A Trial of the Pharmacokinetics, Safety, and Tolerability of Subcutaneous Gamunex® in Primary Immunodeficiency

Immunologic Deficiency Syndrome Clinical Trial

Official title:

An Open-Label Single-Sequence, Crossover Trial to Evaluate the Pharmacokinetics and Safety of Subcutaneous Gamunex® 10% (Immune Globulin Intravenous [Human], 10% Caprylate/Chromatography Purified) in Subjects With Primary Immunodeficiency

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00389324
• Other study ID #: 060001
• Status: Completed
• Phase: Phase 2
• First received: October 17, 2006
• Last updated: March 31, 2015
• Start date: November 2006
• Est. completion date: August 2008

Study information

• Verified date: March 2015
• Source: Grifols Therapeutics Inc.
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationCanada: Health Canada
• Study type: Interventional

Clinical Trial Summary

This study will compare the blood level of Gamunex in patients. Patients will take it as an injection under the skin or in a vein. The study will compare how safe and tolerable the two methods are in the patients. The patients in this study have a defect in their immune system from a genetic cause.

Description:

This is an open-label, single-sequence, multi-center trial with subjects previously diagnosed with primary immune deficiency. Subjects will be on IGIV until a steady state is reached at which time PK profiling during the IV phase will occur. Subjects will begin SC administration 1 week following last IV dose and followed for a period of six months. PK profiling in SC phase will occur when subject reaches approximate steady-state on SC administration.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 35
• Est. completion date: August 2008
• Est. primary completion date: August 2008
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 13 Years to 75 Years

Eligibility

Inclusion Criteria:

• Adults and adolescents (age 13-75 inclusive) with a documented and confirmed pre-existing diagnosis of chronic primary immunodeficiency

• Previously or currently on IgG replacement therapy

• Documented (within 3 months) plasma IgG level of =500 mg/dL on current IgG therapy (IgG level can be obtained at the screening visit if documentation is not available)

• The medical records for all subjects within the previous 2 years should be available to document previous infections and treatment

Exclusion Criteria:

• Clinical evidence of any significant acute or chronic disease that, in the opinion of the investigator, may interfere with successful completion of the trial

• The subject has a known adverse reaction to Gamunex or other blood products

• The subject has a history of blistering skin disease, clinically significant thrombocytopenia, bleeding disorder, diffuse rash, recurrent skin infections or other disorders where subcutaneous therapy would be contraindicated

• The subject has known selective IgA deficiency with the exception of a known selective IgA deficient subject who has no previous documented eventful reaction to products containing IgA

• The subject is pregnant or lactating

• The subject has significant proteinuria and/or has a history of acute renal failure and/or severe renal impairment (BUN or creatinine more than 2.5 times the upper limit of normal) and/or on dialysis

• The subject has known substance or prescription drug abuse in the past 12 months

• The subject has a history of or current diagnosis of deep venous thrombosis

• The subject has an acquired medical condition that is known to cause secondary immune deficiency, such as chronic lymphocytic leukemia, lymphoma, multiple myeloma, chronic or recurrent neutropenia (absolute neutrophil count less than 1000 x 10e6/L), or HIV infection/AIDS

• The subject is receiving any of the following medications: corticosteroids (long-term daily, >1 mg of prednisone equivalent/kg/day for >30 days) (intermittent courses would not exclude subject); immunosuppressants; or immunomodulators

• The subject has non-controlled arterial hypertension (systolic blood pressure >160 mmHg and/or diastolic blood pressure >100 mmHg)

• The subject has anemia (hemoglobin <10 g/dL) at screening

• The subject has participated in another clinical trial 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

Study Design

Allocation: Non-Randomized, Endpoint Classification: Pharmacokinetics Study, Intervention Model: Crossover Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Immunologic Deficiency Syndrome
• Immunologic Deficiency Syndromes

Intervention

Biological:
• Immune Globulin Intravenous (Human)
This trial was an open-label, single-sequence, study. The enrolled subjects received IGIV-C via two routes of administration (IV for 4 -5 weeks and SC for 24 weeks) in order to compare the PK variables, safety and tolerability of SC administration of IGIV-C. Certain subjects required IV IGIV-C dosing during a Run-in Phase (3 - 4 months) for steady-state conditions prior to the IV phase. Subjects received two IV infusions of IGIV (between 200 - 600 mg based on the subject's previous IgG dosing regimen, 3 to 4 weeks apart) until a steady-state was reached at which time PK profiling was performed. Subjects began weekly SC administration (1.37 times the weekly equivalency of each subject's monthly IV dose) 1 week following last IV dose and followed for a period of six months.

Locations

Country	Name	City	State
Canada	McGill University - Montreal General Hospital	Montreal	Quebec
Canada	Dr. Donald F. Stark, Inc	Vancouver	British Columbia
United States	Family Allergy & Asthma Center, PC	Atlanta	Georgia
United States	Pediatric Allergy / Immunology Associates, PA	Dallas	Texas
United States	University of California, Irvine	Irvine	California
United States	UCLA School of Medicine	Los Angeles	California
United States	Allergy, Asthma & Immunology Associates, PC	Omaha	Nebraska
United States	Virginia Commonwealth University	Richmond	Virginia

Sponsors (1)

Lead Sponsor	Collaborator
Grifols Therapeutics Inc.

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Geometric Least Square Means of Area Under the Curve (AUC) for Plasma Total Immunoglobulin G (IgG)	Geometric least-squares mean of steady-state plasma concentration of total IgG vs. time profile (AUC).	IV Phase (21 or 28 days) at IV Visit #1, pre- and post-dose: 0 hr., 1 hr., and 1, 2, 3, 5, 7, 14, 21, and 28 days; SC Phase at Week #17, pre- and post-dose: 0 hr., and 1, 3, 4, 5, and 7 days	No

External Links

•   FDA approved labeling information