Safety and Efficacy Study of Flebogamma 5% DIF IGIV in Pediatric Subjects

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Grifols Biologicals Inc. ( Instituto Grifols, S.A. )
ClinicalTrials.gov Identifier:
NCT00634569
First received: March 5, 2008
Last updated: March 18, 2013
Last verified: March 2013
  Purpose

This is a multi-center, open-label study to assess the efficacy and safety of Flebogamma 5% DIF in the pediatric population.


Condition Intervention Phase
Primary Immune Deficiency Disease
Biological: Flebogamma 5% DIF
Phase 4

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Evaluation of the Efficacy and Safety of Flebogamma 5% DIF [Immune Globulin Intravenous (Human)] for Replacement Therapy in Pediatric Subjects With Primary Immunodeficiency Diseases.

Resource links provided by NLM:


Further study details as provided by Grifols Biologicals Inc.:

Primary Outcome Measures:
  • Number of serious bacterial infections. [ Time Frame: 12 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Days of school/usual activities missed per year [ Time Frame: 12 months ] [ Designated as safety issue: No ]
  • Days of hospitalization per year [ Time Frame: 12 months ] [ Designated as safety issue: No ]
  • Number of visits to physician/ER room for acute problems [ Time Frame: 12 months ] [ Designated as safety issue: No ]
  • Other infections documented by fever and physical exam or positive radiograph. [ Time Frame: 12 months ] [ Designated as safety issue: No ]
  • Number of infectious episodes per year [ Time Frame: 12 months ] [ Designated as safety issue: No ]
  • Number of days on antibiotics (prophylactic and therapeutic). [ Time Frame: 12 months ] [ Designated as safety issue: No ]
  • Number and percent of Adverse Events [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]

Enrollment: 25
Study Start Date: May 2008
Study Completion Date: May 2011
Primary Completion Date: March 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Flebogamma 5% DIF Biological: Flebogamma 5% DIF
Intravenous Immune Globulin (Human)

  Eligibility

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

Inclusion Criteria:

  • The subject is between 2 and 16 years old, of either sex, belonging to any ethnic group, and above a minimum weight of 10 kg (this weight is based on the amount of blood required for testing).
  • The subject has a primary immunodeficiency disease, (e.g., common variable immunodeficiency, X-linked and autosomal forms of agammaglobulinemia, hyper-IgM syndrome, Wiskott-Aldrich Syndrome).
  • The subject has been receiving licensed IGIV replacement therapy at a dose that has not changed by + 50% of the mean dose on a mg/kg basis for at least 3 months before study entry and has maintained a trough level at least 300 mg/dL above baseline serum IgG levels.
  • Trough levels of IgG, dose of IGIV, and treatment intervals for the last 2 consecutive routine IGIV treatments must be documented for each subject before the first infusion in this study can be administered.
  • If a subject is an adolescent female (> 12 years of age) who is or becomes sexually active, she must have a negative result on a pregnancy test (HCG-based assay).
  • The subject, if old enough (generally 6 years to 16) has signed an informed Child Assent form and the subject's parent or legal guardian has signed an informed consent form, both approved by the Institutional Review Board.

Exclusion Criteria:

  • Adult patient (> 17 years old).
  • The subject has a history of any severe anaphylactic reaction to blood or any blood-derived product.
  • The subject is known to be intolerant to any component of the products, such as sorbitol (i.e., intolerance to fructose).
  • The subject has selective IgA deficiency or has demonstrable antibodies to IgA.
  • The subject is currently receiving, or has received, any investigational agent within the prior 3 months.
  • The subject has been exposed to blood or any blood product or derivative within the last 6 months, other than a commercially available IGIV.
  • The adolescent subject is pregnant or is nursing.
  • The subject, at screening, has levels greater than 2.5 times the upper limit of normal as defined at the central laboratory for pediatric patients of any of the following:

    • ALT
    • AST
    • LDH
  • The subject has a severe pre-existing renal impairment (defined by serum creatinine greater than 2 times the ULN or BUN greater than 2.5 times the ULN for that laboratory, or any subject who is on dialysis) or any history of acute renal failure.
  • The subject has a history of DVT or thrombotic complications of IGIV therapy.
  • The subject suffers from any acute or chronic medical condition (e.g., renal disease or predisposing conditions for renal disease, coronary artery disease, or protein losing state) that, in the opinion of the Investigator, may interfere with the conduct of the study.
  • The subject has an acquired medical condition such as lymphocytic leukemia, chronic or recurrent neutropenia (ANC less than 1000), or AIDS known to cause secondary immune deficiency, or is s/p hematopoetic stem cell transplantation.
  • The subject is receiving the following medication:

    • Systemic corticosteroids (long-term, i.e., not intermittent or burst, daily, > 1 mg of prednisone equivalent/kg/day). Topical steroids (ie- nasal, inhaled for asthma, and/or skin preparations for eczema) are not exclusionary.
    • Immunosuppressive drugs
    • Immunomodulatory drugs
  • The subject has non-controlled arterial hypertension (systolic blood pressure > 160 mm Hg and/or diastolic blood pressure > 100 mm Hg).
  • The subject has anemia (hemoglobin < 10 g/dL) at screening.
  • The subject and/or parent/legal guardian of the subject is unlikely to adhere to the protocol requirements of the study or is likely to be uncooperative or unable to provide from the patient a storage serum sample at the screening visit. (Please note, a pre-treatment serum sample to be stored at -94° F (-70º C) for possible future testing is absolutely required).
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00634569

Locations
United States, Florida
University of South Florida
St. Petersburg, Florida, United States, 33701-4899
United States, Georgia
Family Allergy & Asthma Center, PC
Atlanta, Georgia, United States, 30342
United States, Illinois
Rush University Medical Center
Chicago, Illinois, United States
United States, Indiana
The Allergy and Asthma Center
Fort Wayne, Indiana, United States, 46804
United States, New York
The Children's Hospital of Buffalo
Buffalo, New York, United States, 14222
United States, Pennsylvania
Pennsylvania State University, Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States, 17033-0850
United States, Washington
Children's Hospital and Regional Medial Center
Seattle, Washington, United States, 98195-7110
Sponsors and Collaborators
Instituto Grifols, S.A.
Investigators
Principal Investigator: Mark Ballow, MD Children's Hospital of Buffalo
  More Information

No publications provided

Responsible Party: Grifols Biologicals Inc. ( Instituto Grifols, S.A. )
ClinicalTrials.gov Identifier: NCT00634569     History of Changes
Other Study ID Numbers: IG-0705
Study First Received: March 5, 2008
Last Updated: March 18, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Grifols Biologicals Inc.:
CVID, XLA, hyper IgM syndrome, Wiskott-Aldrich Syndrome

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

ClinicalTrials.gov processed this record on April 17, 2014