A Clinical Study of Intravenous Immunoglobulin

NCT00468273 | Completed Phase 3 DMC

• 2 other identifiers: GAM-PID-03-USNCT00468273
• Study Type: interventional
• Target: 57
• Locations: 2 countries
• Sites: 10

Brief Summary

The purpose of this study is to measure the pharmacokinetics, efficacy and safety of Immune Globulin Intravenous (Human) \[IGIV\], 5% Solution Omr-IgG-am™ in patients with primary immunodeficiency diseases.

Conditions

Immunologic Deficiency Syndromes

Keywords

Deficiency Syndromes, Antibody; Antibody Deficiency Syndrome; Bruton's agammaglobulinemia; Common Variable Immune Deficiency; Hyper IgM syndromes

Outcome Measures

Primary Outcomes (1)

• Incidence of acute serious bacterial infections

  Acute serious bacterial infections are defined in The FDA(CBER) Guidance for industry for studies of IGIV to support marketing of IGIV as replacement therapy for primary humoral immunodeficiency (June, 2008).

  one year

Secondary Outcomes (10)

• The number of hospitalizations and days of hospitalization per subject per year for PID related infections

  during treatment with study drug-1 year

• The incidence of infections other than acute serious bacterial infections

  during treatment with study drug-1 year

• The number of days lost from work/school/usual activities

  during treatment with study drug-1 year

• The number of days of antibiotic therapy (prophylactic and treatment)

  during treatment with study drug-1 year

• Pharmacokinetic parameters of IgG subclasses and specific antibodies will be determined in at least 20 patients: AUC0-t, Cmax, Tmax, t1/2, Vd and elimination rate constants.

  after 5th or 6th study infusion

Study Arms (1)

Intravenous Immune Globulin

EXPERIMENTAL

Subjects with primary humoral immunodeficiency

Drug: Immune Globulin Intravenous (Human) Omr-IgG-am IGIV

Interventions

Immune Globulin Intravenous (Human) Omr-IgG-am IGIV DRUG

IGIV infusions of 300-900 mg/kg every 3 or 4 weeks

Intravenous Immune Globulin

Eligibility Criteria

• Age: 3 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Ages 3 to 75 years and weigh at least 27 kg.
• Confirmed clinical diagnosis of a Primary Immune Deficiency disease including hypogammaglobulinemia, preferably with documented antibody deficiency, or agammaglobulinemia.
• Has been receiving licensed IGIV for at least 3 months prior to this study.
• Trough IgG levels, dose of IGIV, and treatment intervals for the last 2 consecutive licensed IGIV treatments must be documented.
• The subject or legal guardian has signed the informed consent form. If appropriate, the subject has signed a child assent form.
• The subject or legal representative has signed the HIPAA declaration.

You may not qualify if:

• Subjects with isolated IgG subclass deficiency or specific antibody deficiency without hypogammaglobulinemia will not be eligible.
• The subject has a history of hypersensitivity or persistent or repeated adverse reactions to human immunoglobulin.
• The subject has selective IgA deficiency, history of reaction to products containing IgA, or is known to have antibodies to IgA.
• The subject is currently participating, or has participated within the previous 30 days, in another clinical study of an investigational product or device.
• The subject is pregnant or is nursing. Women of childbearing potential must agree to using a method of contraception.
• The subject has had an acute bacterial infection within 28 days of screening.
• The subject is seropositive for any of the following at screening:
• Antibodies to HIV 1\&2
• Antibodies to HCV
• HbsAg
• The subject, at screening, has alanine aminotransferase (ALT) levels greater than 2.5 times the upper limit of normal.
• The subject has severe renal impairment.
• The subject has a history of DVT, thrombotic or thrombic complications of IGIV therapy.
• The subject suffers from any acute or chronic medical condition that, in the opinion of the investigator, may interfere with the conduct of the study.
• The subject has an acquired medical condition known to cause secondary immune deficiency or otherwise increase the subject's risk of infection.

Sponsors & Collaborators

• FFF Enterprises: lead
• OMRIX Biopharmaceuticals: collaborator

Study Sites (10)

Mattel Children's Hospital of UCLA

Los Angeles, California, 90095-1752, United States

Location

1st Allergy and Clinical Research Center

Centennial, Colorado, 80112, United States

Location

Allergy Associates of the Palm Beaches

North Palm Beach, Florida, 33408, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

Rainbow Babies and Children's Hospital

Cleveland, Ohio, 44106, United States

Location

Optimed Research, LLC

Columbus, Ohio, 43235, United States

Location

Pediatric Allergy Immunology Associates

Dallas, Texas, 75230, United States

Location

Allergy, Asthma and Immunology Clinic PA

Irving, Texas, 75230, United States

Location

University of Toronto

Toronto, Ontario, M4V1R2, Canada

Location

The Hospital for Sick Children

Toronto, Ontario, M5G1X8, Canada

Location

Related Publications (8)

• Ten RM. Primary immunodeficiencies. Mayo Clin Proc. 1998 Sep;73(9):865-72. doi: 10.4065/73.9.865.

