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A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary Immunodeficiency

Information source: Bio Products Laboratory
Information obtained from ClinicalTrials.gov on February 07, 2013
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Primary Immune Deficiency Disorders; Common Variable Immunodeficiency; X-linked Agammaglobulinemia; Hyper-IgM Syndrome; Wiskott-Aldrich Syndrome

Intervention: Gammaplex (Biological)

Phase: Phase 4

Status: Recruiting

Sponsored by: Bio Products Laboratory

Official(s) and/or principal investigator(s):
Tim J. Aldwinckle, MD, Study Director, Affiliation: Medical Director

Summary

The main objective is to determine the efficacy of Gammaplex by measuring the number of serious acute bacterial infections during treatment with Gammaplex over a 12 month period. The secondary objectives are to assess the safety and tolerability of Gammaplex and to compare the data collected from adult subjects with PID from the GMX01 study

Clinical Details

Official title: A Phase IV, Multicenter, Open-Label Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases (PID) in Children and Adolescents

Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Prevention

Primary outcome: Adverse events

Secondary outcome:

Therapeutic efficacy

Therapeutic efficacy

Adverse events

Adverse events

Vital signs

Laboratory testing

Assessment of viral safety

Physical examination

Pharmacokinetics

Pharmacokinetics

Interval medical history

Pharmacokinetics

Eligibility

Minimum age: 2 Years. Maximum age: 16 Years. Gender(s): Both.

Criteria:

Inclusion Criteria:

- The subject is between the ages of or is equal to 2 and 16 years of age, 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, 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). NB Isolated deficiency of a single IgG subclass, or of specific antibodies without hypogammaglobulinemia per se, does not qualify for inclusion.

- Subjects already receiving IGIV replacement therapy require the following before

their first infusion of Gammaplex:

- Documented IGIV dose(s) and treatment intervals for the last 2 consecutive routine

IGIV treatments (one of which can be the screening visit result). The previous doses should also meet the following conditions before study entry: Have not changed by ± 50% of the mean dose for at least 3 months; be between 300 and 800 mg/kg/infusion; be given every 21-28 days, inclusive; be a licensed or investigational product (Phase III or IIIb).

- Documented previous IgG trough levels for the last 2 consecutive routine IGIV

treatmentsfor the last 2 consecutive routine IGIV treatments: Maintained at least 300 mg/dL above baseline serum IgG levels (defined as before initiation of any gamma globulin treatment for that subject); must be more than/equal to 600 mg/dL.

- If a subject is a female of child-bearing potential, she must have a negative result

on an 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, if old enough (generally 6 years to 16), has signed a Child Assent Form

and the subject's parent or legal guardian has signed the Informed Consent Form, both approved by the IEC/IRB.

Exclusion Criteria:

- Has not been treated with IGIV (treatment naive subject)

- 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. intolerance to fructose).

- The subject has selective 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. If an unlicensed ISG product that is in Phase III or IIIb has been given, the subject cannot be infused with Gammaplex until 20 days after the last dose was given.

- The 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 of any of the following: (Alanine transaminase (ALT); Aspartate transaminase (AST) Lactate dehydrogenase (LDH)).

- The subject has a severe renal impairment (defined as serum creatinine greater than 2

times the upper limit of normal or 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.

- The subject is known to abuse alcohol, opiates, psychotropic agents, or other

chemicals or drugs, or has done so within the past 12 months.

- 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, 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, chronic or recurrent

neutropenia (ANC < 1000 x 109/L) or AIDS known to cause secondary immune deficiency, or is post or recovering from hematopoietic stem cell transplantation.

- The subject is receiving the following medication: Systemic long-term

corticosteroids (i. e. not intermittent or burst, daily, >1 mg of prednisone equivalent/kg/day).

- The subject is receiving Immunosuppressive or Immunomodulatory drugs.

- The subject has non-controlled arterial hypertension.

- The subject has anemia (hemoglobin <10 g/dL) at screening.

Locations and Contacts

Hospital de Niňos Roberto del Río, Santiago 8380418, Chile; Recruiting
Veronica Saavedra, Phone: 56 2575 8143
Carmen Navarrete, MD, Principal Investigator

Safra Children's Hospital, Sheba Medical Center, Tel-Hashomer 52621, Israel; Recruiting
Hannah Mazurski, Phone: 972 50 626 2087
Raz Somech, MD, Principal Investigator

Department of Medicine, University of California, Irvine, California 92697., United States; Recruiting
Maritza Clawges, Phone: 949-824-2032, Email: mclawges@uci.edu
Sudhir Gupta, MD, Principal Investigator

Children's Hospital Los Angeles, Los Angeles, California 90027, United States; Recruiting
Dalila Ortega, Phone: 323-361-4537, Email: dlopez@chla.usc.edu
Joseph A. Church,, MD, Principal Investigator

IMMUNOe International Reseach Centers, Centennial, Colorado 80112, United States; Recruiting
Andrea Beck, CCRP, Phone: 303-773-9000, Email: ABeck@IMMUNOe.com
Isaac Melamed, MD, Principal Investigator

Family Allergy & Asthma Center, PC, Atlanta,, Georgia 30342, United States; Recruiting
Steven D. Goodman, Phone: 404-255-8080, Ext: 115, Email: goodmans@familyallergycenter.com
Robyn Levy, MD, Principal Investigator

Rush University Medical Center, Chicago,, Illinois 60612, United States; Recruiting
Abbi Brees, CCRC, Phone: 312-942-4913, Email: abrees@rush.edu
James Moy, MD, Principal Investigator

Children's Hospital of Pittsburgh of UPMC, Pittsburgh, Pennsylvania 15224, United States; Recruiting
Elizabeth Hartigan, Phone: 412-692-7060, Email: Elizabeth.hartigan@chp.edu
Todd Green, MD, Principal Investigator

Allergy, Asthma & Immunology Clinic, P.A, Irving, Texas 75063, United States; Recruiting
Catherine McNairn, Phone: 972-401-0545, Email: Catherine@dsallergy.com
Daniel Suez, MD, Principal Investigator

Rocky Mountain Allergy, Asthma & Immunology, Layton, Utah 84041, United States; Withdrawn

Children's Hospital of Richmond, VA, Richmond, Virginia 23219, United States; Recruiting
Whitney Douglas, Phone: 804-828-3284, Email: douglaswr@vcu.edu
Anne-Marie Irani, MD, Principal Investigator

Additional Information

Starting date: March 2011
Last updated: January 23, 2013

Page last updated: February 07, 2013

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