Pharmacokinetics, Safety, and Tolerability of Subcutaneous GAMUNEX-C in Pediatric Subjects With Primary Immunodeficiency
Information source: Grifols Therapeutics Inc.
ClinicalTrials.gov processed this data on August 23, 2015
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Primary Immunodeficiency
Intervention: GAMUNEX-C (Biological); GAMUNEX-C (Biological)
Phase: Phase 4
Status: Completed
Sponsored by: Grifols Therapeutics Inc.
Summary
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.
Clinical Details
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
Study design: Allocation: Non-Randomized, Endpoint Classification: Pharmacokinetics Study, Intervention Model: Crossover Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary outcome: Steady-state Area Under the Curve (AUC) for Serum Total Immunoglobulin (IgG)Mean Trough of Serum Total IgG
Detailed description:
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.
Eligibility
Minimum age: 2 Years.
Maximum age: 16 Years.
Gender(s): Both.
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.
Locations and Contacts
Childrens Hospital Los Angeles, Los Angeles, California 90027, United States
IMMUNOe International Research Centers, Centennial, Colorado 80112, United States
Joe DiMaggio Children's Hospital, Hollywood, Florida 33021, United States
The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, United States
Additional Information
Starting date: November 2011
Last updated: February 23, 2015
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