  PMID: 9737224 BACKGROUND

• Bonilla FA, Geha RS. 12. Primary immunodeficiency diseases. J Allergy Clin Immunol. 2003 Feb;111(2 Suppl):S571-81. doi: 10.1067/mai.2003.86.

  PMID: 12592303 BACKGROUND

• Bonilla FA, Bernstein IL, Khan DA, Ballas ZK, Chinen J, Frank MM, Kobrynski LJ, Levinson AI, Mazer B, Nelson RP Jr, Orange JS, Routes JM, Shearer WT, Sorensen RU; American Academy of Allergy, Asthma and Immunology; American College of Allergy, Asthma and Immunology; Joint Council of Allergy, Asthma and Immunology. Practice parameter for the diagnosis and management of primary immunodeficiency. Ann Allergy Asthma Immunol. 2005 May;94(5 Suppl 1):S1-63. doi: 10.1016/s1081-1206(10)61142-8. No abstract available.

  PMID: 15945566 BACKGROUND

• Chapel HM. Consensus on diagnosis and management of primary antibody deficiencies. Consensus Panel for the Diagnosis and Management of Primary Antibody Deficiencies. BMJ. 1994 Feb 26;308(6928):581-5. doi: 10.1136/bmj.308.6928.581. No abstract available.

  PMID: 8148684 BACKGROUND

• Roifman CM, Levison H, Gelfand EW. High-dose versus low-dose intravenous immunoglobulin in hypogammaglobulinaemia and chronic lung disease. Lancet. 1987 May 9;1(8541):1075-7. doi: 10.1016/s0140-6736(87)90494-6.

  PMID: 2883406 BACKGROUND

• Eijkhout HW, van Der Meer JW, Kallenberg CG, Weening RS, van Dissel JT, Sanders LA, Strengers PF, Nienhuis H, Schellekens PT; Inter-University Working Party for the Study of Immune Deficiencies. The effect of two different dosages of intravenous immunoglobulin on the incidence of recurrent infections in patients with primary hypogammaglobulinemia. A randomized, double-blind, multicenter crossover trial. Ann Intern Med. 2001 Aug 7;135(3):165-74. doi: 10.7326/0003-4819-135-3-200108070-00008.

  PMID: 11487483 BACKGROUND

• Roifman CM, Schroeder H, Berger M, Sorensen R, Ballow M, Buckley RH, Gewurz A, Korenblat P, Sussman G, Lemm G. Comparison of the efficacy of IGIV-C, 10% (caprylate/chromatography) and IGIV-SD, 10% as replacement therapy in primary immune deficiency. A randomized double-blind trial. Int Immunopharmacol. 2003 Sep;3(9):1325-33. doi: 10.1016/s1567-5769(03)00134-6.

  PMID: 12890430 BACKGROUND

• Berger M. A history of immune globulin therapy, from the Harvard crash program to monoclonal antibodies. Curr Allergy Asthma Rep. 2002 Sep;2(5):368-78. doi: 10.1007/s11882-002-0069-z.

  PMID: 12165202 BACKGROUND

MeSH Terms

Conditions

Immunologic Deficiency Syndromes; Bruton type agammaglobulinemia; Common Variable Immunodeficiency; Hyper-IgM Immunodeficiency Syndrome

Interventions

gamma-Globulins

Condition Hierarchy (Ancestors)

Immune System Diseases; Dysgammaglobulinemia; Blood Protein Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Primary Immunodeficiency Diseases

Intervention Hierarchy (Ancestors)

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

Study Officials

• Chaim Roifman, MD

  The Hospital for Sick Children

  STUDY CHAIR

• Robert Roberts, MD

  Mattel Children's Hospital of UCLA

  PRINCIPAL INVESTIGATOR

• Isaac R Melamed, MD

  1st Allergey and Clinical Research Center

  PRINCIPAL INVESTIGATOR

• James Moy, MD

  Rush Universitity Medical Centre

  PRINCIPAL INVESTIGATOR

• Eyal Grunebaum, MD

  The Hospital for Sick Children

  PRINCIPAL INVESTIGATOR

• Gordan L Sussman, MD

  University of Toronto

  PRINCIPAL INVESTIGATOR

• Akhilesh Chouksey, MD

  Rainbow Babies and Children's Hospital

  PRINCIPAL INVESTIGATOR

• Mark Stein, MD

  Allergy Associates of the Palm Beaches

  PRINCIPAL INVESTIGATOR

• Richard L Wasserman, MD

  PRINCIPAL INVESTIGATOR

• Daniel Suez, MD

  Allergy, Asthma and Immunology Clinic PA

  PRINCIPAL INVESTIGATOR

• Don McNeil, MD

  Optimed Research LLC

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 1, 2007
• First Posted: May 2, 2007
• Study Start: November 1, 2006
• Primary Completion: May 1, 2009
• Study Completion: August 1, 2009
• Last Updated: August 11, 2014

Record last verified: 2014-08

Locations

US (8); CA (2)