Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases.

NCT00278954 | Completed Phase 3 Results

• 1 other identifier: GMX01
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 7

Brief Summary

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 Primary Immunodeficiency Diseases (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 Immunoglobulin G (IgG) in subjects with PID.

Conditions

Primary Immunodeficiency; Common Variable Hypogammaglobulinemia; X-linked Hypogammaglobulinemia; Hypogammaglobulinemia; Immunodeficiency With Hyper-IgM; Wiskott-Aldrich Syndrome

Keywords

Primary Antibody Deficiency; Common variable hypogammaglobulinemia; X-linked hypogammaglobulinemia; Hypogammaglobulinemia; Immunodeficiency with hyper-IgM; Wiskott-Aldrich Syndrome

Outcome Measures

Primary Outcomes (1)

• Number of Serious, Acute, Bacterial Infections (SABIs) Per Subject Per Year in Subjects With Primary Immunodeficiency Disease.

  By assessing the number of serious, acute, bacterial infections per subject per year in subjects with Primary Immunodeficiency disease.

  12 months

Secondary Outcomes (4)

• The Pharmacokinetic (PK) Half-Life of Immunoglobulin G (IgG)

  -5, 0, 60 minutes (min), 24, 48 hours (hrs), 4, 7, 14, 21 and 28 days

• The Pharmacokinetic (PK) Clearance of Immunoglobulin G (IgG)

  -5, 0, 60 min, 24, 48 hrs, 4, 7, 14, 21 and 28 days

• The Pharmacokinetic (PK) Volume of Distribution (Vz)of Immunoglobulin G (IgG)

  -5, 0, 60 min, 24, 48 hrs, 4, 7, 14, 21 and 28 days

• The Pharmacokinetic (PK) Mean Residence Time (MRT) for Inmuunoglobulin G (IgG)

  -5, 0, 60 min, 24, 48 hrs, 4, 7, 14, 21 and 28 days

Study Arms (1)

Gammaplex

OTHER

Gammaplex

Biological: Gammaplex (Intravenous immunoglobulin)

Interventions

Gammaplex (Intravenous immunoglobulin) BIOLOGICAL

GAMMAPLEX 5g/100 mL, dose is 300-800 mg/kg/infusion every 21 or 28 days, intravenously for 12 months.

Gammaplex

Eligibility Criteria

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

You may qualify if:

• \. 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.
• \. The subject has been receiving licensed or investigational (Phase III or IIIb) immunoglobulin intravenous (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 (milligram per decilitre) above baseline serum IgG levels (defined as before initiation of any gamma globulin treatment for that subject). The trough level must be 600 mg/dL.
• \. 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.
• \. If a subject is a female of child-bearing potential, she must have a negative result on an Human Chorionic Gonadotrophin (HCG)-based pregnancy test.
• \. 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.
• \. The subject is willing to comply with all aspects of the protocol, including blood sampling, for the duration of the study.
• \. 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).

You may not qualify if:

• 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 GAMMAPLEX, such as sorbitol (i.e.(id est / that is) , intolerance to fructose).
• The subject has selective immunoglobulin A (IgA) deficiency, history of reaction to products containing IgA, or has a history of antibodies to IgA.
• Subjects who have completed the study and subjects who have withdrawn cannot participate in the study for a second time.
• The subject is currently receiving, or has received, any investigational agent, other than an immune serum globulin (ISG) preparation that is being evaluated in a Phase III or IIIb study, 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 or other forms of commercially available and licensed ISG or an ISG product that is in Phase III or IIIb studies.
• The subject is pregnant or is nursing.
• The subject is positive for any of the following at screening:
• Serological test for Human immunodeficiency virus (HIV) 1\&2, Hepatitis C virus (HCV), or Hepatitis B surface antigen (HBsAg)
• Nucleic Acid test (NAT) for HCV
• NAT for HIV
• 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:
• Alanine transaminase (ALT)
• Aspartate transaminase (AST)
• The subject has a severe renal impairment (defined as serum creatinine greater than 2 times the upper limit of normal or Blood urea nitrogen (BUN) greater than 2.5 times the upper limit of normal for the range of the laboratory doing the analysis); the subject is on dialysis; the subject has a history of acute renal failure.

Sponsors & Collaborators

• Bio Products Laboratory: lead

Study Sites (7)

Allergy Associates of the Palm Beaches

North Palm Beach, Florida, 33408, United States

Location

Family Allergy and Asthma Center PC

Atlanta, Georgia, 30342, United States

Location

Rush University Medical Centre, University Consultants in Allergy & Immunology

Chicago, Illinois, 60612, United States

Location

Childrens Hospital at Buffalo, Allergy Division

Buffalo, New York, 14222, United States

Location

Allergy, Asthma & Immunology Clinic, P.A.

Irving, Texas, 75063, United States

Location

UCLA Medical Centre

Irving, Texas, 75063, United States

Location

University of Washington School of Medicine, Dept. of Pediatrics

Seattle, Washington, 989109-5235, United States

Location

Related Publications (1)

• Geng B, Clark K, Evangelista M, Wolford E. Low rates of headache and migraine associated with intravenous immunoglobulin infusion using a 15-minute rate escalation protocol in 123 patients with primary immunodeficiency. Front Immunol. 2023 Feb 2;13:1075527. doi: 10.3389/fimmu.2022.1075527. eCollection 2022.

  PMID: 36818468 DERIVED

Related Links

• Sponsor website

MeSH Terms

Conditions

Primary Immunodeficiency Diseases; Common Variable Immunodeficiency; Hypogammaglobulinemia, X-Linked; Agammaglobulinemia; Hyper-IgM Immunodeficiency Syndrome; Wiskott-Aldrich Syndrome

Interventions

gamma-Globulins; Immunoglobulins, Intravenous

Condition Hierarchy (Ancestors)

Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Immunologic Deficiency Syndromes; Immune System Diseases; Blood Protein Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Dysgammaglobulinemia; 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; Immunoglobulin G; Immunoglobulin Isotypes; Antibodies

Results Point of Contact

• Title: Margaret Stratford Bobbitt
• Organization: Bio Products Laboratory

Margaret.Stratford-Bobbitt@bpl.co.uk

+44 20 8258 2288

Study Officials

• Melvin Berger, MD

  Rainbow Babies & Children's Hospital, Cleveland, Ohio

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

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

Study Record Dates

• First Submitted: January 18, 2006
• First Posted: January 19, 2006
• Study Start: January 1, 2006
• Primary Completion: November 1, 2007
• Study Completion: November 1, 2007
• Last Updated: January 24, 2013
• Results First Posted: January 28, 2010

Record last verified: 2013-01

Locations

US (7)