- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01465958
Pharmacokinetics, Safety, and Tolerability of Subcutaneous GAMUNEX-C in Pediatric Subjects With Primary Immunodeficiency (KIDS)
February 23, 2015 updated by: Grifols Therapeutics LLC
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.
Study Overview
Status
Completed
Conditions
Intervention / Treatment
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.
Study Type
Interventional
Enrollment (Actual)
12
Phase
- Phase 4
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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California
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Los Angeles, California, United States, 90027
- Childrens Hospital Los Angeles
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Colorado
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Centennial, Colorado, United States, 80112
- IMMUNOe International Research Centers
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Florida
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Hollywood, Florida, United States, 33021
- Joe DiMaggio Children's Hospital
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Pennsylvania
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Philadelphia, Pennsylvania, United States, 19104
- The Children's Hospital of Philadelphia
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
2 years to 16 years (Child)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Non-Randomized
- Interventional Model: Crossover Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
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
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
|
Experimental: Subcutaneous GAMUNEX-C
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GAMUNEX-C Immune Globulin Injection (Human), 10%, Caprylate/Chromatography Purified, Intravenous Administration: 200-600 mg/kg per intravenous infusion every 3-4 weeks
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
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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
|
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.
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4 to 5 weeks for IV administration; 12 weeks for SC administration
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Mean Trough of Serum Total IgG
Time Frame: 4 - 5 weeks of IV administration and 12 weeks for SC administration
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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).
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4 - 5 weeks of IV administration and 12 weeks for SC administration
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
November 1, 2011
Primary Completion (Actual)
October 1, 2013
Study Completion (Actual)
October 1, 2013
Study Registration Dates
First Submitted
October 24, 2011
First Submitted That Met QC Criteria
November 2, 2011
First Posted (Estimate)
November 6, 2011
Study Record Updates
Last Update Posted (Estimate)
March 13, 2015
Last Update Submitted That Met QC Criteria
February 23, 2015
Last Verified
February 1, 2015
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- T5004-401
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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