-existing diagnosis of PI with features of hypogammaglobulinemia requiring immunoglobulin replacement.
  • Currently on IgG replacement therapy with a serum IgG trough concentration of ≥ 500 mg/dL at the Screening Visit.
  • Adequate normal skin to allow for SC infusions.
  • Signs an assent form, if applicable (per Institutional Review Board [IRB] requirements). Parent or legal guardian must sign an informed consent form.
  • Females of childbearing potential must have a negative urine pregnancy test result and must practice an effective form of contraception (which may include abstinence).

Exclusion Criteria:

  • History of anaphylaxis or severe systemic response to an immunoglobulin or blood product.
  • History of blistering skin disease, clinically significant thrombocytopenia, bleeding disorder, recurrent skin infections or other disorders where subcutaneous therapy could be contraindicated.
  • Has a specific antibody deficiency disorder, IgG subclass deficiency, or transient hypogammaglobulinemia of infancy.
  • History of severe adverse reaction to parenteral products containing immunoglobulin A (IgA).
  • Significant proteinuria and/or has a history of acute renal failure and/or severe renal impairment (serum creatinine more than 2.5 times the upper limit of normal [ULN] for age and gender) and/or is on dialysis.
  • Known substance or prescription drug abuse in the past 12 months.
  • Acquired medical condition that is known to cause secondary immune deficiency.
  • Receiving any of the following medications: systemic corticosteroids (long term daily, >1 mg of prednisone equivalent/kg/day for >30 days) (intermittent courses would not exclude subject); immunosuppressants (i.e., antimetabolites and systemic calcineurin inhibitors; NOTE: inhaled steroids are allowed); or immunomodulators.
  • Non-controlled arterial hypertension at a level of ≥ the 90th percentile blood pressure (either systolic or diastolic) for age and height (based on http://www.nhlbi.nih.gov/guidelines/hypertension/child_tbl.pdf ).
  • History or current diagnosis of thrombotic episodes; venous thrombus that occurred in association with a medical device > 2 years prior to screening are allowed.
  • Currently receiving anti-coagulation therapy.
  • History of Kawasaki disease.
  • Participated in another clinical trial involving exposure to an investigational product or device within 30 days prior to screening (imaging studies without investigative treatments are permitted) or has received any investigational blood product within the previous 3 months.
  • Unable or unwilling to comply with any aspect of the protocol, including blood sampling and completion of the Infusion Site Reactions pages in the SC Infusion Diary.
  • In the opinion of the Investigator the subject may have compliance problems with the protocol and the procedures of the protocol.
  • Pregnant or lactating.
  • Clinical evidence of any significant acute or chronic disease that, in the opinion of the Investigator, may interfere with successful completion of the study.
Both
2 Years to 16 Years
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT01465958
T5004-401
No
Grifols Therapeutics Inc.
Grifols Therapeutics Inc.
Not Provided
Not Provided
Grifols Therapeutics Inc.
November 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP