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

NCT00634569 | Completed Phase 4 Results

• 1 other identifier: IG-0705
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 7

Brief Summary

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

Conditions

Primary Immune Deficiency Disease

Keywords

CVID, XLA, hyper IgM syndrome, Wiskott-Aldrich Syndrome

Outcome Measures

Primary Outcomes (1)

• Serious Bacterial Infections.

  Total number of Bacterial pneumonia, bacteremia or sepsis, osteomyelitis/septic arthritis, visceral abscess or bacterial meningitis

  12 months

Secondary Outcomes (7)

• Days of School/Usual Activities Missed Per Year

  12 months

• Days of Hospitalization Per Year

  12 months

• Number of Visits to Physician/ER Room for Acute Problems

  12 months

• Other Infections Documented by Fever and Physical Exam or Positive Radiograph.

  12 months

• Number of Infectious Episodes Per Year

  12 months

Study Arms (1)

Flebogamma 5% DIF

EXPERIMENTAL

Biological: Flebogamma 5% DIF

Interventions

Flebogamma 5% DIF BIOLOGICAL

Intravenous Immune Globulin (Human)

Flebogamma 5% DIF

Eligibility Criteria

• Age: 2 Years - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Instituto Grifols, S.A.: lead

Study Sites (7)

University of South Florida

St. Petersburg, Florida, 33701-4899, United States

Location

Family Allergy & Asthma Center, PC

Atlanta, Georgia, 30342, United States

Location

Rush University Medical Center

Chicago, Illinois, United States

Location

The Allergy and Asthma Center

Fort Wayne, Indiana, 46804, United States

Location

The Children's Hospital of Buffalo

Buffalo, New York, 14222, United States

Location

Pennsylvania State University, Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033-0850, United States

Location

Children's Hospital and Regional Medial Center

Seattle, Washington, 98195-7110, United States

Location

MeSH Terms

Conditions

Primary Immunodeficiency Diseases; Bruton type agammaglobulinemia; Hyper-IgM Immunodeficiency Syndrome; Wiskott-Aldrich Syndrome

Interventions

gamma-Globulins

Condition Hierarchy (Ancestors)

Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Immunologic Deficiency Syndromes; Immune System Diseases; Dysgammaglobulinemia; Blood Protein Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Lymphopenia; Leukopenia; Cytopenia; Hemorrhagic Disorders; Leukocyte Disorders; Genetic Diseases, X-Linked

Intervention Hierarchy (Ancestors)

Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Results Point of Contact

• Title: Paul J. Pinciaro, PhD
• Organization: Grifols

paul.pinciaro@grifols.com

410-814-7617

Study Officials

• Mark Ballow, MD

  Children's Hospital of Buffalo

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 5, 2008
• First Posted: March 13, 2008
• Study Start: May 1, 2008
• Primary Completion: March 1, 2011
• Study Completion: May 1, 2011
• Last Updated: February 2, 2017
• Results First Posted: December 8, 2016

Record last verified: 2016-12

Locations

US (7)