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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
  Purpose

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.


Condition Intervention Phase
Primary Immunodeficiency
Biological: GAMUNEX®-C
Phase 4

Study Type: Interventional
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

Resource links provided by NLM:


Further study details as provided by Grifols Therapeutics Inc.:

Primary Outcome Measures:
  • 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 ]

Secondary Outcome Measures:
  • 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 ]

Estimated Enrollment: 18
Study Start Date: November 2011
Study Completion Date: October 2013
Primary Completion Date: October 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: Intravenous GAMUNEX®-C 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
Experimental: Subcutaneous GAMUNEX®-C 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

  Eligibility

Ages Eligible for Study:   2 Years to 16 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01465958

Locations
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, Iowa
University of Iowa Hospitals and Clinic
Iowa City, Iowa, United States, 52242
United States, Pennsylvania
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Sponsors and Collaborators
Grifols Therapeutics Inc.
  More Information

No publications provided

Responsible Party: Grifols Therapeutics Inc.
ClinicalTrials.gov Identifier: NCT01465958     History of Changes
Other Study ID Numbers: T5004-401
Study First Received: October 24, 2011
Last Updated: November 26, 2013
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Immunologic Deficiency Syndromes
Immune System Diseases
Immunoglobulins, Intravenous
Rho(D) Immune Globulin
Immunologic Factors
Pharmacologic Actions
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on November 27, 2014