Home
Search
Study Topics
Glossary
|
|
|
|
|
|
Sponsored by: |
Bio Products Laboratory |
---|---|
Information provided by: | Bio Products Laboratory |
ClinicalTrials.gov Identifier: | NCT00278954 |
The main objective of this study is to see if GAMMAPLEX is efficacious with respect to Food and Drug Administration (FDA) minimal requirements (no more than 1 serious, acute, bacterial infection per subject per year) in subjects with PID. The secondary objectives are to assess the safety and tolerability of GAMMAPLEX and to determine if GAMMAPLEX has a pharmacokinetic (PK) profile comparable with that of intact IgG in subjects with PID.
Condition | Intervention | Phase |
---|---|---|
Primary Antibody Deficiency Common Variable Hypogammaglobulinemia X-Linked Hypogammaglobulinemia Autosomal Hypogammaglobulinemia Immunodeficiency With Hyper-IgM Wiskott-Aldrich Syndrome |
Drug: Intravenous immunoglobulin |
Phase III |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
Estimated Enrollment: | 50 |
Study Start Date: | January 2006 |
Study Completion Date: | November 2007 |
Primary Efficacy Variable The primary variable is the number of serious, acute, bacterial infections/subject/year, and it will be based on the total of all of the following events as defined by the FDA: bacterial Pneumonia, bacteremia/sepsis, osteomyelitis/septic arthritis, visceral abscess, and bacterial meningitis.
Secondary Efficacy Variables Secondary efficacy will be determined by using the following variables: number of days of work/school missed because of infection per subject year; number and days of hospitalizations because of infection per subject year; number of visits to physicians for acute problems and/or number of visits to hospital emergency rooms per subject year; other infections documented by fever or a positive result on a radiograph and/or culture; number of infectious episodes per subject per year; number of days on therapeutic antibiotics.These data will be entered into the subject diary, confirmed by the physician, and entered on the e-CRF.
Safety Variables. The variables used to assess safety will be the following: adverse events (AEs); vital signs; clinical laboratory tests and Direct Coombs' Test; transmission of viruses; physical examination.
Test product, dose/mode of administration, batch number(s): The GAMMAPLEX dose is 300-800 mg/kg/infusion every 21 or 28 days, intravenously. At least 2 batches will be used in this study and no more than 1 batch in any given infusion.
Duration of treatment:
The total duration of treatment is 12 months.
Ages Eligible for Study: | 3 Years and older |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
1. The subject is 3 years of age or older, of either sex, belonging to any ethnic group, and above a minimum weight of 27.5 kg. This weight is based on the amount of blood required for testing. If subject is participating in the PK segment, the minimum weight required is 37 kg.
2. The subject has a primary immunodeficiency disease, which has as a significant component of hypogammaglobulinemia and/or antibody deficiency (e.g., common variable immunodeficiency, X-linked and autosomal forms of agammaglobulinemia, hyper-IgM syndrome, Wiskott-Aldrich Syndrome). Isolated deficiency of a single IgG subclass, or of specific antibodies without hypogammaglobulinemia per se, does not qualify for inclusion.
3. The subject has been receiving licensed or investigational (Phase III or IIIb) IGIV replacement therapy at a dose that has not changed by + 50% of the mean dose for at least 3 months before study entry and is between 300 and 800 mg/kg/infusion. The infusion interval must be between 21 and 28 days inclusive. The subject must have maintained a trough level at least 300 mg/dL above baseline serum IgG levels (defined as before initiation of any gamma globulin treatment for that subject). The trough level must be ³ 600 mg/dL.
4. Trough levels of IgG and dose of IGIV, treatment intervals, and the trade name of the IGIV treatments used for the last 2 consecutive routine (licensed or investigational product) must be documented for each subject before the first infusion in this study can be administered.
5. If a subject is a female of child-bearing potential, she must have a negative result on an HCG-based pregnancy test.
6. If a subject is a female who is or becomes sexually active, she must practice contraception by using a method of proven reliability for the duration of the study.
7. The subject is willing to comply with all aspects of the protocol, including blood sampling, for the duration of the study.
8. The subject has signed an informed consent form (if at least 18 years old) or the subject's parent or legal guardian has signed the informed consent form. If appropriate, the subject has signed a child assent form (See Section 12.3).
Exclusion Criteria:
Subjects will be excluded if any of the following exclusion criteria are met:
The subject is positive for any of the following at screening:
The subject, at screening, has levels greater than 2.5 times the upper limit of normal as defined at the central laboratory of any of the following:
The subject is receiving the following medication:
United States, Florida | |
Allergy Associates of the Palm Beaches | |
North Palm Beach, Florida, United States, 33408 | |
United States, Georgia | |
Family Allergy and Asthma Center PC | |
Atlanta, Georgia, United States, 30342 | |
United States, Illinois | |
Rush University Medical Centre, University Consultants in Allergy & Immunology | |
Chicago, Illinois, United States, 60612 | |
United States, New York | |
Childrens Hospital at Buffalo, Allergy Division | |
Buffalo, New York, United States, 14222 | |
United States, Texas | |
Allergy, Asthma & Immunology Clinic, P.A. | |
Irving, Texas, United States, 75063 | |
UCLA Medical Centre | |
Irving, Texas, United States, 75063 | |
United States, Washington | |
University of Washington School of Medicine, Dept. of Pediatrics | |
Seattle, Washington, United States, 989109-5235 |
Principal Investigator: | Melvin Berger, MD | Rainbow Babies & Children's Hospital, Cleveland, Ohio |
Study ID Numbers: | GMX01 |
Study First Received: | January 18, 2006 |
Last Updated: | November 22, 2007 |
ClinicalTrials.gov Identifier: | NCT00278954 |
Health Authority: | United States: Food and Drug Administration |
Primary Antibody Deficiency Common variable hypogammaglobulinemia X-linked hypogammaglobulinemia |
Autosomal hypogammaglobulinemia Immunodeficiency with hyper-IgM Wiskott-Aldrich Syndrome |
Agammaglobulinemia Common variable immunodeficiency Blood Protein Disorders Hemostatic Disorders Purpura, Thrombocytopenic Wiskott-Aldrich Syndrome Hemorrhagic Disorders Thrombocytopenia Genetic Diseases, X-Linked Rho(D) Immune Globulin Hyperkinesis Wiskott Aldrich syndrome Immunoglobulins |
Purpura T cell immunodeficiency primary Hematologic Diseases Blood Platelet Disorders Blood Coagulation Disorders Immunologic Deficiency Syndromes Lymphatic Diseases Thrombocytopathy Antibodies Genetic Diseases, Inborn Immunoglobulins, Intravenous Lymphoproliferative Disorders Common Variable Immunodeficiency |
Blood Coagulation Disorders, Inherited Pathologic Processes Disease Immunologic Factors |
Immune System Diseases Syndrome Physiological Effects of Drugs Pharmacologic Actions |