NCT01465958

Brief 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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
12

participants targeted

Target at below P25 for phase_4

Timeline
Completed

Started Nov 2011

Geographic Reach
1 country

4 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

October 24, 2011

Completed
8 days until next milestone

Study Start

First participant enrolled

November 1, 2011

Completed
6 days until next milestone

First Posted

Study publicly available on registry

November 7, 2011

Completed
1.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 1, 2013

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2013

Completed
1.3 years until next milestone

Results Posted

Study results publicly available

January 28, 2015

Completed
Last Updated

March 13, 2015

Status Verified

February 1, 2015

Enrollment Period

1.9 years

First QC Date

October 24, 2011

Results QC Date

January 19, 2015

Last Update Submit

February 23, 2015

Conditions

Primary Immunodeficiency

Outcome Measures

Primary Outcomes (2)

  • Steady-state Area Under the Curve (AUC) for Serum Total Immunoglobulin (IgG)

    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.

    4 to 5 weeks for IV administration; 12 weeks for SC administration

  • Mean Trough of Serum Total IgG

    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).

    4 - 5 weeks of IV administration and 12 weeks for SC administration

Study Arms (2)

Intravenous GAMUNEX-C

ACTIVE COMPARATOR
Biological: GAMUNEX-C

Subcutaneous GAMUNEX-C

EXPERIMENTAL
Biological: GAMUNEX-C

Interventions

GAMUNEX-CBIOLOGICAL

GAMUNEX-C Immune Globulin Injection (Human), 10%, Caprylate/Chromatography Purified, Intravenous Administration: 200-600 mg/kg per intravenous infusion every 3-4 weeks

Intravenous GAMUNEX-C

Eligibility Criteria

Age2 Years - 16 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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).

You may not qualify if:

  • 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.
  • +2 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

Childrens Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

IMMUNOe International Research Centers

Centennial, Colorado, 80112, United States

Location

Joe DiMaggio Children's Hospital

Hollywood, Florida, 33021, United States

Location

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Related Publications (1)

  • Heimall J, Chen J, Church JA, Griffin R, Melamed I, Kleiner GI. Pharmacokinetics, Safety, and Tolerability of Subcutaneous Immune Globulin Injection (Human), 10 % Caprylate/Chromatography Purified (GAMUNEX(R)-C) in Pediatric Patients with Primary Immunodeficiency Disease. J Clin Immunol. 2016 Aug;36(6):600-9. doi: 10.1007/s10875-016-0311-4. Epub 2016 Jun 25.

    PMID: 27342758DERIVED

MeSH Terms

Conditions

Primary Immunodeficiency Diseases

Interventions

Hizentra

Condition Hierarchy (Ancestors)

Genetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesImmunologic Deficiency SyndromesImmune System Diseases

Results Point of Contact

Title
Henry Li, PhD
Organization
Grifols Therapeutics Inc
henry.li@grifols.com
800-20-2807

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
LTE60
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 4
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
CROSSOVER
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 24, 2011

First Posted

November 7, 2011

Study Start

November 1, 2011

Primary Completion

October 1, 2013

Study Completion

October 1, 2013

Last Updated

March 13, 2015

Results First Posted

January 28, 2015

Record last verified: 2015-02

Locations

US(4)