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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00634569
Recruitment Status : Completed
First Posted : March 13, 2008
Results First Posted : December 8, 2016
Last Update Posted : February 2, 2017
Information provided by (Responsible Party):
Grifols Biologicals, LLC ( Instituto Grifols, S.A. )

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

Condition or disease Intervention/treatment Phase
Primary Immune Deficiency Disease Biological: Flebogamma 5% DIF Phase 4

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 24 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (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.
Study Start Date : May 2008
Actual Primary Completion Date : March 2011
Actual Study Completion Date : May 2011

Arm Intervention/treatment
Experimental: Flebogamma 5% DIF Biological: Flebogamma 5% DIF
Intravenous Immune Globulin (Human)

Primary Outcome Measures :
  1. Serious Bacterial Infections. [ Time Frame: 12 months ]
    Total number of Bacterial pneumonia, bacteremia or sepsis, osteomyelitis/septic arthritis, visceral abscess or bacterial meningitis

Secondary Outcome Measures :
  1. Days of School/Usual Activities Missed Per Year [ Time Frame: 12 months ]
    Mean Days of school/usual activities missed per subject/year

  2. Days of Hospitalization Per Year [ Time Frame: 12 months ]
    Mean Days of hospitalization per subject/year

  3. Number of Visits to Physician/ER Room for Acute Problems [ Time Frame: 12 months ]
    Mean Number of visits to physician/ER room for acute problems

  4. Other Infections Documented by Fever and Physical Exam or Positive Radiograph. [ Time Frame: 12 months ]
  5. Number of Infectious Episodes Per Year [ Time Frame: 12 months ]
    Mean Number of infectious episodes per subject/year

  6. Number of Days on Antibiotics (Prophylactic and Therapeutic). [ Time Frame: 12 months ]
    Median Combined number of days on prophylactic and therapeutic antibiotics

  7. Number of Adverse Events [ Time Frame: 12 months ]
    Total Number of Adverse Events

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   2 Years to 16 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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).

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00634569

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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.
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Principal Investigator: Mark Ballow, MD Children's Hospital of Buffalo
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Responsible Party: Instituto Grifols, S.A. Identifier: NCT00634569    
Other Study ID Numbers: IG-0705
First Posted: March 13, 2008    Key Record Dates
Results First Posted: December 8, 2016
Last Update Posted: February 2, 2017
Last Verified: December 2016
Keywords provided by Grifols Biologicals, LLC ( Instituto Grifols, S.A. ):
CVID, XLA, hyper IgM syndrome, Wiskott-Aldrich Syndrome
Additional relevant MeSH terms:
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Primary Immunodeficiency Diseases
Deficiency Diseases
Immunologic Deficiency Syndromes
Immune System Diseases
Genetic Diseases, Inborn
Nutrition Disorders
Immunoglobulins, Intravenous
Rho(D) Immune Globulin
Immunologic Factors
Physiological Effects of Drugs